Can a new antibody make stem cell transplants safer for CGD patients?
NCT ID NCT05600907
First seen Aug 27, 2026 · Last updated Aug 28, 2026 · Updated 1 time
Summary
Chronic granulomatous disease (CGD) is a rare immune disorder that causes severe infections, and the only cure is a stem cell transplant. Transplants from unrelated donors can be risky, so researchers are testing whether adding the drug JSP191 before the transplant helps the donor cells settle in better. This early-phase trial will enroll 7 people aged 4 to 65 with CGD who have an unrelated donor but no matched sibling. Participants receive JSP191 as part of the conditioning regimen, and researchers will track engraftment, complications, and safety for up to 5 years.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- JSP191, a monoclonal antibody that targets CD117 on stem cells, given before transplant to help donor cells engraft
- What this could lead to
- If it works, this could make unrelated-donor stem cell transplants safer and more successful for people with CGD, potentially offering a cure to more patients.
- What could go wrong
- This is a very early, small pilot study with only 7 participants, so results may not apply broadly. The transplant itself carries risks like graft-versus-host disease and infections.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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7 people
The number who actually took part.
- Started
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Jan 2023
- Expected to finish
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Nov 2036
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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4 to 65 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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