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Stem Cell Transplant for Chronic Granulomatous Disease (CGD)

This study is testing a new way to perform stem cell transplants for people with Chronic Granulomatous Disease (CGD), an immune system disorder that makes you more prone to infections. The goal is to improve the success rate of these transplants. You would receive a combination of drugs including Alemtuzumab, Busulfan, Sirolimus, and Cyclophosphamide, along with donor peripheral blood stem cells. Researchers will be looking at how many people are still alive one year after their transplant. This study is open to individuals with CGD between 4 and 65 years old. The current recruitment status is unclear, and the study plans to enroll 50 participants.

Study design
This is an interventional study with a planned enrollment of 50 participants. The phase of the study is not specified.
What's involved
You will undergo screening, which includes a physical exam, blood and urine tests, heart and breathing function tests, and imaging scans.
Compensation
Not stated in the trial record.
Follow-up
Overall survival will be measured at 1 year post-transplant.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05463133

Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists

Recruiting
PHASE1Ages 4–65InterventionalTreatment
National Institute of Allergy and Infectious Diseases (NIAID)
~50 participants
Updated 2026-05-14 on ClinicalTrials.gov
What's tested:SirolimusCyclophosphamideAlemtuzumabBusulfanPheripheral blood stem cellsEmapalumab-Izsg

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Overall Survival
Measured over 1 years post-transplant.

Conditions

Chronic Granulomatous Disease

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Chronic Granulomatous Disease

NCT05463133

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • National Institutes of Health Clinical Center

    Bethesda, Marylandstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

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

Who to contact

Sandra M Maxwell, R.N.
[email protected]
(240) 627-3078
Email the study team

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Eligibility criteria

Inclusion

Must have the ability to comprehend and a willingness to sign the informed consent. For pediatric patients, must have a parent/guardian who can sign consent if the donor is a minor; assent will be obtained from minors as appropriate.
Must have confirmed diagnosis of CGD.
Must have 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 have a Quartile 1 or 2 residual oxidase production level.
Ages 4 years-65 years.
HLA-matched family donor graft or an HLA-matched unrelated PBSC graft (10/10 or 9/10 mismatch) available.
Must be human immunodeficiency virus (HIV) negative.
When discharged from the hospital the participant must be able to stay within 1 hour s travel of the NIH for the first 3 months after transplantation.
Must have a family member or other designated care provider to assist with care during the post-transplant period when the patient is in the outpatient setting.
Must provide a durable power of attorney for health care decisions to an appropriate adult relative or guardian in accordance with NIH 200 'NIH Durable Power of Attorney for Health Care Decision Making.'
Females of child-bearing potential must agree to consistently use one form of contraception from 1 month prior to study entry and for at least 1 year post transplant. Male participants must agree to consistently use contraception for 1 year post transplant. Acceptable forms of contraception are:
Contraceptive pills or patch, Norplant \[Registered\], Depo-Provera \[Registered\], or other FDA-approved contraceptive method.
Male partner has previously undergone a vasectomy.
Male participants will be advised to consistently use contraception throughout study participation and for 3 months post-transplant.
Stated willingness to comply with all study procedures and is available for protocol visits for the duration of the study when possible.
CRP will be assessed no more than 7 weeks and no less than 6 weeks prior to anticipated transplant to determine on which arm the patient will be treated.

Exclusion

Ejection fraction of less than 30% by echocardiography.
Forced expiratory volume (FEV1%) of less than 35% and/or an adjusted diffusing capacity of lung of carbon monoxide (adj DLCO) of less than 30%.
Transaminases \>5x upper limit of normal based on the individual s clinical situation and at the discretion of the investigator.
Psychiatric disorder or mental deficiency severe enough as to make compliance with the HSCT treatment unlikely, and/or to make regulatorily and legally effective informed consent impossible.
Major anticipated illness or organ failure incompatible with survival from allogeneic peripheral blood stem cell (AlloPBSC) transplant.
Pregnant or lactating.
Uncontrolled seizure disorder per principal investigator (PI) discretion.
Individuals older than 65 years are excluded. It is known from standard transplantation that these individuals have a higher risk of morbidity and mortality related to transplantation. Given the investigational nature of this protocol, the risk-benefit ratio is not warranted to include these individuals at this time.
Active TB infection.
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 \[Registered\]) Distribution Program application or participants whom the Distribution Program committee has determined are not qualified to receive alemtuzumab.

What this trial measures

  • Overall Survival1 years post-transplant.

    Alive at 1 years post-transplant.