Metachromatic Leukodystrophy Clinical Trial
Official title:
Single Patient Expanded Access Protocol: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
This is a single patient expanded access protocol to investigate the effects of a second dose of facilitating cell-enhanced hematopoietic stem cell product.
Hematopoietic stem cell transplantation (HSCT) has been established as an important
therapeutic option for patients with a variety of inherited metabolic disorders (IMD). The
potential life-threatening complications of conventional myeloablative HSCT have limited its
application. Additionally, conventional HSCT is only available to the small minority of
medically suitable candidates who have histocompatibility leukocyte antigen (HLA)-identical
siblings to donate bone marrow or mobilized peripheral blood stem cells.
Donor mobilized peripheral blood stem cells or bone marrow will be processed via a novel
technology to deplete mature immune cells while retaining hematopoietic stem cells (HSC) and
graft facilitating cells (FC).
A now-standard reduced intensity, nonmyeloablative recipient conditioning regimen will be
used to promote mixed allogeneic chimerism, thereby significantly reducing morbidity and
mortality.
These two enhancements are intended to significantly improve the benefit:risk ratio of HSCT
for patients with IMDs. If successful, transplantation will become a more feasible option for
patients without HLA-identical siblings to donate stem cells, and could be offered to
patients early in disease progression.
The objective for the study is to establish chimerism following reduced intensity
conditioning with no grade III/IV graft-versus-host disease (GVHD). The primary endpoint we
will follow is production of the missing enzyme at ≥ 10% of the normal level at day 180
post-transplant as for investigational new drug (IND) 14070. This expanded access protocol
details the approach to providing a second dose of the product.
;
| 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 |
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 |