Clinical Trial: Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide

Study Status: Suspended
Recruit Status: Suspended
Study Type: Interventional



Official Title: Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide

Brief 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 f