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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: Recruiting Phase 1 study of CD40L-HyperIgM Syndrome, sponsored by National Institute of Allergy and Infectious Diseases (NIAID).
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, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (98% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
To determine the safety and efficacy of BE HSPC CD40L and BE T Cells
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 |
What NCT06959771 shows while recruiting
NCT06959771 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 1 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (98% lower).
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 a single indexed study location in 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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT06959771's enrollment target sits among peer trials
1 2001st of 2000 the lowest 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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