Comparing Celiprolol to Placebo for Vascular Ehlers-Danlos Syndrome

A Phase 3 Randomized, Double-Blind, Decentralized Clinical Trial to Compare the Efficacy of Celiprolol to Placebo in the Treatment of Patients With COL3A1-Positive Vascular Ehlers-Danlos Syndrome

This study is testing whether the medication celiprolol can help people with Vascular Ehlers-Danlos Syndrome feel better compared to a placebo.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 150 (estimated)
• Ages: 15 Years to 64 Years
• Sex: All
• Sponsor: Acer Therapeutics Inc. (industry)
• Locations: 1 site (Culver City, California)
• Trial ID: NCT05432466 on ClinicalTrials.gov

What this trial studies

This Phase 3 clinical trial aims to evaluate the efficacy of celiprolol in patients with genetically confirmed Vascular Ehlers-Danlos Syndrome (vEDS) caused by COL3A1 mutations. The study is designed as a randomized, double-blind, placebo-controlled trial, enrolling approximately 150 patients who will be assigned to receive either celiprolol or a placebo. The trial will utilize a decentralized approach, allowing for remote participation and monitoring. The primary endpoint is to assess the occurrence of vEDS-related clinical events requiring medical attention over a specified period.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals aged 15 and older with a confirmed pathogenic COL3A1 variant.

Not a fit: Patients without a confirmed COL3A1-positive test or those with certain recent vascular complications may not benefit from this study.

Why it matters

Potential benefit: If successful, this trial could provide a new treatment option for patients with Vascular Ehlers-Danlos Syndrome, potentially reducing the frequency of serious vascular events.

How similar studies have performed: While this approach is novel in the context of vEDS, similar studies evaluating beta-blockers for vascular conditions have shown promise.

Eligibility 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 prior to randomization.

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 prior to randomization.
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

Where this trial is running

Culver City, California

• Science 37 — Culver City, California, United States (RECRUITING)

Study contacts

• Study coordinator: Sheila Woodhouse, MD; Ph.D.

Conditions: Vascular Ehlers-Danlos Syndrome