Track this trial free. DataLookout checks ClinicalTrials.gov daily and emails you which fields changed on NCT07775313 (status, enrollment, completion dates, endpoints, sites) when this record is updated. Free accounts track up to 5 trials.
MinorStart date: Sep 20, 2026 to Oct 4, 2026Eligibility criteria expanded, about 20 words · Secondary endpoints: details revised at 2 of 4 entries · Study description expanded, about 13 wordsShow 3 more changesShow less
Eligibility criteria expanded, about 20 words
Secondary endpoints: details revised at 2 of 4 entries
Study description expanded, about 13 words
NotableEnrolling by invitation onlyStart date: Sep 16, 2026 to Sep 20, 2026 · Site contact updatedv17Show 2 more changesShow less
Start date: Sep 16, 2026 to Sep 20, 2026
Site contact updated
MinorStart date: Sep 15, 2026 to Sep 16, 2026v16
MinorStart date: Sep 14, 2026 to Sep 15, 2026v15
MinorStart date: Sep 10, 2026 to Sep 14, 2026v14
MinorStart date: Sep 9, 2026 to Sep 10, 2026v13
MinorStart date: Sep 8, 2026 to Sep 9, 2026v12
MinorStart date: Sep 7, 2026 to Sep 8, 2026v11
MinorStart date: Sep 6, 2026 to Sep 7, 2026v10
MinorStart date: Sep 3, 2026 to Sep 6, 2026v9
MinorStart date: Sep 2, 2026 to Sep 3, 2026v8
MinorStart date: Sep 1, 2026 to Sep 2, 2026v7
MinorStart date: Aug 31, 2026 to Sep 1, 2026v6
MinorStart date: Aug 30, 2026 to Aug 31, 2026v5
MinorStart date: Aug 27, 2026 to Aug 30, 2026v4
MinorStart date: Aug 26, 2026 to Aug 27, 2026v3
MinorStart date: Aug 25, 2026 to Aug 26, 2026v2
MinorStudy First Submitted
Stopped: an ongoing trial was terminated, suspended or withdrawn.
Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
Minor: routine progress and housekeeping, such as sites added or enrollment closed.
Background:
Warts, hypogammaglobulinemia, infections and myelokathexis syndrome (WHIMs) is a rare disorder that affects the immune system. People with WHIMs can have severe infections all over their body. WHIMs is caused by a mutation in the CXCR4 gene. Treatment with drugs can help control the infections but does not cure the disorder. Researchers want to try a treatment where they collect stem cells from a person with WHIMS, use base-editing to replace the bad gene with a healthy version, and return the new cells to the person. This could cure WHIMs.
Objective:
To test a treatment using base-edited stem cells in people with WHIMs.
Eligibility:
People aged 3 years and older with WHIMs.
Design:
The study has 4 stages.
Stage 1: Screening. Participants will be screened at 1 or more visits. They will have a physical exam with blood tests. A sample of tissue and fluid (biopsy) will be taken from the bone marrow in the hip.
Stage 2: Apheresis. Blood will be taken from the body through a needle; the blood will pass through a machine that separates out the stem cells. The remaining blood will be returned to the body through a different needle. The collected stem cells will undergo gene editing.
Stage 3: Treatment. Participants will stay in the hospital for about 4 weeks. They will receive 3 drugs to prepare their body for the procedure. Then the edited stem cells will be returned to their bloodstream. They will stay in the hospital until they recover.
Stage 4: Follow-up. Participants will have 8 follow-up visits over 5 years. Long-term visits will continue for 15 years.