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
Acer Therapeutics Inc.
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.
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. The double-blind portion of this study is intended to end if statistical significance is reached at the interim analysis (accrual of 28 vEDS-related events requiring medical attention; estimated to take 24 months) or after accrual of 46 vEDS related clinical events requiring medical attention (estimated to take 40 months). A total of approximately 150 patients who meet all the…
Eligibility
- Age range
- 15–64 years
- Sex
- All
- Healthy volunteers
- No
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 va…
Interventions
- DrugACER-002 (celiprolol) 200 mg BID
ACER-002 (celiprolol) 200 mg BID
- DrugPlacebo BID
placebo for ACER-002
Location
- Science 37Culver City, California