# Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease

> **NCT00295971** · PHASE1 · COMPLETED · sponsor: **University of California, San Francisco** · enrollment: 21 (actual)

## Conditions studied

- Congenital Amegakaryocytic Thrombocytopenia
- Leukemia
- Myelodysplastic Syndromes
- Severe Congenital Neutropenia

## Interventions

- **BIOLOGICAL:** anti-thymocyte globulin
- **BIOLOGICAL:** therapeutic allogeneic lymphocytes
- **DRUG:** fludarabine phosphate
- **DRUG:** thiotepa
- **PROCEDURE:** allogeneic bone marrow transplantation
- **PROCEDURE:** allogeneic hematopoietic stem cell transplantation
- **PROCEDURE:** in vitro-treated peripheral blood stem cell transplantation
- **RADIATION:** total-body irradiation

## Key facts

- **NCT ID:** NCT00295971
- **Lead sponsor:** University of California, San Francisco
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2005-04
- **Primary completion:** 2011-12
- **Final completion:** 2011-12
- **Target enrollment:** 21 (ACTUAL)
- **Last updated:** 2012-11-12

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT00295971

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT00295971, "Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease". Retrieved via AI Analytics 2026-07-17 from https://api.ai-analytics.org/clinical/NCT00295971. Licensed CC0.

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