Using JSP191 to improve stem cell transplants for Chronic Granulomatous Disease

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

This study is testing if a new treatment called JSP191 can help improve the success of stem cell transplants for people with Chronic Granulomatous Disease.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 38 (estimated)
• Ages: 4 Years to 65 Years
• Sex: All
• Sponsor: National Institutes of Health Clinical Center (CC) (NIH)
• Drugs / interventions: alemtuzumab, cyclophosphamide, Cytoxan
• Locations: 1 site (Bethesda, Maryland)
• Trial ID: NCT05600907 on ClinicalTrials.gov

What this trial studies

This pilot study aims to evaluate the effectiveness of JSP191 in enhancing the success rates of stem cell transplants from matched unrelated donors in patients with Chronic Granulomatous Disease (CGD). Participants aged 4 to 65 will undergo a series of screenings and tests to identify suitable transplant donors and assess their health. The study will utilize a conditioning regimen involving alemtuzumab and total body irradiation, followed by the infusion of JSP191 and a high-dose donor graft. The primary focus is to measure engraftment rates and assess overall survival, safety, and efficacy of the treatment.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 4 to 65 with confirmed CGD who require a transplant from an unrelated matched donor.

Not a fit: Patients who have a matched related donor available or those outside the specified age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could significantly improve transplant outcomes and survival rates for patients with CGD.

How similar studies have performed: While this approach is novel, previous studies have shown promise in improving transplant outcomes using similar conditioning regimens.

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.

Where this trial is running

Bethesda, Maryland

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (Recruiting)

Study contacts

• Principal investigator: Elizabeth M Kang, M.D. — National Institute of Allergy and Infectious Diseases (NIAID)
• Study coordinator: Sandra Anaya-O'Brien, R.N.

Conditions Chronic Granulomatous Disease; CGD; Transplant; Hsct; Monoclonal Antibody