ACTIVE_NOT_RECRUITING

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

Conditions

Chronic Granulomatous Disease CGD Transplant Hsct Monoclonal Antibody

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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....

Official Title

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

Quick Facts

Study Start:2023-01-02

Study Completion:2036-11-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05600907

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:4 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	* 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.

Inclusion Criteria

Exclusion 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.

* 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.

Contacts and Locations

Principal Investigator

Elizabeth M Kang, M.D.

PRINCIPAL_INVESTIGATOR

National Institute of Allergy and Infectious Diseases (NIAID)

Study Locations (Sites)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892

United States

Collaborators and Investigators

Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Elizabeth M Kang, M.D., PRINCIPAL_INVESTIGATOR, National Institute of Allergy and Infectious Diseases (NIAID)

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-01-02

Study Completion Date2036-11-30

Study Record Updates

Study Start Date2023-01-02

Study Completion Date2036-11-30

Terms related to this study

Keywords Provided by Researchers

Transplant
Hsct
Monoclonal Antibody

Additional Relevant MeSH Terms

Chronic Granulomatous Disease
CGD