Can a new antibody make stem cell transplants safer for CGD patients?

NCT ID NCT05600907

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

7 people

The number who actually took part.

Started

Jan 2023

Expected to finish

Nov 2036

An estimate. End dates often move.

Lead sponsor

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

4 to 65 years

Sex

Anyone

Healthy volunteers

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

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

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892, United States

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