19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05222906 (ClinicalTrials.gov) | April 18, 2022 | 20/1/2022 | Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 | A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (GD3) Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ERT) | Gaucher's Disease Type III | Drug: Venglustat;Drug: imiglucerase | Genzyme, a Sanofi Company | NULL | Recruiting | 12 Years | N/A | All | 40 | Phase 3 | United States;Argentina;Canada;China;France;Germany;Hungary;Japan |
| 2 | EUCTR2021-005402-10-DE (EUCTR) | 23/03/2022 | 05/01/2022 | Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 | A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO | Gaucher's disease type III MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion INN or Proposed INN: Imiglucerase | Sanofi-aventis recherche & développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 2 | Phase 3 | Brazil;Germany;China;United States;Taiwan;Turkey;United Kingdom;Egypt;France;Hungary;Canada;Argentina;Japan | ||
| 3 | NCT05152914 (ClinicalTrials.gov) | November 1, 2021 | 29/11/2021 | Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2 | Intravitreal Enzyme Replacement Therapy to Prevent Retinal Disease Progression in Children With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) | Neuronal Ceroid Lipofuscinosis Type 2 | Drug: Cerliponase Alfa | David L Rogers, MD | NULL | Enrolling by invitation | 24 Months | 72 Months | All | 5 | Phase 1/Phase 2 | United States |
| 4 | NCT05134571 (ClinicalTrials.gov) | October 28, 2021 | 21/10/2021 | China Post-marketing Surveillance (PMS) Study of Aldurazyme® | A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Replacement Therapy in Participants With Mucopolysaccharidosis I (MPS I) in China | Mucopolysaccharidosis I | Drug: Laronidase | Genzyme, a Sanofi Company | NULL | Active, not recruiting | 5 Years | N/A | All | 12 | Phase 4 | China |
| 5 | NCT05054387 (ClinicalTrials.gov) | October 13, 2021 | 9/9/2021 | China Post-marketing Surveillance (PMS) Study of Fabrazyme® | A Phase 4, Open Label, Safety and Efficacy Study of Fabrazyme® (Agalsidase Beta) as Enzyme Replacement Therapy in Chinese Participants With Fabry Disease | Fabry's Disease | Drug: Agalsidase beta | Genzyme, a Sanofi Company | NULL | Completed | 8 Years | N/A | All | 22 | Phase 4 | China |
| 6 | JPRN-jRCT2031210279 | 15/09/2021 | 27/08/2021 | An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme replacement therapy for the patient in Japan with acid sphingomyelinase deficiency (ASMD) who has completed Study DFI12712 | An open label interventional single-patient study to evaluate the safety of olipudase alfa enzyme replacement therapy for the patient in Japan with acid sphingomyelinase deficiency (ASMD) who has completed Study DFI12712 | Acid sphingomyelinase deficiency | Drug: Olipudase alfa (GZ402665) Pharmaceutical form: lyophilized powder, Route of administration: Intravenous (IV) infusion. | Tanaka Tomoyuki | NULL | Not Recruiting | >= 20age old | Not applicable | Both | 1 | Phase 2-3 | Japan |
| 7 | NCT04877132 (ClinicalTrials.gov) | July 21, 2021 | 3/5/2021 | Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD) | Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD) | Sphingomyelin Lipidosis | Drug: olipudase alfa (GZ402665) | Sanofi | NULL | Approved for marketing | 3 Years | N/A | All | NULL | ||
| 8 | NCT04532047 (ClinicalTrials.gov) | July 1, 2021 | 19/8/2020 | In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases | In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs). | MPS I;MPS II;MPS IVA;MPS VI;Mps VII;Gaucher Disease, Type 2;Gaucher Disease, Type 3;Pompe Disease Infantile-Onset;Wolman Disease | Drug: Aldurazyme (laronidase) | University of California, San Francisco | Duke University | Recruiting | 18 Years | 50 Years | Female | 10 | Phase 1 | United States |
| 9 | NCT04281537 (ClinicalTrials.gov) | March 1, 2020 | 7/2/2020 | A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy. | A Multi-country Time and Motion Study to Describe the Experience of Clinicians, Patients and Their Caregivers During the Treatment of Fabry Disease With Enzyme Replacement Therapy With Agalsidase Alfa and Agalsidase Beta | Fabry Disease | Drug: Agalsidase Beta;Drug: Agalsidase Alpha | Amicus Therapeutics | NULL | Completed | 18 Years | N/A | All | 76 | United States;Brazil;Japan;Taiwan;Turkey | |
| 10 | JPRN-jRCT2051190085 | 06/01/2020 | 19/12/2019 | A long-term study of beta-glucuronidase enzyme replacement therapy for Sly disease | A long-term, open label, extension study to assess the efficacy and safety of beta-glucuronidase enzyme replacement therapy in Japanese patients with mucopolysaccharidosis type VII, Sly disease | Mucopolysaccharidosis type VII Mucopolysaccharidosis VII, Sly syndrome, beta-glucuronidase, vestronidase alfa;D016538 | Investigational drug: Vestronidase alfa [Recombinant human beta-glucuronidase (rhGUS)], 4 mg/kg, IV, QOW | Hamazaki Takashi | NULL | Recruiting | Not applicable | Not applicable | Both | 4 | Phase 3 | Japan |
| 11 | NCT03950050 (ClinicalTrials.gov) | March 1, 2019 | 6/8/2018 | Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy | Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy | Gaucher Disease, Type 1 | Drug: Ambroxol | Shaare Zedek Medical Center | NULL | Completed | 18 Years | 75 Years | All | 40 | Phase 2 | Israel |
| 12 | JPRN-JMA-IIA00416 | 17/01/2019 | 11/03/2019 | A phase 2 open label study to assess the efficacy and safety of beta-glucuronidase enzyme replacement therapy in Japanese patients with mucopolysaccharidosis type VII, Sly disease | A phase 2 open label study to assess the efficacy and safety of beta-glucuronidase enzyme replacement therapy in Japanese patients with mucopolysaccharidosis type VII, Sly disease | Mucopolysaccharidosis type7 | Intervention type:DRUG. Intervention1:ADMINISTRATION, Dose form:INJECTION, Route of administration:INTRAVENOUS DRIP, intended dose regimen:UX003 will be intravenously administered at dose of 4 mg/kg every other week. UX003 will be sterilely diluted in saline and transferred to infusion bag. Administration of undiluted UX003 is prohibited. The rate of infusion will be determined to administer 2.5% of diluted UX003 for the first one hour and rest of the drug for 3 hours.. Control intervention1:NOT APPLICABLE. | Osaka City University HospitalTakashi Hamazaki | NOT APPLICABLE | Completed | >=4 YEARS | <=40 YEARS | BOTH | 3 | Phase 2 | Japan |
| 13 | EUCTR2017-001528-23-IT (EUCTR) | 17/07/2018 | 10/11/2020 | Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT) | A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme¿ (agalsidase beta) or Replagal¿ (agalsidase alfa) - Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa | PROTALIX LTD | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | United States;Czechia;Taiwan;Spain;Turkey;Austria;Italy;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway | ||
| 14 | EUCTR2017-001528-23-DK (EUCTR) | 04/07/2018 | 10/04/2018 | Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT) | A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | United States;Taiwan;Spain;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway | ||
| 15 | EUCTR2017-001528-23-CZ (EUCTR) | 07/05/2018 | 06/03/2018 | Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT) | A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | United States;Taiwan;Spain;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Denmark;Germany;Netherlands;Norway | ||
| 16 | EUCTR2017-001528-23-ES (EUCTR) | 21/12/2017 | 16/10/2017 | Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT) | A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | United States;Czech Republic;Canada;Belgium;Spain;Turkey;Denmark;Netherlands;Germany;Italy;United Kingdom | ||
| 17 | EUCTR2017-001528-23-GB (EUCTR) | 18/10/2017 | 28/06/2017 | Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT) | A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | United States;Czech Republic;Canada;Spain;Belgium;Turkey;Denmark;Netherlands;Germany;United Kingdom | ||
| 18 | EUCTR2017-001528-23-BE (EUCTR) | 21/08/2017 | 28/07/2017 | Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT) | A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 20.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: Pegunigalsidase alfa Product Code: PRX-102 INN or Proposed INN: Pegunigalsidase alfa | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | United States;Czech Republic;Canada;Spain;Belgium;Turkey;Denmark;Netherlands;Germany;United Kingdom | ||
| 19 | NCT03180840 (ClinicalTrials.gov) | July 10, 2017 | 29/5/2017 | Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks | Phase 3 Open-Label Switch Over Study to Assess Safety, Efficacy & PK of Pegunigalsidase Alfa (PRX-102) 2mg/kg IV Every 4 Weeks for 52 Weeks in Fabry Disease Patients Currently Treated With Enzyme Replacement Therapy Fabrazyme® or Replagal™ | Fabry Disease | Biological: Pegunigalsidase alfa | Protalix | NULL | Completed | 18 Years | 60 Years | All | 30 | Phase 3 | United States;Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;Canada;Netherlands;Spain;Taiwan;Turkey |
| 20 | NCT02956954 (ClinicalTrials.gov) | March 25, 2017 | 3/11/2016 | Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease | Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease (AFD): Impact of Treatment by Agalsidase Alpha (Replagal®) | Anderson-Fabry Disease | Drug: Enzyme replacement therapy (Agalsidase alpha (Replagal®));Procedure: Magnetic Resonance Imaging | University Hospital, Rouen | NULL | Unknown status | 18 Years | N/A | All | 25 | N/A | France |
| 21 | EUCTR2015-000104-26-PT (EUCTR) | 03/10/2016 | 03/05/2016 | A safety, tolerability and efficacy study in MPS 7 patients less than 5 years of age receiving enzyme (UX003) replacement by intravenous injection | An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 Years Old | Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not available Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 15 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Portugal;Spain | ||
| 22 | EUCTR2015-001875-32-PT (EUCTR) | 18/04/2016 | 19/01/2016 | A long-term safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injection | A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects with MPS 7 | Mucopolysaccharidosis type 7 ( MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human beta-glucuronidase (rhGUS) Product Code: UX003 INN or Proposed INN: pending Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 12 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Portugal;Mexico;Brazil | ||
| 23 | EUCTR2015-000104-26-ES (EUCTR) | 05/04/2016 | 03/02/2016 | A safety, tolerability and efficacy study in MPS 7 patients less than 5 years of age receiving enzyme (UX003) replacement by intravenous injection | An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 Years Old | Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not available Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 15 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Portugal;United States;Spain | ||
| 24 | NCT02432144 (ClinicalTrials.gov) | November 10, 2015 | 22/4/2015 | A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) | A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7 | Sly Syndrome;MPS VII;Mucopolysaccharidosis;Mucopolysaccharidosis VII | Drug: UX003 | Ultragenyx Pharmaceutical Inc | NULL | Completed | 5 Years | N/A | All | 12 | Phase 3 | United States;Brazil;Mexico;Portugal |
| 25 | NCT02418455 (ClinicalTrials.gov) | July 21, 2015 | 12/4/2015 | Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age | An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years Old | Sly Syndrome;MPS VII;Mucopolysaccharidosis;Mucopolysaccharidosis VII | Drug: UX003 | Ultragenyx Pharmaceutical Inc | NULL | Completed | N/A | 5 Years | All | 8 | Phase 2 | United States;Portugal;Spain |
| 26 | NCT02520934 (ClinicalTrials.gov) | July 2015 | 6/8/2015 | Miglustat on Gaucher Disease Type IIIB | Evaluation of Combination Therapy With Miglustat and Enzyme Replacement Therapy on Gaucher Disease Type IIIB | Gaucher Disease | Drug: Miglustat;Drug: ERT | National Taiwan University Hospital | Actelion | Active, not recruiting | 6 Years | N/A | All | 19 | N/A | Taiwan |
| 27 | NCT02969200 (ClinicalTrials.gov) | April 2015 | 2/11/2016 | Fabry: Renal Function During Long-term ERT by 51Cr-EDTA Clearance | Fabry Disease: Renal Function During Long-term Enzyme Replacement Therapy Evaluated by Gold Standard GFR 51Cr-EDTA Clearance | Fabry Disease | Drug: Enzyme replacement therapy | Ulla Feldt-Rasmussen | NULL | Completed | N/A | N/A | All | 52 | Denmark | |
| 28 | EUCTR2011-002750-31-CZ (EUCTR) | 16/02/2015 | 22/03/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 20.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: KANUMA Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Germany;Japan;Sweden | ||
| 29 | NCT02930655 (ClinicalTrials.gov) | February 1, 2015 | 10/10/2016 | A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease | A Single-center, Open-label, Randomized, Versus a Control Group, Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Oral Lucerastat in Adult Subjects With Fabry Disease Receiving Enzyme Replacement Therapy | Fabry Disease | Drug: Lucerastat;Drug: Enzyme replacement therapy (ERT) | Idorsia Pharmaceuticals Ltd. | NULL | Completed | 18 Years | N/A | All | 14 | Phase 1 | Germany |
| 30 | NCT02230566 (ClinicalTrials.gov) | December 2014 | 22/8/2014 | A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) | A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7 | MPS 7;Sly Syndrome;Mucopolysaccharidosis;MPS VII | Drug: UX003;Other: Placebo | Ultragenyx Pharmaceutical Inc | NULL | Completed | 5 Years | 35 Years | All | 12 | Phase 3 | United States |
| 31 | NCT02228460 (ClinicalTrials.gov) | November 2014 | 27/8/2014 | Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease | A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed With Fabry Disease | Fabry Disease | Drug: GZ/SAR402671 | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | 49 Years | Male | 11 | Phase 2 | United States;France;Poland;Russian Federation;United Kingdom;Czech Republic;Czechia |
| 32 | EUCTR2013-005324-41-GB (EUCTR) | 07/10/2014 | 30/06/2014 | Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ402671 in Treatment-naïve Adult Male Patients with Fabry Disease | A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed with Fabry Disease | Fabry disease MedDRA version: 17.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: GZ402671 / SAR402671 Other descriptive name: Genz-682452-AA Product Code: GZ402671 / SAR402671 Other descriptive name: Genz-682452-AA | Genzyme Corporation | NULL | Not Recruiting | Female: no Male: yes | 8 | Phase 2 | France;United States;Czech Republic;Poland;Russian Federation;United Kingdom | ||
| 33 | EUCTR2013-005324-41-CZ (EUCTR) | 19/09/2014 | 03/07/2014 | Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ402671 in Treatment-naïve Adult Male Patients with Fabry Disease | A Phase 2 Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Enzyme Replacement Therapy (ERT) Treatment-naïve Adult Male Patients Diagnosed with Fabry Disease | Fabry disease MedDRA version: 17.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: GZ402671 / SAR402671 Other descriptive name: Genz-682452-AA Product Code: GZ402671 / SAR402671 Other descriptive name: Genz-682452-AA | Genzyme Corporation | NULL | Not Recruiting | Female: no Male: yes | 8 | Phase 2 | France;United States;Czech Republic;Poland;Russian Federation;United Kingdom | ||
| 34 | EUCTR2013-001152-35-ES (EUCTR) | 21/08/2014 | 04/07/2014 | A safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injection | An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7 | Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not available Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 5 | Phase 1;Phase 2 | Spain;United Kingdom | ||
| 35 | EUCTR2014-000350-11-DE (EUCTR) | 28/05/2014 | 10/03/2014 | Treatment of patients, who have Mucopolysaccharidosis Type I, receiving pentosan polysulfate subcutaneous injections weekly | An open label, randomized study to investigate the safety of weekly pentosan polysulfate injections in adult patients with Mucopolysaccharidosis Type I receiving enzyme replacement therapy. | Mucopolysaccharidosis type I (alpha-L-Iduronidase deficiency) MedDRA version: 17.0;Level: LLT;Classification code 10056888;Term: Mucopolysaccharidosis IS;System Organ Class: 100000004850 MedDRA version: 17.0;Classification code 10056887;Term: Mucopolysaccharidosis IH/S;Classification code 10028094;Term: Mucopolysaccharidosis IH;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Pentosan Polysulfat SP 54® injection solution Product Name: Pentosan Polysulphate SP54® INN or Proposed INN: PENTOSAN POLYSULFATE SODIUM Other descriptive name: Pentosan Polysulphate SP54 | Multiplex Pharma Holdings LLC | NULL | Not Recruiting | Female: yes Male: yes | 6 | Germany | |||
| 36 | NCT02107846 (ClinicalTrials.gov) | April 2014 | 4/4/2014 | An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112 | A Phase 2a, Open-Label, Sequential Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112 (Plant Recombinant Human Glucocerebrosidase) in Enzyme Replacement Therapy-Naïve Subjects With Gaucher Disease | Gaucher Disease | Drug: PRX-112 | Protalix | NULL | Completed | 18 Years | N/A | Both | 10 | Phase 2 | Israel |
| 37 | EUCTR2013-001152-35-GB (EUCTR) | 02/08/2013 | 25/06/2013 | A safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injection | An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7 | Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not available Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | Not Recruiting | Female: yes Male: yes | 5 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Spain;United Kingdom | ||
| 38 | EUCTR2011-002750-31-HR (EUCTR) | 17/07/2013 | 21/05/2014 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 17.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corporation | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | Taiwan;Greece;Spain;Ukraine;Israel;Chile;Russian Federation;Italy;France;Cyprus;Denmark;Australia;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Croatia;Romania;Germany;Sweden | ||
| 39 | EUCTR2011-002750-31-GR (EUCTR) | 26/06/2013 | 15/05/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 16.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corporation | NULL | Not Recruiting | Female: yes Male: yes | 55 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Germany;Sweden | ||
| 40 | EUCTR2011-002750-31-DE (EUCTR) | 29/05/2013 | 29/01/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 20.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004915 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Kanuma Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Germany;Japan;Sweden | ||
| 41 | EUCTR2011-002750-31-ES (EUCTR) | 06/05/2013 | 06/03/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 15.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corporation | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Belgium;Poland;Brazil;Romania;Croatia;Germany;Sweden | ||
| 42 | EUCTR2011-002750-31-IT (EUCTR) | 15/04/2013 | 25/02/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 14.1;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corporation | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | Taiwan;Greece;Spain;Ukraine;Chile;Israel;Russian Federation;Italy;France;Cyprus;Denmark;Australia;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Croatia;Romania;Germany;Sweden | ||
| 43 | EUCTR2011-002750-31-GB (EUCTR) | 18/03/2013 | 11/02/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 19.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: KANUMA Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | United States;Taiwan;Greece;Spain;Ukraine;Chile;Israel;Russian Federation;Italy;France;Cyprus;Denmark;Australia;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Croatia;Romania;Germany;Japan;Sweden | ||
| 44 | NCT01842841 (ClinicalTrials.gov) | March 2013 | 11/4/2013 | Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease | A Multicenter, Open-label Extension Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease | Gaucher Disease | Drug: velaglucerase alfa | Shire | Quintiles, Inc. | Completed | 2 Years | N/A | All | 5 | Phase 3 | Japan |
| 45 | NCT01653444 (ClinicalTrials.gov) | November 2012 | 24/7/2012 | Evaluate the Safety and Exploratory Efficacy of GC1119 | Multicenter and Dose Escalation Phase 1 Study to Evaluate the Safety and Exploratory Efficacy of GC1119(Recombinant Human a-galactosidase A) for Enzyme Replacement Therapy in Fabry Disease Patients | Fabry Disease | Drug: GC1119 | Green Cross Corporation | NULL | Completed | 16 Years | N/A | Male | 8 | Phase 1 | Korea, Republic of |
| 46 | NCT01685216 (ClinicalTrials.gov) | September 2012 | 10/9/2012 | Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease | A Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease | Gaucher Disease, Type 3 | Biological: velaglucerase alfa | Shire | NULL | Completed | 2 Years | 17 Years | All | 7 | Phase 1/Phase 2 | Egypt;India;Tunisia |
| 47 | EUCTR2010-022709-16-GB (EUCTR) | 23/08/2012 | 21/05/2012 | A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT). | AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE | Fabry disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 2 | Belgium;United Kingdom | ||
| 48 | NCT01645189 (ClinicalTrials.gov) | July 2012 | 16/7/2012 | Safety and Efficacy of Hunterase | To Evaluate the Safety and Efficacy of Hunterase(Idursulfase-beta) in Hunter Syndrome Patients < 6 Years of Age Receiving Idursulfase Enzyme Replacement Therapy | Hunter Syndrome | Biological: Hunterase | Green Cross Corporation | NULL | Completed | N/A | 5 Years | Male | 6 | Phase 3 | Korea, Republic of |
| 49 | EUCTR2010-022636-37-DE (EUCTR) | 09/05/2012 | 10/01/2012 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 15.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Hungary;Germany;Turkey;Switzerland;France;Italy;Austria;Australia;Brazil;Israel;United Kingdom;Slovakia;Russian Federation;Taiwan;Mexico;Argentina;Belgium;Denmark;Japan;United States;Greece;Poland | ||
| 50 | NCT01572636 (ClinicalTrials.gov) | March 28, 2012 | 4/4/2012 | Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome | MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH). | Mucopolysaccharidosis Type IH;MPS I;Hurler Syndrome | Drug: Laronidase | Masonic Cancer Center, University of Minnesota | NULL | Terminated | N/A | N/A | All | 20 | United States | |
| 51 | EUCTR2009-015985-75-SI (EUCTR) | 22/03/2012 | 05/03/2012 | Follow on study in adult Fabry Disease patients | An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease | Fabry disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA | Shire Human Genetic Therapies (HGT), Inc. | NULL | Not Recruiting | Female: yes Male: yes | 43 | Phase 3;Phase 4 | United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom | ||
| 52 | NCT01614574 (ClinicalTrials.gov) | March 2, 2012 | 6/6/2012 | Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease | A Multicenter, Open-Label Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease | Gaucher Disease | Biological: velaglucerase alfa | Shire | NULL | Completed | 2 Years | N/A | All | 6 | Phase 3 | Japan |
| 53 | EUCTR2010-022636-37-IT (EUCTR) | 16/12/2011 | 13/03/2012 | A Randomized, Open-Label, Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT - AT1001-012 | A Randomized, Open-Label, Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT - AT1001-012 | Fabry disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrocloride Product Code: AT1001 INN or Proposed INN: migalastat hydrocloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Trade Name: REPLAGAL INN or Proposed INN: Agalsidase alfa Other descriptive name: NA Trade Name: FABRAZYME INN or Proposed INN: Agalsidase beta Other descriptive name: NA | AMICUS THERAPEUTICS, INC | NULL | Not Recruiting | Female: yes Male: yes | 50 | United States;Taiwan;Greece;Turkey;Austria;Russian Federation;Italy;Switzerland;United Kingdom;Mexico;Argentina;Brazil;Belgium;Denmark;Australia;Germany;Japan | |||
| 54 | EUCTR2009-015985-75-FI (EUCTR) | 15/11/2011 | 26/10/2011 | Follow on study in adault Fabry Disease patients | An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease | Fabry disease MedDRA version: 16.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA | Shire Human Genetic Therapies (HGT), Inc. | NULL | Not Recruiting | Female: yes Male: yes | 43 | United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom | |||
| 55 | EUCTR2010-022636-37-GR (EUCTR) | 02/11/2011 | 23/09/2011 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Slovakia;Greece;Turkey;Austria;Israel;Russian Federation;Italy;Switzerland;United Kingdom;France;Hungary;Mexico;Argentina;Brazil;Poland;Belgium;Denmark;Australia;Germany;Japan | ||
| 56 | EUCTR2009-015985-75-GB (EUCTR) | 18/10/2011 | 19/05/2011 | Follow on study in adault Fabry Disease patients | An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease | Fabry disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA | Shire Human Genetic Therapies (HGT), Inc. | NULL | Not Recruiting | Female: yes Male: yes | 43 | Phase 3;Phase 4 | United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom | ||
| 57 | EUCTR2010-022709-16-BE (EUCTR) | 12/09/2011 | 10/08/2011 | A study to find out if an investigational new drug called AT1001 can help people with Fabry disease and if it is safe for use in combination with enzyme replacement therapy (ERT). | AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE | Fabry disease MedDRA version: 14.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 2 | France;Canada;Belgium;Australia;Netherlands;United Kingdom;Switzerland | ||
| 58 | NCT01218659 (ClinicalTrials.gov) | September 8, 2011 | 6/10/2010 | Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease | A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT | Fabry Disease | Drug: migalastat hydrochloride;Biological: agalsidase | Amicus Therapeutics | NULL | Completed | 16 Years | 74 Years | All | 68 | Phase 3 | United States;Australia;Austria;Belgium;Brazil;Denmark;France;Italy;Japan;United Kingdom;Argentina;Germany;Greece;Poland;Switzerland;Taiwan;Turkey |
| 59 | NCT01298141 (ClinicalTrials.gov) | August 10, 2011 | 15/2/2011 | A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease | A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal® (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease | Fabry Disease | Biological: agalsidase alfa | Shire | NULL | Completed | N/A | N/A | All | 171 | Phase 3 | Canada |
| 60 | EUCTR2010-022636-37-AT (EUCTR) | 03/08/2011 | 27/07/2011 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 16.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | France;United States;Greece;Brazil;Belgium;Denmark;Australia;Austria;Germany;Japan;Italy;United Kingdom | ||
| 61 | EUCTR2010-022636-37-DK (EUCTR) | 03/05/2011 | 06/04/2011 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | United States;France;Greece;Belgium;Brazil;Austria;Australia;Denmark;Germany;United Kingdom;Japan;Italy | ||
| 62 | NCT01363492 (ClinicalTrials.gov) | May 2011 | 31/3/2011 | Safety Study of Replagal® Therapy in Children With Fabry Disease | An Open-Label Clinical Trial of Replagal® Enzyme Replacement Therapy in Children With Fabry Disease Who Are Naive to Enzyme Replacement Therapy | Fabry Disease | Biological: Replagal (agalsidase alfa) | Shire | NULL | Completed | 7 Years | 17 Years | All | 15 | Phase 2 | United States |
| 63 | EUCTR2010-022636-37-GB (EUCTR) | 15/04/2011 | 22/12/2010 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | United States;France;Greece;Belgium;Brazil;Denmark;Austria;Australia;Germany;Japan;Italy;United Kingdom | ||
| 64 | EUCTR2010-022636-37-BE (EUCTR) | 18/01/2011 | 16/12/2010 | The ATTRACT Study is a phase 3 clinical study that will measure theeffectiveness and safety of a new investigational medication, when compared to Enzyme Replacement Therapy, for the treatment of patients with Fabry disease | A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT - ATTRACT | Fabry disease MedDRA version: 16.1;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: migalastat hydrochloride Product Code: AT1001 INN or Proposed INN: migalastat hydrochloride Other descriptive name: 1-deoxygalactonojirimycin hydrochloride Product Name: Inactive Reminder Capsules Product Code: Inactive Reminder Capsules INN or Proposed INN: Not applicable Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | Amicus Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 68 | Phase 3 | United States;France;Greece;Brazil;Belgium;Denmark;Austria;Australia;Germany;United Kingdom;Japan;Italy | ||
| 65 | NCT01124643 (ClinicalTrials.gov) | April 13, 2010 | 23/4/2010 | Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease | An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal® Enzyme Replacement Therapy Administered to Adult Patients With Fabry Disease | Fabry Disease | Biological: Replagal | Shire | NULL | Completed | 18 Years | N/A | All | 35 | Phase 3 | United States;Australia;Czechia;Finland;Poland;Slovenia;United Kingdom;Czech Republic;Paraguay |
| 66 | EUCTR2009-015985-75-CZ (EUCTR) | 08/03/2010 | 24/02/2010 | Follow on study in adult Fabry Disease patients | An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease | Fabry disease MedDRA version: 16.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA | Shire Human Genetic Therapies (HGT), Inc. | NULL | Not Recruiting | Female: yes Male: yes | 43 | United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom | |||
| 67 | EUCTR2009-015985-75-PL (EUCTR) | 03/02/2010 | 30/11/2009 | Follow on study in adault Fabry Disease patients | An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease | Fabry disease MedDRA version: 14.0;Level: PT;Classification code 10016016;Term: Fabry's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA | Shire Human Genetic Therapies (HGT), Inc. | NULL | Not Recruiting | Female: yes Male: yes | 43 | United States;Czech Republic;Slovenia;Paraguay;Finland;Poland;Australia;United Kingdom | |||
| 68 | EUCTR2008-005223-28-DE (EUCTR) | 21/01/2010 | 11/08/2009 | A Study of Eliglustat Tartrate (Genz-112638) in Patients with Gaucher Disease Who Have Reached Thereapeutic Goals with Enzyme Replacement Therapy | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have reached therapeutic goals with enzyme replacement therapy - ENCORE | Gaucher Disease type I MedDRA version: 18.0;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Trade Name: Cerezyme Product Name: Cerezyme Product Code: imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | United States;Spain;Turkey;Russian Federation;United Kingdom;Italy;Egypt;France;Czech Republic;Canada;Argentina;Brazil;Australia;Netherlands;Germany | ||
| 69 | EUCTR2008-005825-12-GB (EUCTR) | 17/12/2009 | 28/04/2009 | A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | Gaucher Disease MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: UPLYSO Product Code: Taliglucerase alfa Other descriptive name: recombinant human glucocerebrosidase (prGCD) | Protalix Biotherapeutics | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Spain;Germany;Italy;United Kingdom | ||
| 70 | EUCTR2008-005825-12-DE (EUCTR) | 10/12/2009 | 16/09/2009 | A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | Gaucher Disease MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: UPLYSO Product Code: Taliglucerase alfa INN or Proposed INN: Taliglucerase alfa Other descriptive name: Recombinant Human Glucocerebrosidase (prGCD) | Protalix Biotherapeutics | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | Spain;Germany;Italy;United Kingdom | ||
| 71 | EUCTR2008-005223-28-GB (EUCTR) | 16/11/2009 | 06/07/2009 | A Study of Eliglustat Tartrate (Genz-112638) in Patients with Gaucher Disease Who Have Reached Thereapeutic Goals with Enzyme Replacement Therapy | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCORE | Gaucher Disease type I MedDRA version: 14.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Trade Name: Cerezyme Product Name: Cerezyme Product Code: imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Russian Federation;Italy;United Kingdom;Egypt;France;Czech Republic;Canada;Argentina;Brazil;Australia;Netherlands;Germany | ||
| 72 | EUCTR2008-005223-28-NL (EUCTR) | 04/11/2009 | 01/07/2009 | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCORE | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic Goals with Enzyme Replacement Therapy - ENCORE | Gaucher Disease type I MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat Other descriptive name: not available Trade Name: Cerezyme Product Name: Cerezyme Product Code: Imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: Recombinant human derived macrophage-targeted ß-Glucocerebrosidase | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 186 | Phase 3 | France;Czech Republic;Spain;Germany;Netherlands;Italy;United Kingdom | ||
| 73 | EUCTR2008-005223-28-CZ (EUCTR) | 23/09/2009 | 21/08/2009 | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic goals with Enzyme Replacement Therapy - ENCORE | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1 who have Reached Therapeutic goals with Enzyme Replacement Therapy - ENCORE | Gaucher Disease type I MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: Genz-112638 Product Code: Genz-112638 INN or Proposed INN: Eliglustat tartrate Other descriptive name: not available Trade Name: Cerezyme Product Name: Cerezyme Product Code: Imiglucerase INN or Proposed INN: IMIGLUCERASE Other descriptive name: recombinant human derived macrophage-targeted ß-glucocerebrosidase | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 186 | Phase 3 | Germany;United Kingdom;Czech Republic;Netherlands;France;Spain;Italy | ||
| 74 | NCT00943111 (ClinicalTrials.gov) | September 2009 | 20/7/2009 | A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) | A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) | Gaucher Disease, Type 1 | Drug: Eliglustat tartrate;Drug: Imiglucerase | Genzyme, a Sanofi Company | NULL | Completed | 18 Years | N/A | All | 160 | Phase 3 | United States;Argentina;Australia;Brazil;Canada;Egypt;France;Germany;Italy;Russian Federation;Spain;United Kingdom;Czech Republic;Netherlands;Turkey |
| 75 | EUCTR2008-005825-12-IT (EUCTR) | 28/07/2009 | 16/09/2009 | : A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme) Enzyme Replacement Therapy - PB-06-002 | : A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme) Enzyme Replacement Therapy - PB-06-002 | Gaucher disease MedDRA version: 9.1;Level: SOC;Classification code 10029205 | Product Name: Plant Cell Recombinant Glucocerebrosidase Product Code: prGCD | PROTALIX BIOTHERAPEUTICS | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 3 | Germany;United Kingdom;Spain;Italy | ||
| 76 | EUCTR2008-005825-12-ES (EUCTR) | 04/06/2009 | 06/03/2009 | Ensayo clinico de fase 3, multicéntrico, en abierto y con cambio de tratamiento para evaluar la seguridad y eficacia de Glucocerebrosidasa Humana Recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher tratados con Imiglucerasa (Cerezyme®) como terapia de reemplazo enzimatico. A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | Ensayo clinico de fase 3, multicéntrico, en abierto y con cambio de tratamiento para evaluar la seguridad y eficacia de Glucocerebrosidasa Humana Recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher tratados con Imiglucerasa (Cerezyme®) como terapia de reemplazo enzimatico. A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA | Enfermedad de GaucherGaucher Disease MedDRA version: 9.1;Level: PT;Classification code 10018048;Term: Gaucher's disease | Product Name: recombinant human glucocerebrosidase Product Code: prGCD Other descriptive name: recombinant human glucocerebrosidase | Protalix Biotherapeutics | NULL | Not Recruiting | Female: yes Male: yes | 15 | Phase 3 | Germany;United Kingdom;Spain;Italy | ||
| 77 | NCT00864851 (ClinicalTrials.gov) | December 29, 2008 | 18/3/2009 | Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease | A Multi-Center, Open-Label, Randomized Study Evaluating the Safety and Efficacy of Three Dosing Regimens of Replagal Enzyme Replacement Therapy in Adult Patients With Fabry Disease | Fabry Disease | Biological: Replagal | Shire | NULL | Completed | 18 Years | N/A | All | 44 | Phase 3 | United States;Australia;Czechia;Finland;Paraguay;Poland;Slovenia;United Kingdom;Czech Republic |
| 78 | EUCTR2008-001965-27-ES (EUCTR) | 23/12/2008 | 17/09/2008 | Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease | Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease | Enfermedad de Gaucher de tipo IType I Gaucher disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase | Shire Human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 102 | Spain;United Kingdom | |||
| 79 | EUCTR2008-001965-27-GB (EUCTR) | 29/10/2008 | 15/07/2008 | An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease | An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease | Type I Gaucher disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene-Activated Human Glucocerebrosidase 400U/vial Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa Product Name: Gene-Activated Human Glucocerebrosidase 200U/vial Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase, velaglucerase alfa | Shire Human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 102 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Spain;United Kingdom | ||
| 80 | NCT00882921 (ClinicalTrials.gov) | October 14, 2008 | 16/4/2009 | An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients | A Multi-Center Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients Enrolled in the Hunter Outcome Survey (HOS) Receiving Idursulfase Enzyme Replacement Therapy | Hunter Syndrome | Biological: Idursulfase | Shire | NULL | Completed | 5 Years | N/A | Male | 26 | United States;Brazil;United Kingdom | |
| 81 | NCT00741338 (ClinicalTrials.gov) | September 2008 | 13/8/2008 | Immune Tolerance Study With Aldurazyme® (Laronidase) | A Trial of Antigen-specific Immune Tolerance Induction in Mucopolysaccharidosis I (MPS I) Patients Initiating Enzyme Replacement Therapy With Aldurazyme® (Laronidase) | Mucopolysaccharidosis I | Biological: Laronidase;Drug: Cyclosporine A (CsA);Drug: Azathioprine (Aza) | Genzyme, a Sanofi Company | BioMarin/Genzyme LLC | Completed | N/A | 5 Years | All | 7 | Phase 1/Phase 2 | Brazil;Russian Federation;Ukraine |
| 82 | NCT00635427 (ClinicalTrials.gov) | May 2008 | 6/3/2008 | An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease | An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease | Gaucher Disease, Type 1 | Biological: VPRIV® | Shire | NULL | Completed | 2 Years | N/A | All | 95 | Phase 3 | United States;Argentina;India;Israel;Korea, Republic of;Paraguay;Poland;Russian Federation;Spain;Tunisia;United Kingdom |
| 83 | EUCTR2006-006304-11-IT (EUCTR) | 14/04/2008 | 04/08/2008 | A Multicenter Open-Label Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Previously Treated with Imiglucerase - ND | A Multicenter Open-Label Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Previously Treated with Imiglucerase - ND | Patients with type 1 Gaucher disease who previously received imiglucerase MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB | SHIRE HUMAN GENETIC THERAPIES, INC. | NULL | Not Recruiting | Female: yes Male: yes | 40 | United Kingdom;Spain;Italy | |||
| 84 | EUCTR2007-002840-21-IT (EUCTR) | 25/02/2008 | 15/04/2008 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - GCB039 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - GCB039 | Patients with Gaucher disease of type 1 MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene Activated Human Glucocerebrosidase Product Code: GA-GCB Product Name: imiglucerasi INN or Proposed INN: Imiglucerase | SHIRE HUMAN GENETIC THERAPIES, INC. | NULL | Not Recruiting | Female: yes Male: yes | 32 | United Kingdom;Spain;Italy | |||
| 85 | NCT00638547 (ClinicalTrials.gov) | January 2, 2008 | 11/3/2008 | Intrathecal Enzyme Replacement for Hurler Syndrome | Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome) | Hurler Syndrome | Drug: IRT Laronidase | Masonic Cancer Center, University of Minnesota | NULL | Completed | 6 Months | 3 Years | All | 26 | Phase 1 | United States |
| 86 | NCT00553631 (ClinicalTrials.gov) | January 2008 | 1/11/2007 | Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared With Imiglucerase in Patients With Type I Gaucher Disease | Gaucher Disease, Type 1 | Biological: velaglucerase alfa;Biological: imiglucerase | Shire | NULL | Completed | 2 Years | N/A | All | 34 | Phase 3 | United States;Argentina;India;Israel;Paraguay;Russian Federation;Spain;Tunisia;United Kingdom |
| 87 | NCT00786968 (ClinicalTrials.gov) | January 2008 | 17/6/2008 | Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I | An Extension Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I | Spinal Cord Compression;Mucopolysaccharidosis I;Hurler-Scheie Syndrome;Scheie Syndrome;Lysosomal Storage Disease | Drug: laronidase | Patricia I. Dickson, M.D. | The Ryan Foundation for MPS Children | Terminated | 8 Years | N/A | Both | 3 | Phase 1 | United States;Finland |
| 88 | EUCTR2005-001651-37-HU (EUCTR) | 10/12/2007 | 04/10/2007 | Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. | Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. | Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 18 of the protocol:1BACKGROUND AND RATIONALE1.1Disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Trade Name: Zavesca Product Name: Miglustat Product Code: OGT918 INN or Proposed INN: miglustat | Actelion Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy | |||
| 89 | NCT00607386 (ClinicalTrials.gov) | December 2007 | 22/1/2008 | Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy | A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy | Hunter Syndrome;Mucopolysaccharidosis II;MPS II | Biological: Idursulfase | Shire | Covance;PharmaNet;PRA Health Sciences | Completed | N/A | 5 Years | Male | 28 | Phase 4 | Brazil;Poland;Taiwan |
| 90 | EUCTR2007-002840-21-GB (EUCTR) | 29/11/2007 | 11/10/2007 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - HGT-GCB-039 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher Disease - HGT-GCB-039 | Type I Gaucher disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene Activated Human glucocerebrosidase Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase Trade Name: Cerezyme Product Name: Cerezyme INN or Proposed INN: IMIGLUCERASE Trade Name: Cerezyme Product Name: Cerezyme INN or Proposed INN: IMIGLUCERASE | Shire Human Genetic Therapies, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 32 | United Kingdom;Spain;Italy | |||
| 91 | EUCTR2007-002840-21-ES (EUCTR) | 30/10/2007 | 18/07/2007 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1 - GCB039 | A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1 - GCB039 | Type I Gaucher diseaseEnfermedad de Gaucher tipo 1 MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene Activated Human glucocerebrosidase Product Code: GA-GCB Other descriptive name: Gene activated human glucocerebrosidase Trade Name: Cerezyme INN or Proposed INN: IMIGLUCERASE | Shire Human Genetic Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | 32 | Phase 3 | Spain;Italy;United Kingdom | ||
| 92 | EUCTR2006-006304-11-ES (EUCTR) | 27/08/2007 | 04/03/2010 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa - TKT034 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa - TKT034 | Type I Gaucher DiseaseEnfermedad de Gaucher de Tipo I MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB Other descriptive name: Gene activated human glucocerebrosidase | Shire Human Genetic Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2;Phase 3 | Spain;Germany;Italy;United Kingdom | ||
| 93 | NCT00478647 (ClinicalTrials.gov) | July 25, 2007 | 23/5/2007 | Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase | A Multicenter Open-Label Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase | Gaucher Disease | Biological: GA-GCB (velaglucerase alfa) | Shire | NULL | Completed | 2 Years | N/A | All | 40 | Phase 2/Phase 3 | United States;Israel;Poland;Spain;United Kingdom |
| 94 | EUCTR2006-006304-11-GB (EUCTR) | 03/07/2007 | 12/02/2007 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase - TKT034 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase - TKT034 | Type I Gaucher Disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB Other descriptive name: Gene activated human glucocerebrosidase | Shire Human Genetic Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Human pharmacology (Phase 1): Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): | Spain;Germany;Italy;United Kingdom | ||
| 95 | EUCTR2006-005216-27-FI (EUCTR) | 26/03/2007 | 14/03/2007 | Selkäytimeen annettu entsyymikorvaus mukopolysakkaridoosi I:n selkäytimen puristuman hoidossa. Kansainvälinen monikeskustutkimus. A study of intrathecal enzyme replacement therapy for spinal cord compression in mucopolysaccharidosis I, MIRC-001. - Intrathecal enzyme replacement in MPS I | Selkäytimeen annettu entsyymikorvaus mukopolysakkaridoosi I:n selkäytimen puristuman hoidossa. Kansainvälinen monikeskustutkimus. A study of intrathecal enzyme replacement therapy for spinal cord compression in mucopolysaccharidosis I, MIRC-001. - Intrathecal enzyme replacement in MPS I | Mucopolysaccharidosis I H/S, lysosomal storage disease with clinical manifestations such as progressive joint stiffness, growth retardation, corneal clouding, hepatosplenomegaly, cardiac and respiratory dysfunction, and in severe fore, mental retardation MedDRA version: 9.1;Level: LLT;Classification code 10056887;Term: Mucopolysaccharidosis IH/S | Trade Name: Aldurazyme Other descriptive name: LARONIDASE | HUS, Hospital for Children and Adolescents | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Finland | |||
| 96 | NCT00433147 (ClinicalTrials.gov) | March 23, 2007 | 7/2/2007 | A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy | A Randomized, Open-label Study to Assess the Safety and Tolerability of Multiple Dose Levels and Multiple Dosing Regimens of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy | Gaucher Disease, Type 1;Type 1 Gaucher Disease;Gaucher Disease | Drug: Afegostat tartrate | Amicus Therapeutics | NULL | Completed | 18 Years | 74 Years | All | 30 | Phase 2 | United States |
| 97 | NCT00430625 (ClinicalTrials.gov) | February 15, 2007 | 1/2/2007 | A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease | A Multicenter, Randomized, Double-Blind, Parallel Group, Two-Dose Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease | Gaucher Disease, Type 1 | Biological: VPRIV ®, | Shire | NULL | Completed | 2 Years | N/A | All | 25 | Phase 3 | Argentina;Israel;Paraguay;Russian Federation;Tunisia;United States |
| 98 | NCT00410566 (ClinicalTrials.gov) | December 2006 | 11/12/2006 | Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) | A Phase I, Single-Center, Single Dose, Dose Escalation Study of Recombinant Human Acid Sphingomyelinase (rhASM) in Adults With Acid Sphingomyelinase Deficiency (ASMD) | Acid Sphingomyelinase Deficiency;Niemann-Pick Disease | Drug: rhASM | Genzyme, a Sanofi Company | NULL | Terminated | 18 Years | 65 Years | Both | 11 | Phase 1 | United States |
| 99 | EUCTR2005-001651-37-CZ (EUCTR) | 02/10/2006 | 17/08/2006 | Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. - MAINTENANCE | Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and tolerability of oral miglustat as a maintenance therapy after a switch from enzyme replacement therapy in adult patients with stable type 1 Gaucher disease. - MAINTENANCE | Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:1BACKGROUND AND RATIONALE1.1Disease MedDRA version: 8.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Trade Name: Zavesca Product Name: Miglustat Product Code: OGT918 INN or Proposed INN: miglustat | Actelion Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Hungary;Germany;United Kingdom;Czech Republic;Spain;Italy | |||
| 100 | NCT00962260 (ClinicalTrials.gov) | July 2006 | 18/8/2009 | Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease | An Open-label Expanded Access Trial of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease Who Require Enzyme Replacement Therapy | Gaucher Disease | Drug: Plant cell expressed recombinant glucocerebrosidase (prGCD) | Pfizer | NULL | No longer available | 18 Years | N/A | All | United States;Israel | ||
| 101 | EUCTR2005-001651-37-ES (EUCTR) | 12/04/2006 | 25/01/2006 | A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease | A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease | Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:1BACKGROUND AND RATIONALE1.1Disease | Trade Name: Zavesca Product Name: Miglustat Product Code: OGT918 INN or Proposed INN: miglustat | Actelion Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 4 | Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy | ||
| 102 | EUCTR2005-001651-37-DE (EUCTR) | 28/02/2006 | 16/05/2006 | Open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance therapy after a switch from Enzyme Replacement Therapy in adult patients with stable Type 1 Gaucher disease - MAINTENANCE | Open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance therapy after a switch from Enzyme Replacement Therapy in adult patients with stable Type 1 Gaucher disease - MAINTENANCE | Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 18 of the protocol:1BACKGROUND AND RATIONALE1.1Disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Trade Name: ZAVESCA Product Name: Miglustat Product Code: OGT918 INN or Proposed INN: miglustat | Actelion Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Hungary;Czech Republic;Spain;Germany;Italy;United Kingdom | |||
| 103 | EUCTR2005-001651-37-IT (EUCTR) | 06/02/2006 | 14/03/2006 | A phase IV, open label, non comparative, multicenter study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch form enzyme replacement therapy ERT in adult patients with stable type 1 Gaucher disease - ND | A phase IV, open label, non comparative, multicenter study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch form enzyme replacement therapy ERT in adult patients with stable type 1 Gaucher disease - ND | Gaucher disease is the most common of the glycosphingolipids storage disease and has autosomal recessive inheritance. MedDRA version: 6.1;Level: PT;Classification code 10018048 | Trade Name: ZAVESCA 84CPS 100MG INN or Proposed INN: Miglustat | Actelion Registration Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 4 | Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy | ||
| 104 | NCT00319046 (ClinicalTrials.gov) | February 1, 2006 | 26/4/2006 | Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease | Open-label, Non Comparative, Multi-center Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Oral Miglustat as a Maintenance Therapy After a Switch From Enzyme Replacement Therapy in Adult Patients With Stable Type 1 Gaucher Disease | Gaucher Disease Type 1 | Drug: Miglustat | Actelion | NULL | Completed | 18 Years | N/A | All | 42 | Phase 3 | United States;Australia;Brazil;Canada;Czechia;France;Germany;Hungary;Italy;Netherlands;Spain;Taiwan;United Kingdom;Czech Republic |
| 105 | NCT00215527 (ClinicalTrials.gov) | November 2005 | 19/9/2005 | Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I | A Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I | Mucopolysaccharidosis I;Lysosomal Storage Diseases;Spinal Cord Compression | Drug: laronidase | Patricia I. Dickson, M.D. | Ryan Foundation for MPS Children;University of California, Los Angeles;FDA Office of Orphan Products Development | Terminated | 8 Years | N/A | Both | 4 | Phase 1 | United States;Finland |
| 106 | EUCTR2005-001651-37-GB (EUCTR) | 27/10/2005 | 03/10/2005 | A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease | A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease | Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol: 1BACKGROUND AND RATIONALE 1.1Disease | Trade Name: Zavesca Product Name: Miglustat Product Code: OGT918 INN or Proposed INN: miglustat | Actelion Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | Hungary;Czech Republic;Spain;Germany;Italy;United Kingdom | ||
| 107 | EUCTR2004-002743-27-IT (EUCTR) | 26/07/2005 | 10/08/2005 | An open-label extension of study TKT024 evaluating long-term safety and clinical outcomes in MPS II patients receiving iduronate-2-sulfatase enzyme replacement therapy. | An open-label extension of study TKT024 evaluating long-term safety and clinical outcomes in MPS II patients receiving iduronate-2-sulfatase enzyme replacement therapy. | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 6.1;Level: PT;Classification code 10056889 | Product Name: Idursulfase Product Code: I2S | TKT INC | NULL | Not Recruiting | Female: no Male: yes | 94 | United Kingdom;Germany;Spain;Italy;Sweden | |||
| 108 | EUCTR2004-002743-27-SE (EUCTR) | 11/05/2005 | 17/03/2005 | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 6.1;Level: PT;Classification code 10056889 | Product Name: Idursulfase (I2S) Other descriptive name: Idursulfase | Shire Human Genetic Therapies INC. | NULL | Not Recruiting | Female: no Male: yes | 96 | United Kingdom;Germany;Spain;Italy;Sweden | |||
| 109 | EUCTR2004-002743-27-ES (EUCTR) | 21/04/2005 | 04/04/2006 | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 6.1;Level: PT;Classification code 10056889 | Product Name: Idursulfase (I2S) Other descriptive name: Idursulfase | TKT Inc. | NULL | Not Recruiting | Female: no Male: yes | 94 | Phase 3 | Spain;Germany;Italy;United Kingdom;Sweden | ||
| 110 | NCT00391625 (ClinicalTrials.gov) | February 2005 | 20/10/2006 | Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT) | An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy | Gaucher Disease | Drug: GA-GCB | Shire | NULL | Completed | 18 Years | N/A | All | 10 | Phase 1/Phase 2 | Israel;Romania;Serbia;Former Serbia and Montenegro |
| 111 | EUCTR2004-002743-27-GB (EUCTR) | 31/01/2005 | 23/02/2005 | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 6.1;Level: PT;Classification code 10056889 | Product Name: Idursulfase (I2S) Other descriptive name: Idursulfase | TKT Inc. | NULL | Not Recruiting | Female: no Male: yes | 94 | Phase 3 | Spain;Germany;Italy;United Kingdom;Sweden | ||
| 112 | EUCTR2004-002743-27-DE (EUCTR) | 23/12/2004 | 30/11/2004 | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 6.1;Level: PT;Classification code 10056889 | Product Name: Idursulfase (I2S) Other descriptive name: Idursulfase | Shire Human Genetic Therapies INC. | NULL | Not Recruiting | Female: no Male: yes | 96 | United Kingdom;Germany;Spain;Italy;Sweden | |||
| 113 | NCT00097890 (ClinicalTrials.gov) | November 2004 | 30/11/2004 | Replagal Enzyme Replacement Therapy for Adults With Fabry Disease | An Open Label Six-Month Maintenance Clinical Trial of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease Who Have Completed TKT027 | Fabry Disease | Drug: Replagal (Agalsidase Alfa);Drug: Replagal | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | N/A | N/A | Male | 25 | Phase 4 | United States |
| 114 | NCT00630747 (ClinicalTrials.gov) | September 2004 | 28/2/2008 | Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase | An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy | Hunter Syndrome;Mucopolysaccharidosis II (MPS II) | Biological: Idursulfase | Shire | NULL | Completed | 5 Years | N/A | Male | 94 | Phase 2/Phase 3 | United States;Brazil;Canada;France;Germany;Italy;Romania;Spain;Sweden;United Kingdom |
| 115 | NCT00084084 (ClinicalTrials.gov) | June 2004 | 5/6/2004 | Replagal Enzyme Replacement Therapy for Children With Fabry Disease | An Open Label Clinical Trial of Replagal Enzyme Replacement Therapy In Children With Fabry Disease Who Have Completed Study TKT023 or Who Are Naive to Enzyme Replacement Therapy | Fabry Disease | Drug: Agalsidase alfa | Shire | NULL | Completed | 7 Years | 17 Years | All | 17 | Phase 2 | United States;Canada |
| 116 | NCT00176891 (ClinicalTrials.gov) | March 2004 | 12/9/2005 | Stem Cell Transplant w/Laronidase for Hurler | Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH) | Mucopolysaccharidosis I;Hurler Syndrome | Procedure: Stem Cell Transplant;Drug: Laronidase ERT | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 7 Years | All | 25 | Phase 2 | United States |
| 117 | NCT00357786 (ClinicalTrials.gov) | October 2003 | 26/7/2006 | An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease | An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal® (Registered Trademark) in Patients With Fabry Disease | Fabry Disease | Drug: Replagal agalsidase alfa;Drug: Replagal | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | 39 Years | 45 Years | Male | 3 | Phase 1 | United States |
| 118 | NCT00071877 (ClinicalTrials.gov) | October 2003 | 3/11/2003 | An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease | A Clinical Trial of Replagal Enzyme Replacement Therapy in Children Ages 7 - 17 Years With Fabry Disease | Fabry Disease | Drug: Replagal | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | N/A | N/A | Both | 25 | Phase 2 | United States |
| 119 | NCT00069641 (ClinicalTrials.gov) | September 2003 | 29/9/2003 | Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) | A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II | Mucopolysaccharidosis II | Biological: Iduronate-2-sulfatase enzyme replacement therapy;Biological: iduronate-2-sulfatase enzyme replacement therapy;Biological: Placebo | Shire | NULL | Completed | 5 Years | 25 Years | Male | 96 | Phase 2/Phase 3 | United Kingdom;Brazil;Germany;United States |
| 120 | NCT00048906 (ClinicalTrials.gov) | November 2002 | 8/11/2002 | Alpha-Galactosidase A Replacement Therapy for Fabry Disease | A Safety and Pharmacokinetic Study of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease | Fabry Disease | Drug: DRX005B | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | N/A | N/A | Both | 3 | Phase 2 | United States |
| 121 | EUCTR2012-003427-38-Outside-EU/EEA (EUCTR) | 07/04/2016 | Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease | A Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease | Type 3 Gaucher disease MedDRA version: 18.1;Level: PT;Classification code 10075697;Term: Gaucher's disease type I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: VPRIV Product Name: Gene-Activated Human Glucocerebrosidase 400U/vial Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: VELAGLUCERASE ALFA | Shire Human Genetic Therapies | NULL | NA | Female: yes Male: yes | 6 | Tunisia;Egypt;India | ||||
| 122 | EUCTR2008-005825-12-Outside-EU/EEA (EUCTR) | 10/04/2012 | A multi-centre, open-label, switchover trial to assess safety and efficacy of taliglucerase alfa in adult and paediatric patients with Gaucher disease who are currently being treated with imiglucerase enzyme replacement therapy (ERT). | A multi-centre, open-label, switchover trial to assess safety and efficacy of taliglucerase alfa in adult and paediatric patients with Gaucher disease who are currently being treated with imiglucerase enzyme replacement therapy (ERT). | Gaucher disease MedDRA version: 14.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Taliglucerase alfa - Recombinant human glucocerebrosidase Product Code: prGCD Other descriptive name: TALIGLUCERASE ALFA | Protalix Biotherapeutics | NULL | NA | Female: yes Male: yes | 30 | Canada;Israel;United Kingdom;United States | ||||
| 123 | EUCTR2014-005638-71-Outside-EU/EEA (EUCTR) | 22/12/2014 | An efficacy and safety study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injection | A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7 | Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not available Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | NA | Female: yes Male: yes | 12 | Phase 3 | United States | |||
| 124 | EUCTR2015-000104-26-Outside-EU/EEA (EUCTR) | 23/07/2015 | A safety and efficacy study in young MPS 7 patients (less than 5 years old) receiving enzyme (UX003) replacement by intravenous injection | An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 years old | Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human betaglucuronidase Product Code: UX003 INN or Proposed INN: pending Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | NA | Female: yes Male: yes | 7 | United States | ||||
| 125 | EUCTR2011-002750-31-PL (EUCTR) | 03/04/2013 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 20.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: KANUMA Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | United States;Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Romania;Croatia;Germany;Japan;Sweden | |||
| 126 | EUCTR2015-001875-32-Outside-EU/EEA (EUCTR) | 12/11/2015 | A long-term safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injection | A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects with MPS 7 | Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: pending Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS | Ultragenyx Pharmaceutical Inc. | NULL | NA | Female: yes Male: yes | 12 | United States | ||||
| 127 | EUCTR2007-006044-22-PL (EUCTR) | 21/04/2008 | A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy - not available | A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy - not available | Mucopolysaccharidosis Type II (MPS II or Hunter Syndrome) MedDRA version: 10.1;Level: PT;Classification code 10056889;Term: | Trade Name: Elaprase 2mg/ml Product Name: Idursulfase (I2S) Product Code: I2S INN or Proposed INN: Idursulfase Other descriptive name: Idursulfase | Shire Human Genetic Therapies, Inc. (Shire HGT) | NULL | Not Recruiting | Female: no Male: yes | 30 | Phase 4 | Poland | |||
| 128 | EUCTR2007-000498-42-Outside-EU/EEA (EUCTR) | 10/04/2012 | A double-blind, randomised efficacy and safety study of taliglucerase alfa enzyme replacement therapy in children and adolescents with Gaucher disease (non-neuronopathic and chronic neuronopathic) | A double-blind, randomised efficacy and safety study of taliglucerase alfa enzyme replacement therapy in children and adolescents with Gaucher disease (non-neuronopathic and chronic neuronopathic) | Gaucher disease MedDRA version: 14.1;Level: PT;Classification code 10018048;Term: Gaucher's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Taliglucerase alfa - Recombinant human glucocerebrosidase Product Code: prGCD Other descriptive name: TALIGLUCERASE ALFA | Protalix Biotherapeutics | NULL | NA | Female: yes Male: yes | 10 | Israel;United States | ||||
| 129 | EUCTR2006-006304-11-DE (EUCTR) | 02/11/2007 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase. - TKT034 | A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase. - TKT034 | Type I Gaucher Disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease | Product Name: Gene-Activated Human Glucocerebrosidase Product Code: GA-GCB INN or Proposed INN: velaglucerase alfa Other descriptive name: Gene activated human glucocerebrosidase | Shire Human Genetic Therapies Inc | NULL | Not Recruiting | Female: yes Male: yes | 40 | Phase 2;Phase 3 | Spain;Germany;Italy;United Kingdom | |||
| 130 | EUCTR2011-002750-31-FR (EUCTR) | 12/11/2018 | A study of SBC-102 (enzyme replacement therapy) in patients with lysosomal acid lipase deficiency | A multicenter, randomized, placebo-controlled study of SBC-102 in patients with lysosomal acid lipase deficiency - ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | Lysosomal Acid Lipase Deficiency MedDRA version: 20.0;Level: HLT;Classification code 10024579;Term: Lysosomal storage disorders;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: sebelipase alfa Product Code: SBC-102 INN or Proposed INN: NA Other descriptive name: lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha | Synageva BioPharma Corporation | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | Taiwan;Greece;Spain;Ukraine;Russian Federation;Chile;Israel;Italy;France;Cyprus;Australia;Denmark;South Africa;Netherlands;Turkey;United Kingdom;Czech Republic;Mexico;Argentina;Brazil;Belgium;Poland;Croatia;Romania;Germany;Sweden | |||
| 131 | EUCTR2007-001163-30-Outside-EU/EEA (EUCTR) | 11/05/2015 | A trial of antigen-specific immune tolerance induction in mucopolysaccharidosis I (MPS I) patients initiating enzyme replacement therapy with Aldurazyme® (laronidase) | A trial of antigen-specific immune tolerance induction in mucopolysaccharidosis I (MPS I) patients initiating enzyme replacement therapy with Aldurazyme® (laronidase) | Mucopolysaccharidosis I MedDRA version: 18.0;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Laronidase INN or Proposed INN: LARONIDASE | Genzyme, a Sanofi Company | NULL | NA | Female: yes Male: yes | 12 | Brazil;Russian Federation | ||||
| 132 | EUCTR2021-005402-10-FR (EUCTR) | 27/01/2022 | Study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 | A phase 3, multicenter, multinational, randomized, double-blind, double-dummy, active-comparator study to evaluate the efficacy and safety of venglustat in adult and pediatric patients with Gaucher disease Type 3 (GD3) who have reached therapeutic goals with Enzyme Replacement Therapy (ERT) - LEAP2MONO | Gaucher's disease type III MedDRA version: 24.1;Level: PT;Classification code 10075699;Term: Gaucher's disease type III;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Product Name: venglustat Product Code: SAR402671, GZ402671 or GZ / SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Trade Name: Cerezyme 400 Units Powder for concentrate for solution for infusion INN or Proposed INN: Imiglucerase | Sanofi-aventis recherche & développement | NULL | NA | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Turkey;United Kingdom;France;Egypt;Hungary;Canada;Argentina;Brazil;Germany;China;Japan |