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
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 15
Maximum Age: 64
Healthy Volunteers: f
View:
• 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.
Locations
United States
California
Science 37
RECRUITING
Culver City
Contact Information
Primary
Sheila Woodhouse, MD; Ph.D.
sheila.woodhouse@science37.com
984-377-3737
Backup
Jonathan Cotliar, MD; Ph.D.
jonathan@science37.com
984-377-3737
Time Frame
Start Date: 2022-11-07
Estimated Completion Date: 2029-04-01
Participants
Target number of participants: 150
Treatments
Experimental: ACER-002 (celiprolol) 200 mg BID
ACER-002 200 mg twice daily (BID) (after titration):~200 mg morning and 200 mg evening: 400 mg total daily dose~Titration:~Day 1 to Month 1 - 100 mg once daily (QD) evening: 100 mg total daily dose Month 2 to Month 3 - 100 mg morning and 100 mg evening: 200 mg total daily dose Month 3 to Month 4 - 100 mg morning and 200 mg evening: 300 mg total daily dose Month 4 to End of Treatment Period (BID) - 200 mg morning and 200 mg evening: 400 mg total daily dose
Experimental: Placebo BID
Placebo twice daily (BID) Placebo given orally to mimic ACER-002 (celiprolol) administration
Related Therapeutic Areas
Sponsors
Leads: Acer Therapeutics Inc.