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

    Detailed Summary: Allogeneic transplant using HLA matched donors, both related and unrelated, has proven curative for patients with various immunodeficiencies, including those with ongoing infections. However donor availability remains a limiting factor in the application of this treatment modality. The use of haploidentical donors has in the past been fraught with a greater rate of complications related to both higher rates of GvHD and delayed immunorecovery. Newer transplant regimens appear to have diminished these risks and improved outcomes. We propose using a subablative conditioning regimen followed by post-transplant cyclophosphamide for patients with CGD who do not have an HLA matched donor but whose circumstances necessitate the use of a potentially curative, albeit high-risk treatment modality.
    Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

    Current Primary Outcome: To use a subablative conditioning regimen followed by post-transplant cyclophosphamide to attain an engraftment rate of 100% with no occurrence of acute Grade 3 or higher GvHD (graft versus host disease). [ Time Frame: Day 30; First 100 days ]

    -Engraftment will be determined as 30-50% myeloid chimerism by Day 30.-Acute graft versus host disease will be determined clinically and within the first 100 days.


    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • Stable Chimerism as indicated by 30-50% myeloid engraftment and 50% lymphoid engraftment as assessed by 1 year post transplant [ Time Frame: 1 year post transplant ]
    • Immune reconstitution levels with DHR as a marker of normal neutrophil function by 1 year post transplant. [ Time Frame: 1 year post transplant ]


    Original Secondary Outcome: Same as current

    Information By: National Institutes of Health Clinical Center (CC)

    Dates:
    Date Received: November 4, 2014
    Date Started: October 22, 2014
    Date Completion: January 1, 2030
    Last Updated: May 12, 2017
    Last Verified: April 7, 2017