19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2019-000667-24-IT (EUCTR) | 28/03/2022 | 23/09/2021 | A Phase I/II Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects with Fabry Disease | A Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease - STAAR | Fabry Disease (X-linked lysosomal storage 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] | Product Name: Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A Product Code: [ST-920] Other descriptive name: Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA Trade Name: PARACETAMOLO DOC GENERICI - 1000 MG COMPRESSA 16 COMPRESSE Product Name: Paracetamolo Product Code: [Paracetamolo] INN or Proposed INN: PARACETAMOLO Trade Name: ALISERIN - 25 MG GRANULATO EFFERVESCENTE 20 BUSTINE Product Name: difenidramina cloridrato Product Code: [difenidramina cloridrato] INN or Proposed INN: DIFENIDRAMINA CLORIDRATO Trade Name: PREDNISONE DOC GENERICI - 5 MG COMPRESSE 10 COMPRESSE IN BLISTER PVC-PVDC/ALU Product Name: Prednisone Product Code: [Prednisone] INN or Proposed INN: PREDNISONE | Sangamo Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 1;Phase 2 | United States;Taiwan;Canada;Australia;Germany;United Kingdom;Italy | ||
| 2 | EUCTR2019-000667-24-GB (EUCTR) | 12/11/2019 | 19/03/2019 | A Phase I/II Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects with Fabry Disease | A Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease | Fabry Disease (X-linked lysosomal storage 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] | Product Name: Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A Product Code: ST-920 INN or Proposed INN: Not yet assigned Other descriptive name: Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA | Sangamo Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 30 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom | ||
| 3 | NCT02995993 (ClinicalTrials.gov) | November 2016 | 14/12/2016 | Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease | An Open-Label, Multi-Center Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGal in Patients With Fabry Disease | Fabry Disease | Drug: Moss-aGal (recombinant human alpha-galactosidase A produced in moss) | Greenovation Biotech GmbH | FGK Clinical Research GmbH | Completed | 18 Years | 65 Years | All | 6 | Phase 1 | Germany |
| 4 | NCT02800070 (ClinicalTrials.gov) | July 2016 | 15/4/2016 | Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease | Clinical Pilot Study of Autologous Stem Cell Transplantation of Cluster of Differentiation 34 Positive (CD34+) Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease | Fabry Disease | Biological: Lentivirus Alpha-gal A transduced stem cells | University Health Network, Toronto | Ozmosis Research Inc. | Active, not recruiting | 18 Years | 50 Years | Male | 5 | Phase 1 | Canada |
| 5 | 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 | ||
| 6 | 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 | ||
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 |
| 11 | 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 |
| 12 | NCT00048906 (ClinicalTrials.gov) | November 2002 | 8/11/2002 | Alpha-Galactosidase A Replacement Therapy for Fabry Disease | A Safety and Pharmacokinetic Study of Replagal Enzyme Replacement Therapy in Patients With Fabry Disease | Fabry Disease | Drug: DRX005B | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | N/A | N/A | Both | 3 | Phase 2 | United States |