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

Description

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.

Conditions

Vascular Ehlers-Danlos Syndrome

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Vascular Ehlers-Danlos Syndrome
Intervention / Treatment

-

Contacts and Locations

Culver City

Science 37, Culver City, California, United States, 90230

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • 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

Ages Eligible for Study

15 Years to 64 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Acer Therapeutics Inc.,

Study Record Dates

2029-04-01