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Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD)
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Summary
Background: Chronic granulomatous disease (CGD) is a rare immune disorder that can cause serious infections throughout the body. The only cure for CGD is a stem cell transplant. Transplants from a sibling are best, but many people must get transplants from unrelated donors. However, these transplants can cause serious complications in people with CGD. Objective: To see if a study drug (JSP191) can help improve the success rates of stem cell transplants for people with CGD from an unrelated donor. Eligibility: People aged 4 to 65 years with CGD who require a transplant. Design: Participants will be screened. Part of the screening will help to identify the best match to a transplant donor. Participants will have a physical exam, including dental and eye exams. They will have blood and urine tests. They will have tests of their breathing and heart function. A bone marrow sample will be taken. They will have their stem cells collected. Participants will have a catheter inserted into a vein in their chest. It will remain in place for the entire period of transplant and recovery. Participants will be in the hospital 40 to 50 days for the transplant. This will include a conditioning phase, to prepare their body for the procedure, as well as the transplant and recovery phases. As part of the conditioning phase, participants will receive JSP191 through a vein for 1 hour. After discharge, participants will have follow-up visits 2 times a week for 100 days. Additional follow-up visits will continue for 5 years....
Official title: Pilot Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD)
Key Details
Gender
All
Age Range
4 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
7
Start Date
2023-01-02
Completion Date
2036-11-30
Last Updated
2025-08-26
Healthy Volunteers
No
Conditions
Interventions
JSP191
A monoclonal antibody that targets human CD117
Locations (1)
National Institutes of Health Clinical Center
Bethesda, Maryland, United States