Allogeneic Reduced-Intensity Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease: a Single-Center Prospective Trial.
Mark Parta, Corin Kelly, Nana Kwatemaa...
https://pubmed.ncbi.nlm.nih.gov/28752258Actively Recruiting
Phase 1
Phase 2
Age: 4Years - 65Years
All Genders
ID05463133
Phase I/II Study of Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease Using Alemtuzumab, Busulfan, Total Body Irradiation, and 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
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying Chronic Granulomatous Disease (CGD), an inherited immune disorder that increases the risk of infections and inflammatory complications. Current treatments can cause serious side effects, and while stem cell transplants can cure CGD, they don't always succeed. This trial aims to evaluate whether a new drug regimen can improve the success rates of stem cell transplants in people with CGD, including those with high inflammation levels. Participants will be divided into two groups based on their inflammation marker levels. Both groups receive conditioning drugs including alemtuzumab, busulfan, and total body irradiation, followed by donor peripheral blood stem cell infusions through a catheter placed in a vein. The high-risk group with elevated inflammation also receives additional drugs targeting specific inflammatory cytokines. Post-transplant medications such as sirolimus and cyclophosphamide will be given to prevent complications. During the study, participants undergo thorough screening including physical exams, blood and urine tests, heart and lung function tests, imaging scans, and bone marrow biopsies. They will stay in the hospital for pre-transplant treatments and for six weeks after receiving the stem cells, followed by frequent clinic visits for three months and continued follow-up for five years. The main outcome measured is overall survival one year after transplant, with other assessments including infection rates, engraftment success, and transplant-related complications.
CONDITIONS
Brief 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
Age: 4Years - 65Years
All Genders
Eligibility Criteria
You may qualify if you...
- Ability to understand and willingness to sign informed consent (parent/guardian consent and minor assent as appropriate)
- Confirmed diagnosis of Chronic Granulomatous Disease (CGD)
- Significant complications warranting transplantation or specific residual oxidase production levels
- Ages 4 to 65 years
- Availability of HLA-matched family donor or matched unrelated peripheral blood stem cell graft
- HIV negative status
- Ability to stay within 1 hour of the NIH for 3 months post-transplant
- Presence of a family member or caregiver to assist during outpatient care
- Provision of durable power of attorney for health care decisions
- Females of child-bearing potential and male participants agree to use contraception as specified
- Willingness to comply with all study procedures and visits
- High-risk participants with CRP over 100 mg/mL eligible for high-risk arm
You will not qualify if you...
- Ejection fraction below 30% by echocardiography
- Forced expiratory volume (FEV1) less than 35% or adjusted lung diffusing capacity (DLCO) below 30%
- Liver enzyme levels (transaminases) more than 5 times the upper limit of normal
- Severe psychiatric disorder or mental deficiency preventing compliance or informed consent
- Major illness or organ failure incompatible with stem cell transplant survival
- Pregnancy or lactation
- Uncontrolled seizure disorder
- Age over 65 years
- Active tuberculosis infection
- Conditions making protocol compliance difficult
- Unwillingness to provide information for alemtuzumab distribution program or not qualified for the drug's use
AI-Screening
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Run-in Period
Duration - Up to 24 days depending on risk group
Participants receive pretreatment with cytokine antagonists based on their inflammation levels before transplant conditioning begins.
1 to 2 visits depending on CRP levels
Treatment
Duration - Approximately 2 weeks
Participants undergo conditioning with Alemtuzumab, Busulfan, Total Body Irradiation, and receive donor peripheral blood stem cell infusion followed by post-transplant cyclophosphamide and sirolimus to prevent graft rejection and graft versus host disease.
Several visits including daily treatments during conditioning and post-transplant visits
Follow-up
Duration - Up to 1 year
Participants are monitored for engraftment, graft versus host disease, infection rates, and overall survival for up to 1 year post-transplant.
Multiple follow-up visits including assessments at Day 14, 30, 60, 100, 6 months, and 1 year post-transplant
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
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Research Team
S
Sandra M Maxwell, R.N.
E
Elizabeth M Kang, M.D.
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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Stephan Kadauke, Regina M Myers, Yimei Li...
https://pubmed.ncbi.nlm.nih.gov/33417474Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults.
Robert Chiesa, Junfeng Wang, Henric-Jan Blok...
https://pubmed.ncbi.nlm.nih.gov/32614953