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NCT06325709 · ClinicalTrials.gov registry record · Phase 1
Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
A Phase 1 study of Chronic Granulomatous Disease (CGD) and X-Linked Chronic Granulomatous Disease, sponsored by National Institute of Allergy and Infectious Diseases (NIAID).
- Recruiting
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
- 1
- Study location
NCT06325709: Recruiting Phase 1 study of Chronic Granulomatous Disease (CGD) and X-Linked Chronic Granulomatous Disease, sponsored by National Institute of Allergy and Infectious Diseases (NIAID).
NCT06325709 is a Phase 1 study of Chronic Granulomatous Disease (CGD) and X-Linked Chronic Granulomatous Disease that is actively recruiting participants, run by National Institute of Allergy and Infectious Diseases (NIAID). The registered enrollment target is 10 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06325709, a Phase 1 study of Chronic Granulomatous Disease (CGD) and X-Linked Chronic Granulomatous Disease, is actively recruiting participants, sponsored by National Institute of Allergy and Infectious Diseases (NIAID).
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: Chronic granulomatous disease (CGD) is a rare immune disorder caused by a mutation in the CYBB gene. People with CGD have white blood cells that do not work properly and are at greater risk of getting infections. Gene therapy using lentivector has helped people with CGD. Researchers want to know if the base-edited stem cells can improve the white cells' functioning and result in fewer CGD-related infections. Objective: To learn if base-edited stem cells will correct the white blood cells in people with CGD. Eligibility: Males aged 18 years and older with X-linked CGD. Design: This is a non-randomized study. Participants with the specific mutation under study will be screened during the initial phase. During the development phase, participants will undergo apheresis to collect stem cells for base-editing correction of the mutation. During the treatment phase, participants will receive the base-edited cells after chemotherapy with busulfan. Participants will remain in the hospital until their immunity recovers. Participants will be maintained on sirolimus to prevent an immune response to the new protein expressed by the base-edited cells. Follow-up visits will continue for 15 years....
Primary Outcome
Safety of gene therapy using base-edited autologous hematopoietic stem and progenitor cells as measured by study agent related adverse events and serious adverse events
Conditions Studied
Interventions
- DRUG Busulfan
- DRUG Plerixafor
- DRUG Filgrastim
- DRUG Palifermin
- BIOLOGICAL Base-edited hematopoietic stem and progenitor cells
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2024-04-17 |
| Est. Completion | 2032-12-31 |
| Phase | Phase 1 |
What NCT06325709 shows while recruiting
NCT06325709 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower).
The record links to 2 conditions, with Chronic Granulomatous Disease (CGD) appearing as the primary indexed condition, and to 5 interventions - of which Busulfan is the first listed.
NCT06325709 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT06325709 about?
NCT06325709 is a clinical study titled "Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease". Background: Chronic granulomatous disease (CGD) is a rare immune disorder caused by a mutation in the CYBB gene. People with CGD have white blood cells that do not work properly and are at greater risk of getting infections. Gene therapy using lentivector has helped people with CGD. Researchers wan...
What is the current status of trial NCT06325709?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2024-04-17. Estimated completion is 2032-12-31.
What conditions does trial NCT06325709 study?
This clinical trial studies the following conditions: Chronic Granulomatous Disease (CGD), X-Linked Chronic Granulomatous Disease.
What interventions are being tested in trial NCT06325709?
The interventions under investigation include: Busulfan (DRUG), Plerixafor (DRUG), Filgrastim (DRUG), Palifermin (DRUG), Base-edited hematopoietic stem and progenitor cells (BIOLOGICAL).
Who is sponsoring clinical trial NCT06325709?
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 NCT06325709 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 NCT06325709's enrollment target sits among peer trials
10 1914th of 2000 higher than 46 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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.
Source ClinicalTrials.gov registry export · 2026-08-08
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