Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD)
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ClinicalTrials.gov Identifier: NCT05600907 |
Recruitment Status :
Recruiting
First Posted : November 1, 2022
Last Update Posted : May 13, 2024
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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....
Condition or disease | Intervention/treatment | Phase |
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Chronic Granulomatous Disease CGD | Biological: JSP191 | Early Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 30 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Pilot Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD) |
Actual Study Start Date : | January 2, 2023 |
Estimated Primary Completion Date : | November 30, 2036 |
Estimated Study Completion Date : | November 30, 2036 |
Arm | Intervention/treatment |
---|---|
Experimental: Single arm study
Eligible CGD patients will receive one time infusion of study product along with Alemtuzumab and TBI as part of HSCT conditioning regimen
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Biological: JSP191
A monoclonal antibody that targets human CD117 |
- Early and long term engraftment [ Time Frame: Day 100 and 2 years post transplant ]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.
- Incidence of viral reactivation [ Time Frame: Through study completion about 104 weeks ]To compare the incidence of peri and post transplant complications including viral reactivation with this new regimen compared to historical data.
- Incidence of graft-vs-host disease (GvHD) [ Time Frame: Through study completion about 104 weeks ]To determine and compare the rates and severity of GvHD.
- Safety [ Time Frame: Through study completion about 104 Weeks ]To determine and assess the toxicity of JSP191 in combination with Campath, TBI and post transplant cyclophosphamide.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 4 Years to 65 Years (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
- 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.
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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.
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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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05600907
Contact: Sandra Anaya-O'Brien, R.N. | (301) 346-9781 | sobrien@niaid.nih.gov | |
Contact: Elizabeth M Kang, M.D. | (301) 402-7567 | ekang@niaid.nih.gov |
United States, Maryland | |
National Institutes of Health Clinical Center | Recruiting |
Bethesda, Maryland, United States, 20892 | |
Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 ext TTY dial 711 ccopr@nih.gov |
Principal Investigator: | Elizabeth M Kang, M.D. | National Institute of Allergy and Infectious Diseases (NIAID) |
Responsible Party: | National Institute of Allergy and Infectious Diseases (NIAID) |
ClinicalTrials.gov Identifier: | NCT05600907 |
Other Study ID Numbers: |
10000609 000609-I |
First Posted: | November 1, 2022 Key Record Dates |
Last Update Posted: | May 13, 2024 |
Last Verified: | May 8, 2024 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Plan Description: | .Presently there is no plan to share individual data on this study. |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Transplant Hsct Monoclonal Antibody |
Pathologic Processes Granuloma Granulomatous Disease, Chronic Lymphoproliferative Disorders Lymphatic Diseases Phagocyte Bactericidal Dysfunction Leukocyte Disorders |
Hematologic Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Immunologic Deficiency Syndromes Immune System Diseases Chronic Disease Disease Attributes |