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

NCT05600907 | Active Not Recruiting Early Phase 1 FDA Drug

• 2 other identifiers: 10000609000609-I
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

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....

Conditions

Chronic Granulomatous Disease; CGD

Keywords

Transplant; Hsct; Monoclonal Antibody

Outcome Measures

Primary Outcomes (1)

• Early and long term engraftment

  To determine the engraftment rates, both early and long term, when using JSP191 in conjunction with TBI, Alemtuzumab and post transplant cyclophosphamide and sirolimus for unrelated donor transplantation of patients with Chronic Granulomatous Disease.

  Day 100 and 2 years post transplant

Secondary Outcomes (3)

• Incidence of viral reactivation

  Through study completion about 104 weeks

• Incidence of graft-vs-host disease (GvHD)

  Through study completion about 104 weeks

• Safety

  Through study completion about 104 Weeks

Study Arms (1)

Single arm study

EXPERIMENTAL

Eligible CGD patients will receive one time infusion of study product along with Alemtuzumab and TBI as part of HSCT conditioning regimen

Biological: JSP191

Interventions

JSP191 BIOLOGICAL

A monoclonal antibody that targets human CD117

Single arm study

Eligibility Criteria

• Age: 4 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• National Institute of Allergy and Infectious Diseases (NIAID): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Granulomatous Disease, Chronic

Condition Hierarchy (Ancestors)

Phagocyte Bactericidal Dysfunction; Leukocyte Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Elizabeth M Kang, M.D.

  National Institute of Allergy and Infectious Diseases (NIAID)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 26, 2022
• First Posted: November 1, 2022
• Study Start: January 2, 2023
• Primary Completion (Estimated): November 30, 2036
• Study Completion (Estimated): November 30, 2036
• Last Updated: August 26, 2025

Record last verified: 2025-08-21

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)