Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide
Status:
Terminated
Trial end date:
2019-12-10
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
National Institute of Allergy and Infectious Diseases (NIAID)