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NCT05463133 · ClinicalTrials.gov registry record · Phase 1
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
A Phase 1 study of Chronic Granulomatous Disease, sponsored by National Institute of Allergy and Infectious Diseases (NIAID).
- Recruiting
- Registry status
- Phase 1
- Development phase
- 50
- Enrollment target
- 1
- Study location
NCT05463133: Recruiting Phase 1 study of Chronic Granulomatous Disease, sponsored by National Institute of Allergy and Infectious Diseases (NIAID).
NCT05463133 is a Phase 1 study of Chronic Granulomatous Disease that is actively recruiting participants, run by National Institute of Allergy and Infectious Diseases (NIAID). The registered enrollment target is 50 participants, above the 28-participant average among 10 other Chronic Granulomatous Disease trials with a reported enrollment target (79% higher). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05463133, a Phase 1 study of Chronic Granulomatous Disease, is actively recruiting participants, sponsored by National Institute of Allergy and Infectious Diseases (NIAID).
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 50 participants
- Enrollment target
- 1
- Study location
Study Summary
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.
Primary Outcome
Alive at 1 years post-transplant.
Conditions Studied
Interventions
- DRUG Cyclophosphamide
- DRUG Sirolimus
- DRUG Busulfan
- DRUG Alemtuzumab
- BIOLOGICAL Pheripheral blood stem cells
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 50 participants |
| Start Date | 2022-07-08 |
| Est. Completion | 2032-12-31 |
| Phase | Phase 1 |
What NCT05463133 shows while recruiting
NCT05463133 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 50 participants, a relatively small participant target, above the 28-participant average among 10 other Chronic Granulomatous Disease trials with a reported enrollment target (79% higher).
The record links to 1 condition, with Chronic Granulomatous Disease appearing as the primary indexed condition, and to 5 interventions - of which Cyclophosphamide is the first listed.
NCT05463133 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT05463133 about?
NCT05463133 is a clinical study titled "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". 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 alw...
What is the current status of trial NCT05463133?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 50 participants. The study started on 2022-07-08. Estimated completion is 2032-12-31.
What conditions does trial NCT05463133 study?
This clinical trial studies the following conditions: Chronic Granulomatous Disease.
What interventions are being tested in trial NCT05463133?
The interventions under investigation include: Cyclophosphamide (DRUG), Sirolimus (DRUG), Busulfan (DRUG), Alemtuzumab (DRUG), Pheripheral blood stem cells (BIOLOGICAL).
Who is sponsoring clinical trial NCT05463133?
This trial is sponsored by National Institute of Allergy and Infectious Diseases (NIAID), which has 2,006 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05463133 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT05463133's enrollment target sits among peer trials
50 4th of 10 higher than 7 of 10 other Chronic Granulomatous Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Chronic Granulomatous Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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