Comparing Celiprolol to Placebo for Vascular Ehlers-Danlos Syndrome

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Science 37
  Culver City, California, United States Recruiting

Study contacts

• Study coordinator: Sheila Woodhouse, MD; Ph.D.
• Email: sheila.woodhouse@science37.com
• Phone: 984-377-3737

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Registry for Ehlers-Danlos Syndrome

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.