Metachromatic Leukodystrophy Clinical Trial
Official title:
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).
OTL-200 is a cryopreserved dispersion for infusion containing autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. MLD is an autosomal recessive lysosomal storage disorder (LSD) characterized by severe and progressive demyelination affecting the central and peripheral nervous system. The aim of this clinical study is to assess the pharmacodynamic effect and long-term clinical efficacy and safety of OTL-200 in Late Juvenile MLD patients.
n/a
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Withdrawn |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
|
||
Active, not recruiting |
NCT03392987 -
A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)
|
Phase 2 | |
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 | |
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 |