Phase 3 Study Comparing Celiprolol and Placebo for Patients Aged 15 to 64 With COL3A1-Positive Vascular Ehlers-Danlos Syndrome

What this Trial Is About

Researchers are evaluating the effects of celiprolol in patients diagnosed with vascular Ehlers-Danlos Syndrome vEDS confirmed by a COL3A1 gene mutation. This Phase 3, randomized, double-blind, placebo-controlled study uses a decentralized design to compare celiprolol with a placebo. The trial aims to measure the time until the first vEDS-related serious clinical event that requires medical attention, such as cardiac or arterial events, uterine or intestinal rupture, or unexplained sudden death. Participants will be randomly assigned in a 21 ratio to receive either celiprolol or placebo. Celiprolol dosing starts with a titration phase beginning at 100 mg once daily and gradually increasing to 200 mg twice daily by the fourth month, continuing at that dose for the remainder of the treatment period. The placebo group will receive a matching oral placebo twice daily. After completing the double-blind treatment or experiencing a vEDS-related event, participants may choose to join an open-label extension phase. Throughout the study, participants will undergo assessments including magnetic resonance angiograms MRA at local facilities and monitoring for vEDS-related clinical events. Researchers will track the occurrence of adverse events, serious adverse events, deaths, and discontinuations during an estimated 40-month double-blind period. The total duration of participation varies depending on the timing of events and extension enrollment.

Clinical Trial to Compare the Efficacy of Celiprolol to Placebo in Patients With Vascular Ehlers-Danlos Syndrome