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 | EUCTR2019-004645-32-DE (EUCTR) | 25/04/2022 | 17/12/2021 | A Long-term Follow-up Study of Fabry Disease Subjects Treated with FLT190 (MARVEL 2) | A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects with Fabry Disease - Marvel 2 | 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] | Product Name: FLT190 Product Code: FLT190 INN or Proposed INN: NA Other descriptive name: FLT190 | Freeline Therapeutics Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 50 | Phase 2 | France;United States;Denmark;Austria;Norway;Germany;United Kingdom;Italy | ||
| 2 | EUCTR2018-002097-51-AT (EUCTR) | 02/12/2021 | 24/03/2021 | A Fabry Disease Gene Therapy Study (MARVEL 1) | A Phase 1/2, Baseline-controlled, Non-randomized, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry disease - MARVEL1 | 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] | Product Name: FLT190 Product Code: FLT190 INN or Proposed INN: NA Other descriptive name: FLT190 | Freeline Therapeutics Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 1;Phase 2 | United States;France;Denmark;Austria;Norway;Germany;United Kingdom;Switzerland;Italy | ||
| 3 | EUCTR2018-002097-51-DK (EUCTR) | 04/12/2020 | 08/07/2019 | A Fabry Disease Gene Therapy Study | A Phase 1/2, Baseline-controlled, Non-randomised, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry disease - MARVEL1 | 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] | Product Name: FLT190 Product Code: FLT190 INN or Proposed INN: NA Other descriptive name: FLT190 | Freeline Therapeutics Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 12 | Phase 1;Phase 2 | United States;France;Spain;Denmark;Norway;Germany;United Kingdom;Italy | ||
| 4 | NCT04455230 (ClinicalTrials.gov) | September 8, 2020 | 29/6/2020 | A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 | A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry Disease | Fabry Disease;Lysosomal Storage Diseases | Genetic: FLT190 | Freeline Therapeutics | NULL | Enrolling by invitation | 18 Years | N/A | Male | 50 | Phase 1/Phase 2 | Germany;United Kingdom |
| 5 | EUCTR2019-004645-32-GB (EUCTR) | 24/03/2020 | 31/12/2019 | A Long-term Follow-up Study of Fabry Disease Subjects Treated with FLT190 (MARVEL 2) | A Multicentre, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects with Fabry Disease - Marvel 2 | 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] | Product Name: FLT190 Product Code: FLT190 INN or Proposed INN: NA Other descriptive name: FLT190 | Freeline Therapeutics Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 2 | United States;France;Denmark;Norway;Germany;Italy;United Kingdom | ||
| 6 | EUCTR2018-002097-51-DE (EUCTR) | 12/02/2020 | 28/02/2019 | A Fabry Disease Gene Therapy Study | A Phase 1/2, Baseline-controlled, Non-randomised, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry disease - MARVEL1 | 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] | Product Name: FLT190 Product Code: FLT190 INN or Proposed INN: NA Other descriptive name: FLT190 | Freeline Therapeutics Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 12 | Phase 1;Phase 2 | Austria;Norway;United States;Denmark;Italy;United Kingdom;France;Germany;Spain | ||
| 7 | NCT04040049 (ClinicalTrials.gov) | July 8, 2019 | 26/7/2019 | A Fabry Disease Gene Therapy Study | A Phase 1/2, Baseline-controlled, Non-randomized, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry Disease | Fabry Disease;Lysosomal Storage Diseases | Genetic: FLT190 | Freeline Therapeutics | NULL | Recruiting | 18 Years | N/A | Male | 15 | Phase 1/Phase 2 | United States;Austria;Canada;Germany;Italy;Norway;United Kingdom |
| 8 | EUCTR2018-002097-51-IT (EUCTR) | 26/06/2019 | 19/01/2021 | A Fabry Disease Gene Therapy Study (MARVEL 1) | A Phase 1/2, Baseline-controlled, Non-randomised, Open-label, Singleascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry disease - MARVEL1 | 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] | Product Name: FLT190 Product Code: [FLT190] Other descriptive name: FLT190 | Freeline Therapeutics Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 12 | Phase 1;Phase 2 | France;United States;Denmark;Norway;Germany;United Kingdom;Italy |