Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-24 @ 8:02 PM
Ignite Modification Date:
2025-12-24 @ 8:02 PM
NCT ID:
NCT02282904
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 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.
Detailed 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.
Study:
NCT02282904
Study Brief:
Protocol Section:
NCT02282904