Actively Recruiting
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
Led by National Institute of Allergy and Infectious Diseases (NIAID) · Updated on 2026-05-14
50
Participants Needed
1
Research Sites
547 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Chronic granulomatous disease (CGD) affects the immune system. People with CGD are more likely to get infections. Drugs can help control infections, but these treatments can cause side effects including kidney failure and deafness. Stem cell transplants can cure CGD, but these don t always work. Objective: To find out if a different drug treatment can improve the success rates of stem cell transplants in people with CGD. Eligibility: People aged 4-65 years with CGD. Design: Participants will undergo screening. They will have a physical exam. They will have blood and urine tests and tests of their heart function and breathing. They will have imaging scans. They will have a bone marrow biopsy; a needle will be inserted into their hip to draw a sample of tissue from the bone. A tube called a catheter will be placed into a vein in the participant s chest. This catheter will remain in place for the transplant and recovery period. Blood for tests can be drawn from the catheter, and medications and the stem cells can be administered through it. Participants will be in the hospital for either 10 or 21 days to receive 3 or 4 drugs before the transplant. They will get 2 doses of total body radiation on the same day. Participants will receive donor stem cells through the catheter. They will remain in the hospital for 6 weeks afterward. Participants will visit the clinic 2 to 3 times per week for 3 months after discharge. Follow-up visits will continue for 5 years.
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Ability to understand and sign informed consent; parent/guardian consent and minor assent if applicable
- Confirmed diagnosis of Chronic Granulomatous Disease (CGD)
- Presence of complications from CGD warranting transplant or specific oxidase production levels
- Age between 4 and 65 years
- Availability of HLA-matched family or unrelated donor graft
- HIV negative
- Ability to stay within 1 hour travel of NIH for 3 months post-transplant
- Has a family member or designated caregiver for post-transplant care
- Provides durable power of attorney for health care decisions
- Females of child-bearing potential agree to use contraception 1 month before and 1 year after transplant; males agree to contraception for 1 year post transplant
- Willingness to comply with study procedures and visits
- Participants with CRP greater than 100 mg/mL can enroll in high-risk arm
You will not qualify if you...
- Ejection fraction less than 30%
- Forced expiratory volume (FEV1%) less than 35% or adjusted DLCO less than 30%
- Liver enzymes (transaminases) over 5 times upper normal limit
- Severe psychiatric disorder or mental deficiency preventing compliance or consent
- Major illness or organ failure preventing survival from transplant
- Pregnant or lactating
- Uncontrolled seizure disorder
- Older than 65 years
- Active tuberculosis infection
- Any condition causing difficulty in protocol compliance
- Unwillingness to submit to alemtuzumab distribution program or disqualification from it
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
S
Sandra M Maxwell, R.N.
CONTACT
E
Elizabeth M Kang, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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