PREVEnt (Prevention of Rupture with Enzastaurin in Vascular EhlersDanlos Syndrome Trial)
Research type
Research Study
Full title
A multicenter, randomized, double-blind, placebo-controlled study of enzastaurin for the prevention of arterial events in patients with vascular Ehlers-Danlos Syndrome (vEDS) confirmed with COL3A1 mutations, followed by an open label extension (OLE)
IRAS ID
1005213
Contact name
Suzane Kennedy
Contact email
Sponsor organisation
Aytu BioPharma, Inc.
Eudract number
2021-006574-23
Clinicaltrials.gov Identifier
Research summary
Vascular Ehlers-Danlos syndrome (vEDS) is an inherited connective tissue disorder which causes new arterial problems which can be very serious or even fatal. An artery is a type of blood vessel that carries blood and oxygen from the heart to the rest of the body. vEDS affects about 1 in 50,000 people worldwide. Nearly 50% of patients with this condition die before the age of 50 years. Currently there are no approved treatments for vEDS, and the current standard of care is arterial monitoring.
The investigational study medication is called enzastaurin. The purpose of this study is to learn more about how enzastaurin might provide benefit to patients with vEDS and see how effective it is in preventing arterial problems.
The study is double-blind placebo controlled, meaning the participants will receive either the study medication or matching placebo (which has no therapeutic effect and is used as a control). Enzastaurin will be available as 125mg orange-brown tablets and will be administered orally (by mouth) with food at a dose of 500mg once daily (four 125mg tablets). The placebo tablet will match this. Neither the participant nor the study doctor/Sponsor will know which treatment group participants are in. However, the study doctor is able to find out this information if necessary for safety reasons. After completing the double-blind phase, all participants will be able to roll over to the Open Label Extension (OLE) phase where all participants will receive enzastaurin.
The double-blind phase will last for up to 30 months and the OLE phase will last until enzastaurin becomes commercially available in the UK or it is no longer viable for the sponsor to commercialise it. The study will include around 260 patients from 6 countries.
The Sponsor of the study is Aytu BioPharma, Inc.REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
22/EM/0228
Date of REC Opinion
21 Dec 2022
REC opinion
Further Information Favourable Opinion