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

• Status: Not yet recruiting
• Trial end date: 2025-11-30
• Target enrollment: 0
• Participant gender: All

Summary

This is a prospective, Phase 3, randomized, double-blind, placebo-controlled efficacy study to evaluate celiprolol in patients genetically confirmed as COL3A1-positive vEDS using a decentralized clinical trial design.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Acer Therapeutics Inc.
• Treatments: Celiprolol

Criteria

Inclusion Criteria:

1. Willingness to obtain magnetic resonance angiogram (MRA) image at local imaging facility.

2. A genetic test confirming the presence of a pathogenic COL3A1 variant (classified as likely pathogenic or pathogenic according to ACMG/AMP Guidelines.

3. Patients must be ≥ 15 years of age at the time of randomization.

4. Able and willing to discontinue use of β-blockers 28 days prior to enrollment .

Exclusion Criteria:

1. Lack of a COL3A1-positive test at screening (e.g., COL3A1 benign, likely benign, variant of unknown significance [VUS] or no variant) or presence of a COL3A1 variant but demonstration of a COL3A1 variant reported to be a haploinsufficiency variant.

2. Arterial rupture or dissection, uterine rupture, and/or intestinal rupture within 6 months prior to Screening.

3. Patients unable to discontinue β-blocker treatment within 28 days of enrollment.

4. Unable or unwilling to complete the study procedures.

5. Breastfeeding, pregnancy, or planned pregnancy during the trial.

6. Any medical condition that in the opinion of the Investigator may pose a safety risk to the patient in this study, which may confound efficacy or safety assessment, or may interfere with study participation.

7. Use of any prohibited medications