HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (NCT02143830)
A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
🩺 Plain-English Summary (8th-Grade Level)
The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.
Who can join: Review the eligibility criteria below.
Location: Memorial Sloan Kettering Cancer Center — New York, New York
Age range: 3 Months
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (3 Sites)
- Memorial Sloan Kettering Cancer Center — New York, New York
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio
- Fred Hutchinson Cancer Research Center — Seattle, Washington
📞 Contact the Study Coordinator
Phone: (513) 803-1102
Email: Jamie.Wilhelm@cchmc.org
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Fanconi Anemia
- Eltrombopag for People With Fanconi Anemia — Bethesda, Maryland
- Prenatal Transplantation for Fetuses With Fanconi Anemia — San Francisco, California
- MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients — Minneapolis, Minnesota
- Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab — Stanford, California
- Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders — Minneapolis, Minnesota
Explore by condition: All Fanconi Anemia clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.