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Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study
NCT06959771 · View on ClinicalTrials.gov ↗
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. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. 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 1,295 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06959771 reports 1 study location spanning 1 distinct geographic area — top geographies include Maryland. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
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). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06959771?
This trial is sponsored by National Institute of Allergy and Infectious Diseases (NIAID), which has 1,295 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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