Study Status: RECRUITING
Recruit Status: RECRUITING
Study Type: INTERVENTIONAL
Official Title: Pilot Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD)
Brief 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....