RecruitingPhase 1NCT06817590

Nucleoside Therapy in Patients With Telomere Biology Disorders


Sponsor

Suneet Agarwal

Enrollment

36 participants

Start Date

Sep 29, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

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.


Eligibility

Min Age: 1 YearMax Age: 70 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called deoxycytidine and a drug called deoxythymidine for people with dyskeratosis congenita, hoyeraal-hreidarsson syndrome, and other related conditions. The study is currently recruiting participants at 1 location.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGdeoxycytidine

Oral administration, in combination with deoxythymidine

DRUGdeoxythymidine

Oral administration, in combination with deoxycytidine


Locations(1)

Boston Childrens Hospital

Boston, Massachusetts, United States

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NCT06817590


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