Clinical Research Directory
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2 clinical studies listed.
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Tundra lists 2 Telomere Biology Disorders With Bone Marrow Failure clinical trials. Each listing includes eligibility criteria, study locations, and direct links to research sites in the Tundra directory.
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NCT06817590
Nucleoside Therapy in Patients With Telomere Biology Disorders
The goal of this clinical trial is to learn if a combination therapy of deoxycytidine (dC) plus deoxythymidine (dT) is safe in patients with telomere biology disorders. The main questions it aims to answer are: * Is the therapy safe with tolerable side effects in patients with telomere biology disorders? * Are problems with the bone marrow or blood or lungs changed after 6 months of dC+dT treatment in patients with telomere biology disorders? Participants will: * Take study drug by mouth three times daily for 24 weeks * Make approximately 2 visits to Boston Children's Hospital during the 24 weeks: once at the beginning of treatment and once at the end of treatment. * Go to a lab for a blood draw an additional 6 times during treatment. * Have 9 phone calls with a research nurse, including one 4 weeks after treatment ends. * Keep a diary to track doses of study drug that were taken or missed.
Gender: All
Ages: 1 Year - 70 Years
Updated: 2026-03-18
1 state
NCT05868499
Rollover Study From EXG-US-01
This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).
Gender: All
Ages: 18 Years - Any
Updated: 2024-07-12
1 state