RecruitingPhase 3

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

United States150 participantsStarted 2022-11-07

Plain-language 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.

Who can participate

Age range15 Years – 64 Years

SexALL

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Inclusion criteria

✓. Willingness to obtain magnetic resonance angiogram (MRA) image at local imaging facility.
✓. A genetic test confirming the presence of a pathogenic COL3A1 variant (classified as likely pathogenic or pathogenic according to ACMG/AMP Guidelines.
✓. Patients must be ≥ 15 years of age at the time of randomization.
✓. Able and willing to discontinue use of β-blockers prior to randomization.

Exclusion criteria

✕. 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.
✕. Arterial rupture or dissection, uterine rupture, and/or intestinal rupture within 6 months prior to Screening.
✕. Patients unable to discontinue β-blocker treatment prior to randomization.
✕. Unable or unwilling to complete the study procedures.
✕. Breastfeeding, pregnancy, or planned pregnancy during the trial.
✕. 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.
✕. Use of any prohibited medications

What they're measuring

Time to first occurrence of a vEDS-related clinical event requiring medical attention: Fatal/nonfatal cardiac or arterial events [including dissection or rupture], uterine rupture, intestinal rupture, and/or unexplained sudden death

Timeframe: Over the double-blind period (estimated to be 40 months)

Trial details

NCT IDNCT05432466

SponsorAcer Therapeutics Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-03-01

Contact for this trial

Sheila Woodhouse, MD; Ph.D.

984-377-3737 sheila.woodhouse@science37.com

View on ClinicalTrials.gov More Vascular Ehlers-Danlos Syndrome trials