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

NCT05432466 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: ACER-002-301
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• 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

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

Secondary Outcomes (5)

• Number and proportion of patients reporting 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

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

• Number and percentage of patients with adverse events

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

• Number and percentage of Serious Adverse Events (SAE)

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

• Number and percentage of patient deaths

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

• Number and percentage of patient discontinuations

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

Study Arms (2)

ACER-002 (celiprolol) 200 mg BID

EXPERIMENTAL

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

Drug: ACER-002 (celiprolol) 200 mg BID

Placebo BID

EXPERIMENTAL

Placebo twice daily (BID) Placebo given orally to mimic ACER-002 (celiprolol) administration

Drug: Placebo BID

Interventions

ACER-002 (celiprolol) 200 mg BID DRUG

ACER-002 (celiprolol) 200 mg BID

Also known as: ACER-002, celiprolol

ACER-002 (celiprolol) 200 mg BID

Placebo BID DRUG

placebo for ACER-002

Also known as: placebo

Placebo BID

Eligibility Criteria

• Age: 15 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Acer Therapeutics Inc.: lead

Study Sites (1)

Science 37

Culver City, California, 90230, United States

RECRUITING

MeSH Terms

Conditions

Ehlers-Danlos Syndrome, Type IV

Interventions

Celiprolol; BID protein, human

Condition Hierarchy (Ancestors)

Aortic Dissection; Dissection, Blood Vessel; Aneurysm; Vascular Diseases; Cardiovascular Diseases; Ehlers-Danlos Syndrome; Hemostatic Disorders; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Phenoxypropanolamines; Propanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Propanols; Phenylurea Compounds; Urea; Amides; Amines; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons

Central Study Contacts

Sheila Woodhouse, MD; Ph.D.

CONTACT

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

Jonathan Cotliar, MD; Ph.D.

CONTACT

984-377-3737; jonathan@science37.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The patient, the Investigator, and other members of the staff involved with the study will remain blinded to the two study treatments (celiprolol and placebo).
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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 at Month 40 assuming that at least 46 vEDS-related clinical events have occurred. If the number of vEDS-related events is fewer than 46 at Month 40, the study will continue until the required number of events have occurred prior to ending the double-blind portion of the study. Following the double-blind treatment period or occurrence of a vEDS-related clinical event, patients have the option to participate in an OLE period. A total of approximately 150 patients who meet all the inclusion and none of the exclusion criteria will be enrolled and randomized 2:1 to receive either celiprolol or placebo, respectively. An approximately 40-month treatment period followed by an optional open-label extension study.

Study Record Dates

• First Submitted: June 21, 2022
• First Posted: June 27, 2022
• Study Start: November 7, 2022
• Primary Completion (Estimated): March 1, 2029
• Study Completion (Estimated): April 1, 2029
• Last Updated: August 24, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)