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

Vascular Ehlers-Danlos Syndrome Clinical Trial

— DiSCOVER  

Official title:

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 Details — Status: Recruiting

Administrative data

• NCT number: NCT05432466
• Other study ID #: ACER-002-301
• Status: Recruiting
• Phase: Phase 3
• Start date: November 7, 2022
• Est. completion date: April 1, 2029

Study information

• Verified date: June 2024
• Source: Acer Therapeutics Inc.
• Contact: Sheila Woodhouse, MD; Ph.D.
• Phone: 984-377-3737
• Email: sheila.woodhouse[@]science37.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 inclusion and none of the exclusion criteria will be enrolled and randomized 2:1 to receive either celiprolol or placebo, respectively.

Following the double-blind treatment period or occurrence of vEDS-related clinical event, patients have the option to participate in an open label extension period.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: April 1, 2029
• Est. primary completion date: March 1, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 15 Years to 64 Years

Eligibility

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 ß-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 ß-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

Related Conditions & MeSH terms

• Ehlers-Danlos Syndrome
• Syndrome
• Vascular Ehlers-Danlos Syndrome

Intervention

Drug:
• ACER-002 (celiprolol) 200 mg BID
ACER-002 (celiprolol) 200 mg BID
• Placebo BID
placebo for ACER-002

Locations

Country	Name	City	State
United States	Science 37	Culver City	California

Sponsors (1)

Lead Sponsor	Collaborator
Acer Therapeutics Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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)
Secondary	Number and percentage of patients with adverse events	An Adverse Event (AE) is defined as any untoward medical occurrence associated with the use of the investigational product in humans, whether or not considered related to investigational product. An AE can be any unfavorable and unintended sign (e.g., an abnormal laboratory finding), symptom, or disease temporally associated with any use of the investigational product, without any judgment about causality and irrespective of route of administration, formulation, or dose, including an overdose.	Over the double-blind period (estimated to be 40 months)
Secondary	Number and percentage of Serious Adverse Events (SAE)	An AE is considered "serious" if, in the view of either the investigator or Acer, it results in any of the following outcomes: Death, Is immediately life threatening; Requires in-patient hospitalization or prolongation of existing hospitalization; Results in persistent or significant disability or incapacity; Results in a congenital abnormality or birth defect; Is an important medical event that may jeopardize the subject or may require medical intervention to prevent one of the outcomes listed above.	Over the double-blind period (estimated to be 40 months)
Secondary	Number and percentage of patient deaths		Over the double-blind period (estimated to be 40 months)
Secondary	Number and percentage of patient discontinuations	Discontinuation or withdrawal from the study	Over the double-blind period (estimated to be 40 months)