NCT05463679

Investigate Efficacy, Safety, and Pharmacokinetics of Enzastaurin for the Prevention of Arterial Events in Patients With Vascular Ehlers-Danlos Syndrome.

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)

• Status: Suspended
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 260 (estimated)
• Sponsor: Aytu BioPharma, Inc. · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to investigate the efficacy of enzastaurin compared to placebo in preventing arterial events (rupture, dissection, pseudoaneurysm, carotid-cavernous sinus fistula, or aneurysm, fatal or not) leading to intervention or mortality attributable to an arterial event in patients with vEDS confirmed with pathogenic heterozygous COL3A1 gene mutations predicted to derive a mutant protein.

Detailed description

The primary efficacy endpoint of this study will be defined as the time to intervention for an arterial event (rupture, dissection, pseudoaneurysm, carotid-cavernous sinus fistula, or aneurysm, fatal or not) or mortality attributable to an arterial event, as adjudicated by an Event Committee, and will be analyzed for difference of active vs. placebo treatments on top of background standard of care, using survival statistical analysis. Furthermore, secondary endpoints will include the rate of intestinal rupture, pneumothorax, and retinal detachment, as adjudicated by an Event Committee, safety and tolerability, as well as hospitalizations and Health Related Quality of Life (HQRL) measures.

Conditions

• Vascular Ehlers-Danlos Syndrome

Interventions

Type	Name	Description
DRUG	Enzastaurin	500 mg QD orally in the form of four 125 mg tablets with background standard of care
DRUG	Placebo	Placebo to match enzastaurin 500 mg QD orally in the form of four 125 mg tablets with background standard of care

Timeline

• Start date: 2025-01-01
• Primary completion: 2025-06-01
• Completion: 2027-03-01
• First posted: 2022-07-19
• Last updated: 2024-03-08

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study