AR101 - PREVEnt
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 typically caused by a mutation in the COL3A1 gene. This leads to significant risk for serious arterial events like dissections (tearing of the inner wall of artery), pseudoaneurysms and ruptures. 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 therapies for this, and the current standard of care is monitoring it.
The investigational study medication (Enzastaurin) is a well characterized PKCβ inhibitor, which potentially acts as a mediator in preventing spontaneous aortic rupture . The purpose of this study is to learn more about how Enzastaurin might provide benefit to patients with vEDS confirmed with COL3A1 mutations. This study is designed to see how effective Enzastaurin is in specifically in preventing arterial events in patients with vEDS confirmed with pathogenic heterozygous COL3A1 gene mutations.
The study is double-blind placebo controlled, meaning the study will consist of 2 arms and the participants will be randomised in a 1:1 ratio to receive either the study medication or matching placebo. Enzastaurin will be available as 125mg orange-brown tablets and will be administered orally (by mouth) at a dose of 500mg once daily (four 125mg tablets). The placebo 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 it is necessary for safety reasons.
The study is planned to last for approximately 3 years (this includes a treatment period that is approximately 2 ½ years followed by 6 months of open label extension). The study will include around 260 patients from approximately 6 countries.
The Sponsor of the study is Aytu BioPharma Inc.REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
22/EM/0101
Date of REC Opinion
30 May 2022
REC opinion
Further Information Unfavourable Opinion