# HSCT for High Risk Inherited Inborn Errors

> **NCT00383448** · PHASE2 · COMPLETED · sponsor: **Masonic Cancer Center, University of Minnesota** · enrollment: 38 (actual)

## Conditions studied

- 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

## Key facts

- **NCT ID:** NCT00383448
- **Lead sponsor:** Masonic Cancer Center, University of Minnesota
- **Sponsor class:** OTHER
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2006-09
- **Primary completion:** 2014-09
- **Final completion:** 2014-09
- **Target enrollment:** 38 (ACTUAL)
- **Last updated:** 2019-07-11


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT00383448

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT00383448, "HSCT for High Risk Inherited Inborn Errors". Retrieved via AI Analytics 2026-06-09 from https://api.ai-analytics.org/clinical/NCT00383448. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*