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NCT06959771 · ClinicalTrials.gov registry record · Phase 1
Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study
A Phase 1 study of CD40L-HyperIgM Syndrome, sponsored by National Institute of Allergy and Infectious Diseases (NIAID).
- Recruiting
- Registry status
- Phase 1
- Development phase
- 1
- Enrollment target
- 1
- Study location
NCT06959771 is a Phase 1 study of CD40L-HyperIgM Syndrome that is actively recruiting participants, run by National Institute of Allergy and Infectious Diseases (NIAID). The registered enrollment target is 1 participants. The trial reports 1 study location across 1 state.
The verdict
NCT06959771, a Phase 1 study of CD40L-HyperIgM Syndrome, is actively recruiting participants, sponsored by National Institute of Allergy and Infectious Diseases (NIAID).
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 1 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: X-linked hyper-IgM (HIGM) syndrome is caused by a mutation in the CD40 ligand (CD40L) gene. People with this disease have white blood cells that do not work properly. These people are at risk of severe infections and autoimmune diseases. Researchers want to know if these base-edited stem cells and T cells can help people with CD40L-HIGM syndrome. Objective: To test base-edited stem cells and base-edited T cells in 1 person with CD40L-HIGM syndrome. Eligibility: A single male with CD40L-HIGM syndrome. Design: A single participant is planned to receive a single dose of edited stem cells and supportive treatment with edited T cells. Participant stem and T cells will undergo base editing to repair the mutation. In preparation for the gene therapy, the participant will receive busulfan chemotherapy and alemtuzumab. After treatment, the participant will have follow-up visits every few months in the first 2 years after treatment. Long-term visits will continue annually for 15 years.
Conditions Studied
Interventions
- DRUG Sirolimus
- DRUG Busulfan
- DRUG Alemtuzumab
- 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 | 1 participants |
| Start Date | 2025-07-16 |
| Est. Completion | 2027-10-28 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06959771
The ClinicalTrials.gov registry entry for NCT06959771 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 1 participants, a figure that helps gauge the scale of data the investigators plan to collect. 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 CD40L-HyperIgM Syndrome appearing as the primary indexed condition, and to 5 interventions - of which Sirolimus is the first listed.
NCT06959771 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT06959771 about?
NCT06959771 is a clinical study titled "Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study". Background: X-linked hyper-IgM (HIGM) syndrome is caused by a mutation in the CD40 ligand (CD40L) gene. People with this disease have white blood cells that do not work properly. These people are at risk of severe infections and autoimmune diseases. Researchers want to know if these base-edited ste...
What is the current status of trial NCT06959771?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 1 participants. The study started on 2025-07-16. Estimated completion is 2027-10-28.
What conditions does trial NCT06959771 study?
This clinical trial studies the following conditions: CD40L-HyperIgM Syndrome.
What interventions are being tested in trial NCT06959771?
The interventions under investigation include: Sirolimus (DRUG), Busulfan (DRUG), Alemtuzumab (DRUG), Palifermin (DRUG), Base-edited hematopoietic stem and progenitor cells (BIOLOGICAL).
Who is sponsoring clinical trial NCT06959771?
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 NCT06959771 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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