Recruiting
Phase 3

Celiprolol

Sponsor:

Acer Therapeutics Inc.

Code:

NCT05432466

Conditions

Vascular Ehlers-Danlos Syndrome

Eligibility Criteria

Sex: All

Age: 15 - 64

Healthy Volunteers: Not accepted

Interventions

ACER-002 (celiprolol) 200 mg BID

Placebo BID

Study Details

Brief 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.

Conditions

Vascular Ehlers-Danlos Syndrome

Study ID

NCT05432466

Start date

Nov 7, 2022

Status verified date

Aug, 2025

Completion date

Apr 1, 2029

Anticipated

Primary completion date

Mar 1, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 15 - 64

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

150 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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

Interventions

ACER-002 (celiprolol) 200 mg BID

ACER-002 (celiprolol) 200 mg BID

Placebo BID

placebo for ACER-002

Primary outcome measure

  • 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 [ Time Frame: Over the double-blind period (estimated to be 40 months) ]

Central Contacts and Locations

Central contacts

Jonathan Cotliar, MD; Ph.D.

984-377-3737jonathan@science37.com

Locations

Science 37

Recruiting

Culver City, California, United States, 90230

Contacts

More Information

Sponsor

Acer Therapeutics Inc.

Last update posted

Aug 24, 2025

Last verified

Aug, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Acer Therapeutics Inc. on 2025-08-24.