Gene editing trial for X-linked chronic granulomatous disease
Treatments studied
Part of Blood & lymphatic, Genetic & congenital, Immune system & allergy clinical trials.
This trial tests a new gene-editing treatment for men with a specific genetic mutation that causes X-linked chronic granulomatous disease (CGD). The goal is to fix the faulty gene in your own blood stem cells to help your immune system fight infections better.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be a male age 18 or older.
- You must have the CYBB c.676 C>T gene mutation confirmed by a blood test.
- You must have had at least one serious infection or inflammation that needed hospital care.
- Your doctor must have told you that your condition has a poor outlook.
- A store of your own blood stem cells must be ready for treatment.
- If you are sexually active, you and your partner must use effective birth control during and for 6 months after treatment.
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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This trial tests a new gene therapy for X-linked chronic granulomatous disease (CGD), a condition where the immune system cannot fight certain infections. The treatment uses your own blood stem cells, which are modified in the lab to help your body produce working immune cells.
This trial tests a new gene therapy (SGX-001) for chronic granulomatous disease caused by a specific genetic change. The treatment tries to fix the gene in your immune cells so they can fight infections better, and the trial will follow participants for up to 15 years to make sure it's safe and effective.
This study is checking levels of antibodies and immune-signaling molecules (cytokines) in people with chronic granulomatous disease (CGD). It aims to better understand the immune system in CGD, which may help with future treatments.
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