Metachromatic Leukodystrophy Clinical Trial
Official title:
and Safety of METAZYM (Recombinant Human Arylsulfatase A or rhASA) for the Treatment of Patients With Late Infantile MLD Who Had Previously Hematopoietic Stem Cell Transplantation
There is currently no effective treatment for late infantile MLD once clinical symptoms are evident. METAZYM is a recombinant human arylsulfatase A developed for an intravenous ERT for the treatment of late infantile MLD. The overall objective of this study is to evaluate the efficacy and safety of intravenous rhASA treatment in a patient with late infantile MLD who had previously received hematopoietic stem cell transplantation (HCT).
Metachromatic Leukodystrophy (MLD) is a rare autosomal recessive disorder caused by the deficiency of the Arylsulfatase A enzyme (ARSA), resulting in accumulation of galactosyl sulfatide (cerebroside sulfate), a major constituent of the myelin sheath. Accumulation of galactosyl sulfatides leads to a progressive degeneration of the white matter in the central and peripheral nervous system (CNS, PNS) and neuronal degeneration. The late infantile form of MLD, which usually is diagnosed in the second year of life, is the most frequent and severe form of the disease. The prognosis is severe, leading to vegetative stage or death within few years after the diagnosis. There is no treatment for patients affected with this early onset form of the disease. In patients with late-onset MLD (juvenile and adult forms), allogeneic hematopoietic stem cell transplantation can stabilize the cerebral demyelination. This treatment is however inefficient in patients with late infantile MLD at a symptomatic stage. The overall objective is to evaluate the efficacy and safety of rhASA treatment in a patient with late infantile MLD who had received HCT at a presymptomatic stage of the disease. Patient will receive rhARSA (100 U/kg) intravenously every other week for a period of 18 months. ;
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT00383448 -
HSCT for High Risk Inherited Inborn Errors
|
Phase 2 | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
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 |
NCT00004378 -
Stem Cell Transplantation (SCT) for Genetic Diseases
|
N/A | |
Completed |
NCT00176904 -
Stem Cell Transplant for Inborn Errors of Metabolism
|
Phase 2/Phase 3 | |
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 |