19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
| 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 | NCT05067868 (ClinicalTrials.gov) | April 30, 2022 | 24/9/2021 | A Study of Replagal in Children and Adults With Fabry Disease in India | A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Agalsidase Alfa (r-DNA Origin) (Replagal™) in Indian Children and Adults With Fabry Disease | Fabry Disease | Biological: Replagal | Shire | NULL | Not yet recruiting | N/A | N/A | All | 5 | Phase 4 | India |
| 2 | 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 | Recruiting | 8 Years | N/A | All | 22 | Phase 4 | China |
| 3 | EUCTR2018-004689-32-FI (EUCTR) | 12/10/2021 | 17/08/2021 | Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease | A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease | REPLAGAL is intended for use for patients with Fabry disease. MedDRA version: 20.0;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] | Trade Name: REPLAGAL ® Product Name: REPLAGAL Product Code: SHP675 INN or Proposed INN: AGALSIDASE ALFA Other descriptive name: Gene Activated a-Galactosidase A | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 45 | Phase 3 | Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Brazil;Belgium;Poland;Romania;Croatia;Australia;Germany;Latvia;Sweden;Bosnia and Herzegovina | ||
| 4 | EUCTR2018-004689-32-PL (EUCTR) | 08/10/2021 | 07/10/2021 | Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease | A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease | Fabry disease MedDRA version: 24.1;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] | Trade Name: REPLAGAL ® Product Name: REPLAGAL Product Code: SHP675 INN or Proposed INN: AGALSIDASE ALFA Other descriptive name: Gene Activated a-Galactosidase A | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 36 | Phase 3 | Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Belgium;Brazil;Romania;Croatia;Australia;Latvia;Germany;Sweden;Bosnia and Herzegovina | ||
| 5 | EUCTR2018-004689-32-GR (EUCTR) | 06/10/2021 | 06/10/2021 | Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease | A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease | REPLAGAL is intended for use for patients with Fabry disease. MedDRA version: 20.0;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] | Trade Name: REPLAGAL ® Product Name: REPLAGAL Product Code: SHP675 INN or Proposed INN: AGALSIDASE ALFA Other descriptive name: Gene Activated a-Galactosidase A | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 45 | Phase 3 | Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Australia;Germany;Latvia;Sweden;Bosnia and Herzegovina | ||
| 6 | EUCTR2018-004689-32-PT (EUCTR) | 24/09/2021 | 11/08/2021 | Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease | A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease | Fabry disease MedDRA version: 20.0;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] | Trade Name: REPLAGAL ® Product Name: REPLAGAL Product Code: SHP675 INN or Proposed INN: AGALSIDASE ALFA Other descriptive name: Gene Activated a-Galactosidase A | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 45 | Phase 3 | Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Australia;Germany;Latvia;Sweden;Bosnia and Herzegovina | ||
| 7 | EUCTR2018-004689-32-SE (EUCTR) | 24/08/2021 | 17/08/2021 | Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease | A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease | Fabry disease MedDRA version: 20.0;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] | Trade Name: REPLAGAL ® Product Name: REPLAGAL Product Code: SHP675 INN or Proposed INN: AGALSIDASE ALFA Other descriptive name: Gene Activated a-Galactosidase A | Shire Human Genetic Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 45 | Phase 3 | Portugal;Slovenia;Greece;Finland;Spain;Ukraine;Italy;Hungary;Canada;Argentina;Poland;Brazil;Belgium;Romania;Croatia;Australia;Germany;Latvia;Sweden;Bosnia and Herzegovina | ||
| 8 | NCT04143958 (ClinicalTrials.gov) | September 2020 | 28/10/2019 | To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease | A Randomized, Open-label, Active Comparator, 2-arm, Prospective Study to Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease | Fabry's Disease | Drug: agalsidase beta (GZ419828);Drug: agalsidase alfa | Sanofi | NULL | Withdrawn | 16 Years | 45 Years | Male | 0 | Phase 4 | Czechia |
| 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 | Recruiting | 18 Years | N/A | All | 120 | United States | |
| 10 | EUCTR2019-000064-21-GB (EUCTR) | 20/12/2019 | 11/02/2020 | To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfa | A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme®) versus continuing on agalsidase alfa (Replagal) in male patients with classic Fabry disease. - Fabry study with Fabrazyme and Replagal | Fabry's disease MedDRA version: 20.0;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] | Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Trade Name: Replagal Product Name: Replagal INN or Proposed INN: AGALSIDASE ALFA | Sanofi Aventis Groupe (SAG) | NULL | Not Recruiting | Female: no Male: yes | 35 | Phase 4 | France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Norway;Germany;Italy;United Kingdom | ||
| 11 | EUCTR2016-000378-38-FR (EUCTR) | 13/05/2019 | 15/05/2019 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | 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 alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Denmark;Australia;Norway;Netherlands;Germany | ||
| 12 | EUCTR2016-000378-38-FI (EUCTR) | 12/03/2019 | 06/02/2019 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | 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 alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 78 | Phase 3 | United States;Czechia;Slovenia;Finland;Spain;Turkey;United Kingdom;Switzerland;Italy;France;Hungary;Canada;Argentina;Brazil;Belgium;Australia;Denmark;Norway;Germany;Netherlands;Sweden | ||
| 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 | EUCTR2016-001318-11-NL (EUCTR) | 27/03/2018 | 17/10/2017 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With 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 alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Germany;Netherlands;United Kingdom | ||
| 17 | EUCTR2016-001318-11-DE (EUCTR) | 15/12/2017 | 10/01/2017 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With 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 alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 18 | EUCTR2016-000378-38-IT (EUCTR) | 20/11/2017 | 06/02/2018 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta - Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients | 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 alpha Trade Name: FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Trade Name: FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA | PROTALIX LTD | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Spain;Turkey;Italy;United Kingdom;Czech Republic;Hungary;Canada;Belgium;Australia;Germany;Netherlands;Norway | ||
| 19 | EUCTR2016-001318-11-SI (EUCTR) | 13/11/2017 | 05/10/2017 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With 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 alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 20 | 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 | ||
| 21 | NCT03180840 (ClinicalTrials.gov) | September 27, 2017 | 29/5/2017 | Study of the Safety, Efficacy, & PK of Pegunigalsidase Alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients | Phase 3, Open-Label, Switch Over Study to Assess Safety, Efficacy & PK of Pegunigalsidase Alfa 2 mg/kg Administered Every 4 Weeks for 52 Weeks in Fabry Disease Patients Currently Treated With Enzyme Replacement Therapy: Fabrazyme® (Agalsidase Beta) or Replagal™ (Agalsidase Alfa) | Fabry Disease | Biological: Pegunigalsidase alfa | Protalix | NULL | Completed | 18 Years | 60 Years | All | 30 | Phase 3 | United States;Belgium;Canada;Czechia;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom |
| 22 | 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 | ||
| 23 | EUCTR2016-000378-38-SI (EUCTR) | 14/08/2017 | 30/06/2017 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | 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 alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Denmark;Norway;Netherlands;Germany | ||
| 24 | EUCTR2016-000378-38-NL (EUCTR) | 24/05/2017 | 17/01/2017 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | 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 alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Germany | ||
| 25 | 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 |
| 26 | EUCTR2016-001318-11-GB (EUCTR) | 24/03/2017 | 04/10/2016 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;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 alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 27 | NCT03018730 (ClinicalTrials.gov) | February 23, 2017 | 9/1/2017 | Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa) | Fabry Disease | Biological: PRX-102 (pegunigalsidase alfa) | Protalix | NULL | Completed | 18 Years | 60 Years | All | 22 | Phase 3 | Australia;Canada;Czechia;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;Czech Republic |
| 28 | EUCTR2016-001318-11-CZ (EUCTR) | 19/01/2017 | 12/12/2016 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;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 alpha | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 3 | Czech Republic;Slovenia;Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 29 | EUCTR2016-001318-11-ES (EUCTR) | 11/01/2017 | 24/10/2016 | Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa) | An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa) | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;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 alpha | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 22 | Phase 3 | Canada;Spain;Australia;Norway;Netherlands;Germany;United Kingdom | ||
| 30 | EUCTR2016-000378-38-BE (EUCTR) | 05/12/2016 | 30/09/2016 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | 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 alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Germany | ||
| 31 | EUCTR2016-000378-38-HU (EUCTR) | 15/09/2016 | 11/07/2016 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | 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 alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Finland;Spain;Turkey;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Brazil;Australia;Germany;Netherlands;Norway | ||
| 32 | EUCTR2016-000378-38-GB (EUCTR) | 07/09/2016 | 08/07/2016 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | 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 alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Slovenia;Paraguay;Spain;Turkey;United Kingdom;Czech Republic;Hungary;Canada;Argentina;Belgium;Brazil;Australia;Norway;Netherlands;Germany | ||
| 33 | EUCTR2016-000378-38-ES (EUCTR) | 22/08/2016 | 15/07/2016 | Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta | A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta | Fabry disease (a-galactosidase A deficiency) MedDRA version: 19.0;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 alpha Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A Trade Name: Fabrazyme Product Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Other descriptive name: N/A | Protalix Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 78 | Phase 3 | United States;Paraguay;Spain;Turkey;United Kingdom;Czech Republic;Hungary;Canada;Belgium;Australia;Germany;Netherlands;Norway | ||
| 34 | NCT02795676 (ClinicalTrials.gov) | June 2016 | 2/6/2016 | Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function | A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta | Fabry Disease | Biological: PRX-102 (pegunigalsidase alfa);Biological: agalsidase beta | Protalix | NULL | Active, not recruiting | 18 Years | 60 Years | All | 78 | Phase 3 | United States;Argentina;Australia;Belgium;Brazil;Canada;Czechia;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;Czech Republic |
| 35 | 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 | ||
| 36 | 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 | ||
| 37 | NCT01650779 (ClinicalTrials.gov) | April 2012 | 24/7/2012 | A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta | Evaluation of Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (The INFORM Study) | Fabry Disease | Biological: Agalsidase beta | Genzyme, a Sanofi Company | NULL | Completed | N/A | N/A | Male | 15 | Phase 4 | United States |
| 38 | 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 | ||
| 39 | 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 | |||
| 40 | NCT01304277 (ClinicalTrials.gov) | November 17, 2011 | 15/2/2011 | This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes. | A Phase II Comparability Study Between Replagal® Produced From Agalsidase Alfa Manufactured by 2 Different Processes in Adult Male Patients With Fabry Disease | Fabry Disease | Biological: agalsidase alfa | Shire | NULL | Completed | 18 Years | 65 Years | Male | 17 | Phase 2 | Canada |
| 41 | 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 | |||
| 42 | 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 | ||
| 43 | 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 | ||
| 44 | 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 | ||
| 45 | 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 |
| 46 | 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 |
| 47 | 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 | ||
| 48 | 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 | ||
| 49 | 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 |
| 50 | 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 | ||
| 51 | EUCTR2007-005543-22-SI (EUCTR) | 18/03/2011 | 24/02/2011 | An investigation of 3 dosages of Replagal in adult patients with Fabry disease | A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in 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 alpha Other descriptive name: Replagal | Shire Human Genetic Therapies, Inc. (Shire HGT) | NULL | Not Recruiting | Female: yes Male: yes | 43 | United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom | |||
| 52 | NCT01196871 (ClinicalTrials.gov) | February 2, 2011 | 7/9/2010 | Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease | An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Subjects With Fabry Disease | Fabry Disease | Drug: Migalastat HCl;Biological: Agalsidase Beta;Biological: Agalsidase Alfa | Amicus Therapeutics | NULL | Completed | 18 Years | 65 Years | Male | 20 | Phase 2 | United States;Australia;Belgium;Canada;Netherlands;France;United Kingdom |
| 53 | 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 | ||
| 54 | EUCTR2007-005543-22-FI (EUCTR) | 18/05/2010 | 23/03/2010 | An investigation of 3 dosages of Replagal in adult patients with Fabry disease | A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in 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 alpha Other descriptive name: Replagal | Shire Human Genetic Therapies, Inc. (Shire HGT) | NULL | Not Recruiting | Female: yes Male: yes | 43 | Phase 3;Phase 4 | United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom | ||
| 55 | EUCTR2007-005543-22-GB (EUCTR) | 06/05/2010 | 17/08/2009 | An investigation of 3 dosages of Replagal in adult patients 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 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 alpha Other descriptive name: Replagal | Shire Human Genetic Therapies, Inc. (Shire HGT) | NULL | Not Recruiting | Female: yes Male: yes | 43 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): yes | United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom | ||
| 56 | 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 | |||
| 57 | EUCTR2006-001910-33-AT (EUCTR) | 02/03/2010 | 03/02/2010 | A Multicenter, Multinational Study of the Effects of Fabrazyme® (agalsidase beta) Treatment on Lactation and Infants - Effects of Fabrazyme Treatment on Lactation and Infants | A Multicenter, Multinational Study of the Effects of Fabrazyme® (agalsidase beta) Treatment on Lactation and Infants - Effects of Fabrazyme Treatment on Lactation and Infants | Fabry disease MedDRA version: 12.1;Level: PT;Classification code 10016016;Term: Fabry's disease | Trade Name: Fabrazyme Product Name: Fabrazyme Product Code: Agalsidase beta INN or Proposed INN: agalsidase beta Other descriptive name: agalsidase beta | Genzyme Europe BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: no | 10 | United Kingdom;Austria | |||
| 58 | NCT01031173 (ClinicalTrials.gov) | February 17, 2010 | 8/12/2009 | Treatment Protocol of Replagal for Patients With Fabry Disease | An Open-label Treatment Protocol to Evaluate the Safety of Replagal Treatment in Patients With Fabry Disease. | Fabry Disease | Biological: agalsidase alfa | Shire | NULL | No longer available | N/A | N/A | All | United States | ||
| 59 | 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 | |||
| 60 | EUCTR2007-005668-28-FR (EUCTR) | 12/10/2009 | 16/03/2009 | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) | Fabry disease MedDRA version: 9.1;Level: LLT;Classification code 10016016;Term: Fabry's disease | Trade Name: Fabrazyme Product Name: Fabrazyme Product Code: Agalsidase beta INN or Proposed INN: agalsidase beta Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL | Genzyme Europe BV | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 3 | Portugal;France;Czech Republic;Germany;Netherlands;United Kingdom | ||
| 61 | EUCTR2007-005668-28-CZ (EUCTR) | 27/11/2008 | 10/12/2008 | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) | Fabry disease MedDRA version: 9.1;Level: LLT;Classification code 10016016;Term: Fabry's disease | Trade Name: Fabrazyme Product Name: Fabrazyme Product Code: Agalsidase beta INN or Proposed INN: agalsidase beta Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL | Genzyme Europe BV | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 3 | France;Portugal;Czech Republic;Germany;Netherlands;United Kingdom | ||
| 62 | EUCTR2007-005668-28-DE (EUCTR) | 19/11/2008 | 04/08/2008 | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) | Fabry disease MedDRA version: 9.1;Level: LLT;Classification code 10016016;Term: Fabry's disease | Trade Name: Fabrazyme 35 mg Product Name: Fabrazyme Product Code: Agalsidase beta INN or Proposed INN: agalsidase beta Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL Trade Name: Fabrazyme 5 mg Product Name: Fabrazyme Product Code: Agalsidase beta INN or Proposed INN: agalsidase beta Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL | Genzyme Europe BV | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 3 | Portugal;Czech Republic;United Kingdom;Germany;Netherlands;France | ||
| 63 | EUCTR2007-005668-28-PT (EUCTR) | 03/10/2008 | 15/07/2008 | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) | Fabry disease MedDRA version: 9.1;Level: LLT;Classification code 10016016;Term: Fabry's disease | Trade Name: Fabrazyme Product Name: Fabrazyme Product Code: Agalsidase beta INN or Proposed INN: agalsidase beta Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL | Genzyme Europe BV | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 3 | Portugal;Czech Republic;Germany;United Kingdom;Netherlands;France | ||
| 64 | EUCTR2007-005668-28-NL (EUCTR) | 04/09/2008 | 02/06/2008 | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) | Fabry disease MedDRA version: 9.1;Level: LLT;Classification code 10016016;Term: Fabry's disease | Trade Name: Fabrazyme Product Name: Fabrazyme Product Code: Agalsidase beta INN or Proposed INN: agalsidase beta Other descriptive name: recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL | Genzyme Europe BV | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 3 | France;Portugal;Czech Republic;Germany;Netherlands;United Kingdom | ||
| 65 | NCT00701415 (ClinicalTrials.gov) | September 2008 | 17/6/2008 | A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (Agalsidase Beta) in Treatment-Naïve Male Pediatric Patients With Fabry Disease Without Severe Symptoms | Fabry Disease | Biological: Agalsidase beta | Genzyme, a Sanofi Company | NULL | Completed | 5 Years | 18 Years | Male | 31 | Phase 3 | United States;Argentina;Brazil;Canada;Czech Republic;Netherlands;Norway;Poland;United Kingdom;Chile;Portugal |
| 66 | EUCTR2007-005668-28-GB (EUCTR) | 29/07/2008 | 04/04/2008 | A study to assess two alternative dosing regimens of Fabrazyme (agalsidase beta) in boys with Fabry disease who have never received any specific treatment for this disease and who do not have severe symptoms of the disease. | A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms - FIELD (Fabrazyme: Intervening Early at a Lower Dose) | 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Fabrazyme Product Name: Fabrazyme Product Code: Agalsidase beta INN or Proposed INN: agalsidase beta Other descriptive name: recombinant human alpha-galactosidase | Genzyme Europe BV | NULL | Not Recruiting | Female: no Male: yes | 35 | Phase 3 | France;Portugal;United States;Czech Republic;Canada;Argentina;Brazil;Poland;Germany;Netherlands;Norway;United Kingdom | ||
| 67 | EUCTR2007-005543-22-CZ (EUCTR) | 19/06/2008 | 02/01/2008 | An investigation of 3 dosages of Replagal in adult patients with Fabry disease | A Multi-center, Open-Label, Randomized Study Evaluating the Safety andEfficacy of Three Dosing Regimens of Replagal Enzyme ReplacementTherapy in 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 alpha Other descriptive name: Replagal | Shire Human Genetic Therapies, Inc. (Shire HGT) | NULL | Not Recruiting | Female: yes Male: yes | 43 | United States;Czech Republic;Slovenia;Paraguay;Finland;Australia;United Kingdom | |||
| 68 | EUCTR2006-001910-33-GB (EUCTR) | 05/12/2007 | 02/05/2006 | A Multicenter, Multinational Study of the Effects of Fabrazyme® (agalsidase beta) Treatment on Lactation and Infants - Effects of Fabrazyme Treatment on Lactation and Infants | A Multicenter, Multinational Study of the Effects of Fabrazyme® (agalsidase beta) Treatment on Lactation and Infants - Effects of Fabrazyme Treatment on Lactation and Infants | Fabry disease MedDRA version: 9.1;Level: PT;Classification code 10016016;Term: Fabry's disease | Trade Name: Fabrazyme Product Name: Fabrazyme Product Code: Agalsidase beta INN or Proposed INN: agalsidase beta Other descriptive name: agalsidase beta | Genzyme Europe BV | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | United Kingdom;Austria | |||
| 69 | NCT00230607 (ClinicalTrials.gov) | August 30, 2006 | 29/9/2005 | Study of the Effects of Fabrazyme Treatment on Lactation and Infants | A Multicenter, Multinational Study of the Effects of Fabrazyme (Agalsidase Beta) Treatment on Lactation and Infants | Fabry Disease;Alpha Galactosidase A Deficiency | Drug: agalsidase beta | Genzyme, a Sanofi Company | NULL | Recruiting | N/A | N/A | All | 20 | Phase 4 | United States;Austria;United Kingdom |
| 70 | NCT00312767 (ClinicalTrials.gov) | April 2006 | 7/4/2006 | A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency. | A Multicenter, Open-Label, Cross-Over Trial to Evaluate the Pharmacokinetics of Fabrazyme During Simultaneous Fabrazyme Infusion and Chronic Hemodialysis in Patients With Fabry Disease. | Fabry Disease | Drug: Fabrazyme (agalsidase beta) | Genzyme, a Sanofi Company | NULL | Withdrawn | 18 Years | 65 Years | Both | 0 | Phase 4 | United States |
| 71 | NCT00140621 (ClinicalTrials.gov) | July 2005 | 30/8/2005 | A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease | A Multicenter Open-label Study of the Safety and Efficacy of a-galactosidase A (R-h a-GAL) Replacement Therapy in Patients With Cardiac Fabry Disease | Fabry Disease | Drug: Agalsidase beta | Genzyme, a Sanofi Company | NULL | Completed | 20 Years | 64 Years | All | 6 | Phase 4 | Japan |
| 72 | NCT00487630 (ClinicalTrials.gov) | June 2005 | 15/6/2007 | Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease | A Multicenter, Phase 4, Randomized, Controlled Study to Evaluate the Efficacy and Safety of Recombinant Alpha-Galactosidase A (Agalsidase Beta, FABRAZYME) in Heterozygous Females for Fabry Disease | Fabry Disease | Drug: recombinant alpha-galactosidase A | Assistance Publique - Hôpitaux de Paris | NULL | Recruiting | 15 Years | N/A | Female | 34 | Phase 4 | France |
| 73 | 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 |
| 74 | EUCTR2004-000772-14-CZ (EUCTR) | 23/07/2004 | 28/06/2004 | A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients with Fabry Disease (TKT027) - TKT027 | A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients with Fabry Disease (TKT027) - TKT027 | Fabry Disease MedDRA version: 6.1;Level: PT;Classification code 10016016 | Trade Name: Replagal Product Name: Replagal INN or Proposed INN: agalsidase alfa | TKT Inc | NULL | Not Recruiting | Female: no Male: yes | 20 | Phase 1 | Czech Republic | ||
| 75 | 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 |
| 76 | NCT00233870 (ClinicalTrials.gov) | June 2004 | 5/10/2005 | A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease. | Special Survey in Long-Term Use of Fabrazyme | Fabry Disease | Drug: Agalsidase beta (recombinant form) | Genzyme, a Sanofi Company | NULL | Completed | N/A | N/A | Both | 405 | Japan | |
| 77 | NCT00081497 (ClinicalTrials.gov) | January 2004 | 14/4/2004 | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease | Multi-Center, Open-Label Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease That Previously Participated in the AGAL-008-00 Study | Fabry Disease | Biological: agalsidase beta | Genzyme, a Sanofi Company | NULL | Completed | 16 Years | N/A | All | 67 | Phase 4 | United States;Canada;Czech Republic;Hungary;Poland;United Kingdom |
| 78 | 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 |
| 79 | NCT00196716 (ClinicalTrials.gov) | June 2003 | 12/9/2005 | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease | A Multicenter, Open-label Study of Low Dose Maintenance Treatment of Fabrazyme (Recombinant Human Alpha-Galactosidase A (R-h Alpha-GAL)) Replacement Therapy in Patients With Fabry Disease | Fabry Disease | Biological: Fabrazyme (agalsidase beta) | Genzyme, a Sanofi Company | NULL | Completed | 16 Years | N/A | Male | 21 | Phase 2 | Czech Republic;Estonia;Poland;Slovakia |
| 80 | NCT00837824 (ClinicalTrials.gov) | December 2002 | 23/10/2008 | Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme | A Phase 2, Randomized, Open Label, Dose-Ranging, Multiple Dose Study of Fabrazyme® In Patients With Fabry Disease and With Severe Renal Disease | Fabry Disease;Chronic Kidney Disease, Stage IV (Severe) | Biological: Fabrazyme (agalsidase beta) | Genzyme, a Sanofi Company | CRL/Medinet | Terminated | 16 Years | N/A | All | 20 | Phase 2 | United States |
| 81 | NCT00074958 (ClinicalTrials.gov) | October 2002 | 24/12/2003 | A Study of Fabrazyme in Pediatric Patients With Fabry Disease | A Multi-center, Phase 2, Open-Label Study of Fabrazyme (Recombinant Human a-Galactosidase A) Replacement Therapy in Pediatric Patients With Fabry Disease | Fabry Disease | Biological: Fabrazyme (agalsidase beta) | Genzyme, a Sanofi Company | NULL | Completed | 7 Years | 15 Years | All | 16 | Phase 2 | United States;France;Poland;United Kingdom;Italy |
| 82 | NCT00074984 (ClinicalTrials.gov) | February 2001 | 24/12/2003 | A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease | Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Fabrazyme on Progression of Renal Disease and Significant Clinical Events in Patients With Fabry Disease | Fabry Disease | Biological: Fabrazyme (agalsidase beta);Biological: Placebo | Genzyme, a Sanofi Company | NULL | Completed | 16 Years | N/A | All | 82 | Phase 4 | United States;Canada;Czech Republic;Hungary;Poland;United Kingdom |
| 83 | NCT00074971 (ClinicalTrials.gov) | October 1999 | 24/12/2003 | A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease | A Multi-center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients With Fabry Disease | Fabry Disease | Drug: Fabrazyme (agalsidase beta) | Genzyme, a Sanofi Company | NULL | Completed | 16 Years | N/A | Both | 58 | Phase 3 | United States;France;Netherlands;Puerto Rico;United Kingdom |
| 84 | EUCTR2015-000697-35-Outside-EU/EEA (EUCTR) | 24/04/2015 | A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta | Evaluation of Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (The INFORM Study) - The INFORM Study | Fabry Disease MedDRA version: 18.0;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 Name: Agalsidase beta INN or Proposed INN: AGALSIDASE BETA Other descriptive name: AGALSIDASE BETA | Genzyme, a Sanofi Company | NULL | NA | Female: no Male: yes | 15 | United States | ||||
| 85 | EUCTR2019-000064-21-NO (EUCTR) | 15/08/2019 | To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfa | A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical benefits of agalsidase beta (Fabrazyme®) in male patients with classic Fabry disease switching from agalsidase alfa (Replagal®) | Fabry's disease MedDRA version: 20.0;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] | Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA | Sanofi Aventis Groupe (SAG) | NULL | Not Recruiting | Female: no Male: yes | 35 | Phase 4 | France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Germany;Norway;Italy;United Kingdom | |||
| 86 | EUCTR2021-002320-20-ES (EUCTR) | 19/11/2021 | A study to evaluate the effect of venglustat tablets on left ventricular mass index in male and female adult participants with Fabry disease | A randomized, open-label, parallel-group, 18-month Phase 3 study to evaluate the effect of venglustat compared with usual standard of care on left ventricular mass index in participants with Fabry disease and left ventricular hypertrophy | Fabry’s disease MedDRA version: 24.1;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 Name: Venglustat Product Code: SAR402671, GZ402671 or GZ/SAR402671 INN or Proposed INN: Venglustat malate Other descriptive name: GZ/SAR402671 Trade Name: Replagal INN or Proposed INN: Agalsidase alfa Trade Name: Fabrazyme INN or Proposed INN: Agalsidase beta Trade Name: Galafold INN or Proposed INN: Migalastat Other descriptive name: Migalastat | sanofi-aventis recherche et développement | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Czechia;Taiwan;Greece;Spain;Turkey;Austria;United Kingdom;Italy;France;Canada;Poland;Denmark;Norway;Netherlands;Germany;China;Japan;Korea, Republic of | |||
| 87 | EUCTR2019-000064-21-CZ (EUCTR) | 29/08/2019 | To assess the glycosphingolipid clearance and clinical benefits of agalsidase beta in male patients with classic Fabry disease switching from agalsidase alfa | A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme) versus continuing on agalsidase alfa (Replagal) in male patients with classic Fabry disease | Fabry's disease MedDRA version: 20.0;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] | Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Trade Name: Replagal INN or Proposed INN: AGALSIDASE ALFA | Sanofi Aventis Groupe (SAG) | NULL | Not Recruiting | Female: no Male: yes | 35 | Phase 4 | France;Czech Republic;Canada;Spain;Turkey;Denmark;Austria;Norway;Germany;Italy;United Kingdom | |||
| 88 | EUCTR2019-000065-20-NO (EUCTR) | 31/10/2019 | To assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme®) versus continuing on migalastat (Galafold®) in male patients with classic Fabry disease | A randomized, open-label, active comparator, 2-arm, prospective study to assess the glycosphingolipid clearance and clinical effects of switching to agalsidase beta (Fabrazyme®) versus continuing on migalastat (Galafold®) in male patients with classic Fabry disease - BCLEAR2 | Fabry disease MedDRA version: 20.0;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] | Trade Name: Fabrazyme INN or Proposed INN: AGALSIDASE BETA Trade Name: Galafold INN or Proposed INN: MIGALASTAT Other descriptive name: Galafold | Sanofi Aventis Groupe (SAG) | NULL | Not Recruiting | Female: no Male: yes | 35 | Phase 4 | United States;France;Canada;Norway |