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

Launched by NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES (NIAID) · Oct 31, 2022

Keywords

ClinConnect Summary

This clinical trial is exploring a new study drug called JSP191 to see if it can help improve the success of stem cell transplants for people with Chronic Granulomatous Disease (CGD). CGD is a rare immune disorder that makes it hard for the body to fight infections, and a stem cell transplant is often the only cure. The trial is looking for participants aged 4 to 65 years who have CGD and need a transplant from an unrelated donor. To participate, individuals must have a matched unrelated donor available and be able to travel to the National Institutes of Health (NIH) for follow-up care after the transplant.

If someone joins the trial, they will undergo several tests to check their health and ensure they are a good candidate for the transplant. Participants will spend about 40 to 50 days in the hospital for the transplant procedure and recovery. During this time, they will receive JSP191 to help prepare their body for the transplant. After leaving the hospital, they will need to attend follow-up appointments twice a week for the first 100 days and then continue with check-ups for up to five years. It’s important for participants to have a family member or companion to support them during this time.

Gender

ALL

Eligibility criteria

• * 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 Group (ECOG) or equivalent performance status \>= 3 (see Supportive Care guidelines, available at https://training.seer.cancer.gov/followup/procedures/dataset/ecog.html).
• • Left ventricular ejection fraction \< 40%.
• • Transaminases \> 5x upper limit of normal based on the individual s clinical situation and at the discretion of the investigator.
• • CRP \> 100 mg/dL within 6 weeks of the transplant.
• • Psychiatric disorder or mental deficiency severe enough as to make compliance with the HSCT unlikely, and/or to make regulatorily and legally effective informed consent impossible.
• • Major anticipated illness or organ failure incompatible with survival from allogeneic HSCT.
• • Pregnant or breastfeeding.
• • HIV positive.
• • Uncontrolled seizure disorder.
• • Any condition or circumstance that the PI feels would create difficulty in maintaining compliance with the requirements of this protocol.
• • Individuals who are not willing to submit their information as part of the alemtuzumab (Campath) Distribution Program application or participants whom the Distribution Program committee has determined are not qualified to receive alemtuzumab.
• • NOTE: Alemtuzumab (IV formulation) is no longer distributed commercially. In order to receive product, the physician must contact the program for the patient. If the patient is not willing to consent to submit their information (demographics, contact information, and rationale for use) to the program such that we can obtain the drug, then we cannot proceed with conditioning; therefore, the individual will not be eligible for this protocol.