Stem cell transplant for CGD using targeted medications and steroids-free support
Treatments studied
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This trial tests an intensive stem cell transplant approach for people with chronic granulomatous disease (CGD). The study aims to improve outcomes by preparing the body for a donor stem cell graft and using immune “brakes” to reduce immune problems.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or the patient’s guardian, if a minor) can understand the study and sign consent; the child will also assent when appropriate.
- You have a confirmed diagnosis of chronic granulomatous disease (CGD).
- You are age 4 to 65, and you must have significant CGD problems (ongoing inflammation/autoimmunity or CGD infections even on prevention) or low residual enzyme activity in the top levels stated by the study.
- A matched donor is available (a family donor or unrelated donor stem cells) with 10/10 matching or 9/10 matching.
- You must be HIV-negative, not pregnant or breastfeeding, and able to stay within 1 hour of the NIH for the first 3 months after transplant.
- You must be well enough for transplant tests (heart, lung, and liver), and you must agree to the medication/drug access program for alemtuzumab.
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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