We are very excited to share that the Unravel Biosciences exploratory study for Pitt Hopkins syndrome is officially on clinicaltrials.gov. More details can be found here: https://clinicaltrials.gov/study/NCT07150026.

This new study will test RVL-001, a repurposed formulation of vorinostat, in individuals with Pitt Hopkins Syndrome (PTHS). Vorinostat is a histone deacetylase inhibitor that works by modulating gene activity— potentially improving cellular function. The trial will take place in Medellín, Colombia, enrolling six participants and comparing RVL-001 against placebo. It will be conducted with PECET, the University of Antioquia’s certified clinical research unit, under Colombia’s INVIMA priority review program for rare diseases. Enrollment is anticipated to begin in late 2025.