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 | NCT04283227 (ClinicalTrials.gov) | January 17, 2022 | 13/2/2020 | OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) | An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD). | Lysosomal Storage Diseases;Metachromatic Leukodystrophy | Genetic: OTL-200 | Orchard Therapeutics | Ospedale San Raffaele | Recruiting | N/A | N/A | All | 6 | Phase 3 | Italy |
| 2 | EUCTR2019-002636-82-IT (EUCTR) | 02/12/2019 | 30/07/2019 | A clinical trial to evaluate the effect of OTL-200, a gene therapy treatment in patients with Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD) | An open label, non-randomised trial to evaluate the safety and efficacy of a single infusion of OTL-200 in patients with Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD) | Metachromatic Leukodystrophy MedDRA version: 20.0;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: OTL-200 INN or Proposed INN: Not Applicable Other descriptive name: OTL-200 Dispersion for Infusion | Orchard Therapeutics (Europe) Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 6 | Phase 3 | Italy | ||
| 3 | NCT03392987 (ClinicalTrials.gov) | January 25, 2018 | 12/12/2017 | A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) | A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD) | Lysosomal Storage Disease;Metachromatic Leukodystrophy | Genetic: OTL-200 | Orchard Therapeutics | Ospedale San Raffaele | Active, not recruiting | N/A | 6 Years | All | 10 | Phase 2 | Italy |
| 4 | EUCTR2017-001730-26-IT (EUCTR) | 30/11/2017 | 08/04/2019 | Gene therapy study using a frozen formulation of OTL-200 in patients with Metachromatic Leukodystrophy (MLD). | A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with lentiviral vector containing human ARSA cDNA OTL-200, for the treatment of early onset Metachromatic Leukodystrophy (MLD). - A clinical study using cryopreserved OTL-200 for treatment of MLD. | Metachromatic Leukodystrophy MedDRA version: 20.0;Level: PT;Classification code 10067609;Term: Metachromatic leukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: OTL-200 Dispersion for Infusion Product Code: [OTL-200] | Orchard Therapeutics (Europe) Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 3 | Italy | ||
| 5 | NCT01560182 (ClinicalTrials.gov) | April 9, 2010 | 16/3/2012 | Gene Therapy for Metachromatic Leukodystrophy (MLD) | A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy | Lysosomal Storage Disease;Metachromatic Leukodystrophy | Genetic: OTL-200 Gene Therapy | Orchard Therapeutics | Ospedale San Raffaele | Active, not recruiting | N/A | 7 Years | All | 20 | Phase 1/Phase 2 | Italy |