Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 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.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05463133
The ClinicalTrials.gov registry entry for NCT05463133 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 50 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 28-participant average among 10 other Chronic Granulomatous Disease trials with a reported enrollment target (79% higher). The listed sponsor is National Institute of Allergy and Infectious Diseases (NIAID), which has 2,006 total studies on file at ClinicalTrials.gov.
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 1 study location spanning 1 distinct geographic area - top geographies include 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
Similar trials for Chronic Granulomatous Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
RECRUITING · Phase 1
-
A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )
ACTIVE NOT RECRUITING · Phase 1
-
Stem Cell Transplant Following Low-Intensity Chemotherapy to Treat Chronic Granulomatous Disease
COMPLETED · Phase 1
-
Effect of IFN-γ on Innate Immune Cells
COMPLETED · Phase 1
-
Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD)
ACTIVE NOT RECRUITING · Early Phase 1
-
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
ACTIVE NOT RECRUITING · NA
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.