Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide
Stopped early · Phase 1/Phase 2
Conditions studied: Chronic Granulomatous Disease
In brief
Background: \- Chronic Granulomatous Disease (CGD) causes immune system problems. Treatment is usually a bone marrow transplant from a fully matched donor. Researchers want to try using partially matched donors for patients who do not have a fully matched donor available. The researchers will also use the drug cyclophosphamide to try to improve the outcomes when using a partially matched donor. Objective: \- To learn the effectiveness of using cyclophosphamide with a transplant from a partially matched donor in treating CGD. Eligibility: \- Recipients: age 2-65 with CGD with an ongoing infection that has not been cured by standard treatment and no fully matched donor available in an appropriate timeframe. Design: * Recipients will: * be admitted to the hospital 2 weeks before transplant. * be screened with blood and urine tests, breathing and heart health tests, X-rays, and/or magnetic resonance imaging. They may have a bone marrow aspiration and biopsy. * meet with a social worker and dentist. * get chemotherapy, radiation, and other medicines. * get an intravenous (IV) catheter in their chest. * have the transplant. * get more medicines and standard supportive care. * have blood drawn frequently. * have to stay in the Washington, D.C. area for 3 months post-transplant. * be followed closely for the first 6 months, and then less frequently for at least 5 years.
Key facts
- Study ID
- NCT02282904
- Run by
- National Institute of Allergy and Infectious Diseases (NIAID)
- People needed
- 7
- Starts
- 2014-10-23
- Expected to finish
- 2019-12-10
- Last updated by the study team
- 2020-05-12
Who can join
Age: 2 and older, up to 65. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- Major anticipated illness or organ failure incompatible with survival from Allo-transplant
- Inadequate collection from prospective donors.
Where it is running
- National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States
Full record on ClinicalTrials.gov
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