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Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation
NCT00176904Phase 3Completed
- Sponsor
- Masonic Cancer Center
- Start
- Jan 1995
- Primary completion
- Jun 2010
- Modality
- Stem Cell Therapies
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of hematopoietic stem cells in adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD), globoid cell leukodystrophy (GLD) and 8 more, sponsored by Masonic Cancer Center. Primary endpoint: Overall Survival at 3 years. Started Jan 1995; status: completed.
02
Drugs
hematopoietic stem cells
03
Conditions
adrenoleukodystrophy (ALD)metachromatic leukodystrophy (MLD)globoid cell leukodystrophy (GLD)Gaucher disease (GD)Fucosidosis
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