Clinical Trials Logo
  1. Home
  2. Metachromatic Leukodystrophy
  3. NCT03392987

A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)

Metachromatic Leukodystrophy Clinical Trial

Official title:
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)

Clinical Trial Summary

OTL-200 is autologous CD34+ cells transduced with lentiviral vector containing human arylsulfatase A (ARSA) complementary deoxyribonucleic acid (cDNA) used for the treatment of MLD. MLD is an autosomal recessive lysosomal storage disorder (LSD) characterized by severe and progressive demyelination affecting the central and peripheral nervous system. This study will assess safety and efficacy of treatment using cryopreserved formulation of OTL-200 in pediatric subjects with pre-symptomatic Early Onset MLD (Late Infantile (LI) to Early Juvenile (EJ) MLD) and early symptomatic EJ MLD.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03392987
Study type Interventional
Source Orchard Therapeutics
Contact
Status Active, not recruiting
Phase Phase 2
Start date January 25, 2018
Completion date April 3, 2028
View Details
See also
  Status Clinical Trial Phase
Completed NCT00383448 - HSCT for High Risk Inherited Inborn Errors Phase 2
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Completed NCT01303146 - Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation Phase 2
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Active, not recruiting NCT04628364 - The Natural History of Metachromatic Leukodystrophy Study (HOME Study)
Enrolling by invitation NCT05368038 - ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Withdrawn NCT00639132 - The Natural History of Metachromatic Leukodystrophy
Active, not recruiting NCT01801709 - Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy Phase 1/Phase 2
Completed NCT00683189 - Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy N/A
Recruiting NCT02171104 - MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Phase 2
Active, not recruiting NCT02699190 - LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Recruiting NCT02559830 - Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy Phase 1/Phase 2
Completed NCT00176904 - Stem Cell Transplant for Inborn Errors of Metabolism Phase 2/Phase 3
Completed NCT00004378 - Stem Cell Transplantation (SCT) for Genetic Diseases N/A
Active, not recruiting NCT04283227 - OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) Phase 3
Recruiting NCT03047369 - The Myelin Disorders Biorepository Project
Recruiting NCT04925349 - Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
Active, not recruiting NCT00005900 - Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation N/A
Active, not recruiting NCT01560182 - Gene Therapy for Metachromatic Leukodystrophy (MLD) Phase 1/Phase 2
Completed NCT01586455 - Human Placental-Derived Stem Cell Transplantation Phase 1