Gene Therapy for Metachromatic Leukodystrophy (MLD)

Status Active, not recruiting

Clinical Trial Summary

This Phase I/II clinical trial consists of the application of lentiviral vector-based gene therapy to patients affected by Metachromatic Leukodystrophy (MLD), a rare inherited Lysosomal Storage Disorder (LSD) resulting from mutations in the gene encoding the Arylsulfatase A (ARSA) enzyme. The medicinal product consists of autologous CD34+ hematopoietic stem/progenitor cells in which a functional ARSA cDNA is introduced by means of 3rd generation VSV-G pseudotyped lentiviral vectors.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT01560182
Study type	Interventional
Source	Orchard Therapeutics
Contact
Status	Active, not recruiting
Phase	Phase 1/Phase 2
Start date	April 9, 2010
Completion date	March 15, 2025

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Withdrawn	`NCT00639132` - The Natural History of Metachromatic Leukodystrophy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
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
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
Completed	`NCT01586455` - Human Placental-Derived Stem Cell Transplantation	Phase 1