HSCT for High Risk Inherited Inborn Errors

NCT00383448 · clinicaltrials.gov ↗

PHASE2

Phase

COMPLETED

Status

38

Enrollment

OTHER

Sponsor class

Conditions

Adrenoleukodystrophy
Metachromatic Leukodystrophy
Globoid Cell Leukodystrophy
Tay Sachs Disease
Sandhoffs Disease
Wolman Disease
I-Cell Disease
Sanfilippo Syndrome
GM1 Gangliosidosis

Interventions

DRUG: Clofarabine
PROCEDURE: Total body Irradiation
DRUG: Melphalan
BIOLOGICAL: Hematopoietic Stem Cell Transplantation
DRUG: Alemtuzumab
DRUG: mycophenylate mofetil
DEVICE: Cyclosporine A
DRUG: Hydroxyurea

Sponsor

Masonic Cancer Center, University of Minnesota