Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide

Chronic Granulomatous Disease Clinical Trial

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

Status Terminated

Clinical Trial 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.

Clinical Trial Description

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. ;

Related Conditions & MeSH terms

• Chronic Granulomatous Disease
• Granuloma
• Granulomatous Disease, Chronic

• NCT number: NCT02282904
• Study type: Interventional
• Source: National Institutes of Health Clinical Center (CC)
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: October 23, 2014
• Completion date: December 10, 2019