Gene therapy for WHIM syndrome
Treatments studied
Part of Blood & lymphatic, Immune system & allergy, Infections, Skin clinical trials.
This trial tests a one-time gene therapy treatment for WHIM syndrome, a rare immune disorder. It aims to fix the genetic cause to improve your body's ability to fight infections. Your stem cells will be collected, edited in a lab, and given back to you after some chemotherapy to prepare your body.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age 3 years or older and weigh at least 15 kilograms (about 33 pounds)
- You must have the specific genetic change (CXCR4-R334X) that causes WHIM syndrome
- Able to have your stem cells collected through apheresis, a process similar to donating blood
- Past lab results showing low white blood cell counts (neutropenia) or low antibody levels (IgG) requiring treatment
- Willing to store samples of your blood and tissue for research, and use effective birth control if you can become pregnant or cause pregnancy
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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