Could a simple sugar pill fix faulty telomeres? early trial launches

NCT ID NCT06817590

Recruiting now Disease control Sponsor: Suneet Agarwal Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Aug 05, 2026 · Updated 3 times

Summary

This early-stage trial is testing whether a combination of two natural substances, deoxycytidine and deoxythymidine, is safe for people with telomere biology disorders. These rare genetic conditions cause premature aging, bone marrow failure, and lung scarring. Up to 36 participants will take the oral medication three times daily for 24 weeks, with close monitoring for side effects and changes in blood counts and lung function.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: deoxycytidine and deoxythymidine (nucleosides taken by mouth)
What this could lead to: If safe and effective, this treatment could help improve blood counts and lung function in people with telomere biology disorders, offering a new way to manage these rare conditions.
What could go wrong: This is a very early phase 1 trial with only 36 participants, so safety and any benefits are uncertain. Side effects like diarrhea may occur, and the treatment may not work for everyone.

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Conditions

The condition(s) this trial relates to.

Bone Marrow Failure Disorders dyskeratosis congenita dyskeratosis congenita and related telomere biology disorder Hoyeraal-Hreidarsson syndrome inherited interstitial lung disease pulmonary fibrosis pulmonary fibrosis and/or bone marrow failure, telomere-related Revesz syndrome telomere syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DYSKERATOSIS CONGENITA HOYERAAL HREIDARSSON SYNDROME INTERSTITIAL LUNG DISEASE DUE TO SYSTEMIC DISEASE (TELOMERE BIOLOGY DISORDER) PULMONARY FIBROSIS, FAMILIAL (TELOMERE BIOLOGY DISORDER) REVESZ SYNDROME TELOMERE BIOLOGY DISORDERS TELOMERE BIOLOGY DISORDERS WITH BONE MARROW FAILURE

Contacts and locations

Locations

Boston Childrens Hospital
RECRUITING

Boston, Massachusetts, 02115, United States

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