New hope for kids with progeria: experimental drug combo enters mid-stage trial

NCT ID NCT06775041

First seen Jan 26, 2026 · Last updated May 02, 2026 · Updated 13 times

Summary

This study tests an experimental drug called progerinin, given together with the standard medicine lonafarnib, in children with Hutchinson-Gilford progeria syndrome (a rare disease that causes rapid aging). The goal is to find the best dose and check if the combination is safe and tolerable. About 10 children aged 1 year and older will take either lonafarnib alone or lonafarnib plus progerinin.

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Contacts and locations

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

Conditions

Explore the condition pages connected to this study.