drugset / Trial / NCT02171104
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
RandomizedSingle-groupOpen-labelTreatment
Summary
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).
Timeline
- Start
- 2014-07-10
- Primary completion
- 2026-01-05
- Completion
- 2029-07-14
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE) | Protein / enzyme biologic | — | — |
| Subject | Alemtuzumab | Monoclonal antibody | — | — |
| Subject | Busulfan | Small molecule | — | — |
| Subject | Celecoxib | Other / unclassified | — | — |
| Subject | Fludarabine | Small molecule | — | — |
| Subject | N-acetylcysteine | Small molecule | — | — |
| Subject | Rituximab | Monoclonal antibody | — | — |
| Subject | Thiotepa | Other / unclassified | — | — |
| Subject | Vitamin E | Other / unclassified | — | — |
| Subject | alpha-lipoic acid | Small molecule | — | — |
Indications
Hurler syndrome
Krabbe disease
Niemann-Pick disease type B
Niemann-Pick disease, type C2
X-linked cerebral adrenoleukodystrophy
Zellweger spectrum disorders
alpha-mannosidosis
alpha-methylacyl-CoA racemase deficiency
aspartylglucosaminuria
d-bifunctional protein deficiency
fucosidosis
glycoprotein metabolism disease
leukoencephalopathy, diffuse hereditary, with spheroids 1
metachromatic leukodystrophy
mitochondrial neurogastrointestinal encephalomyopathy
mitochondrial trifunctional protein deficiency
mucopolysaccharidosis type 6
osteopetrosis
peroxisomal acyl-CoA oxidase deficiency