Active — Not RecruitingEarly Phase 1

Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD)

United States7 participantsStarted 2023-01-02

Plain-language 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....

Who can participate

Age range4 Years – 65 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

* INCLUSION CRITERIA: * Has confirmed CGD. * Has sufficient complications from underlying disease to warrant undergoing transplantation (either a history of or ongoing inflammation/CGD-related autoimmunity OR a CGD-related infection while on prophylaxis) OR has a Quartile 1 or 2 residual oxidase production level. * Aged 4 years to 65 years. * Has an unrelated matched donor available (but no matched related donor available). * Must be able to stay within 1 hours travel of the NIH for the first 3 months after transplantation and have a family member or other designated companion to stay with during the post-transplant period. * Must provide a durable power of attorney for health care decisions to an appropriate adult relative or guardian in accordance with form NIH 200 NIH Durable Power of Attorney for Health Care Decision Making. * If of childbearing potential, must agree to consistently use contraception from 1 month prior to baseline, throughout study participation, and for 1 year after receiving transplanted cells. Acceptable forms of contraception are: * Contraceptive pills or patch, Norplant, Depo-Provera, or other FDA-approved contraceptive method. * Male partner has previously undergone a vasectomy. * If able to impregnate a partner, must agree to consistently use contraception from the time of enrollment through 3 months post-transplant. Acceptable forms of contraception are: * Male condom with spermicide. EXCLUSION CRITERIA: * Eastern Cooperative Oncology Gr…

What they're measuring

Early and long term engraftment

Timeframe: Day 100 and 2 years post transplant

Trial details

NCT IDNCT05600907

SponsorNational Institute of Allergy and Infectious Diseases (NIAID)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2036-11-30