19. ライソゾーム病
［臨床試験数：784，薬物数：673（DrugBank：101），標的遺伝子数：68，標的パスウェイ数：184］
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | NCT04532047 (ClinicalTrials.gov) | December 1, 2020 | 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 | Not yet recruiting | 18 Years | 50 Years | Female | 10 | Phase 1 | United States |
| 2 | 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 |
| 3 | 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 |
| 4 | 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 | ||
| 5 | 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 | ||
| 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 |
| 6 | 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 2A | Belgium;United Kingdom | ||
| 7 | 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 | ||
| 8 | 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 | |
| 9 | 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 | |||
| 10 | 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 | ||
| 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 |
| 11 | 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 | ||
| 12 | 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 |
| 13 | 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 | ||
| 14 | 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 | ||
| 15 | 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 | ||
| 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 |
| 16 | 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 | ||
| 17 | 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 | ||
| 18 | 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 | Actelion Pharmaceuticals Ltd | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 4 | Hungary;Czech Republic;Spain;Germany;Italy;United Kingdom | |||
| 19 | 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 |
| 20 | 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 |