Stem Cell Transplant + Drug Therapy for Chronic Granulomatous Disease
Trial Summary
Will I have to stop taking my current medications?
The trial protocol does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor to get specific guidance based on your situation.
What data supports the effectiveness of the treatment for Chronic Granulomatous Disease?
Research shows that allogeneic hematopoietic stem cell transplantation, which includes drugs like alemtuzumab and busulfan, is a curative treatment for Chronic Granulomatous Disease. Studies have reported successful outcomes with high survival rates and low incidence of infections in patients who underwent this treatment.12345
What safety data exists for the drugs used in stem cell transplant and drug therapy for chronic granulomatous disease?
The safety of cyclophosphamide, one of the drugs used in this treatment, has been studied in other conditions. It is known to cause side effects like bone marrow suppression, which can lead to a low white blood cell count, increasing the risk of infections. Careful monitoring and dosage adjustments are important to manage these risks.678910
How is the Stem Cell Transplant + Drug Therapy for Chronic Granulomatous Disease unique?
This treatment is unique because it combines stem cell transplantation with a specific set of drugs, including Alemtuzumab, Busulfan, Cyclophosphamide, and Emapalumab-Izsg, along with Total Body Irradiation, to potentially improve outcomes for patients with Chronic Granulomatous Disease. This combination aims to enhance the effectiveness of the transplant and reduce the risk of complications compared to traditional conditioning regimens.1251112
What is the purpose of this trial?
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.
Research Team
Elizabeth M Kang, M.D.
Principal Investigator
National Institute of Allergy and Infectious Diseases (NIAID)
Eligibility Criteria
This trial is for people aged 4-65 with Chronic Granulomatous Disease (CGD) who have complications from the disease or a specific level of oxidase production. They must not be HIV positive, pregnant, or breastfeeding and should agree to use contraception. Participants need an HLA-matched stem cell donor and must stay close to the NIH post-transplant.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Conditioning and Transplantation
Participants receive conditioning regimen including Alemtuzumab, Busulfan, and TBI, followed by donor stem cell transplantation
Post-Transplant Recovery
Participants remain in the hospital for recovery and monitoring after receiving donor stem cells
Initial Follow-up
Participants visit the clinic for monitoring and assessment of engraftment and infection rates
Long-term Follow-up
Participants are monitored for overall survival, engraftment stability, and absence of GvHD
Treatment Details
Interventions
- Alemtuzumab
- Busulfan
- Cyclophosphamide
- Emapalumab-Izsg
- Tociluzumab
- Total Body Irradiation
Alemtuzumab is already approved in United States, European Union for the following indications:
- Chronic lymphocytic leukemia
- Multiple sclerosis
- Multiple sclerosis
- Chronic lymphocytic leukemia
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Institute of Allergy and Infectious Diseases (NIAID)
Lead Sponsor