Nucleoside therapy for telomere disorders
Part of Blood & lymphatic, Genetic & congenital, Lungs & breathing, Skin clinical trials.
This trial tests a nucleoside therapy (dC/dT) for people with telomere biology disorders, which cause premature aging and problems like low blood counts, lung or liver issues. The goal is to see if the treatment can help manage these symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age 1 to 70 years old
- Diagnosed with a telomere biology disorder (based on very short telomeres or a specific gene mutation)
- Have at least one active symptom like low blood counts, bone marrow problems, lung or liver issues, bleeding, immune problems, or eye/nerve issues
- Able to take liquids by mouth or feeding tube
- Not currently needing a bone marrow transplant for very severe aplastic anemia within the next 6 months
- Not taking certain blood-boosting medications (androgens, growth factors) within the past 30 days
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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