What side effects are associated with this type of intervention?

Common side effects of beta-blockers, such as Celiprolol, include fatigue, cold hands and feet, weight gain, and depression. Some patients may also experience shortness of breath, bradycardia (slow heart rate), and gastrointestinal issues like nausea or diarrhea. It is important to monitor for these side effects and discuss any concerns with a healthcare provider.

What prior FDA approvals exist?

There is limited information on FDA-approved drugs specifically for Ehlers-Danlos Syndrome (EDS). Celiprolol, a beta-blocker being studied for its efficacy in treating vascular EDS, works by decreasing heart rate and blood pressure. Beta-blockers, in general, are used to manage cardiovascular symptoms associated with EDS, but specific FDA approvals for EDS treatments are not well-documented. Patients with EDS often receive treatments aimed at managing symptoms and preventing complications rather than targeting the underlying condition directly.

What other types of research exist?

As of 2024, promising alternatives to Celiprolol for Ehlers-Danlos Syndrome patients include Losartan, an angiotensin II receptor blocker, which has shown potential in reducing aortic dilation in related connective tissue disorders. Additionally, ongoing research into gene therapy and other targeted molecular treatments may offer new avenues for managing vEDS more effectively.

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Beta Blocker

Celiprolol for Ehlers-Danlos Syndrome (DiSCOVER Trial)

Phase 3

Recruiting

Research Sponsored by Acer Therapeutics Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Must not have

Use of any prohibited medications

Unable or unwilling to complete the study procedures

Timeline

Screening 3 weeks

Treatment Varies

Follow Up over the double-blind period (estimated to be 40 months)

Awards & highlights

Summary

This trial is testing celiprolol, a medication, in patients with a genetic condition called COL3A1-positive vEDS. The goal is to see if it can make their blood vessels stronger and reduce medical issues related to their condition. Celiprolol has shown protective effects in patients with vascular Ehlers-Danlos syndrome (vEDS).

Who is the study for?

This trial is for individuals with genetically confirmed vascular Ehlers-Danlos Syndrome (vEDS) who are at least 15 years old. Participants must not have had certain medical events like arterial rupture in the past 6 months, be pregnant, or use β-blockers within 28 days prior to starting the study.

What is being tested?

The study tests Celiprolol's effectiveness against a placebo in patients with vEDS. It's a Phase 3 trial where participants are randomly assigned to receive either Celiprolol or a placebo twice daily, and it uses remote monitoring methods.

What are the potential side effects?

While specific side effects of Celiprolol aren't listed here, common ones may include low blood pressure, dizziness, fatigue, headache, upset stomach or heart-related issues. Placebo pills typically have no active ingredients but can cause perceived side effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a confirmed genetic mutation in the COL3A1 gene.

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I am 15 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not taking any medications that are not allowed in the study.

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I am unable or unwilling to follow the study's required procedures.

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My test for COL3A1 was either negative or showed a variant not linked to disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ over the double-blind period (estimated to be 40 months)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~over the double-blind period (estimated to be 40 months)

This trial's timeline: 3 weeks for screening, Varies for treatment, and over the double-blind period (estimated to be 40 months) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Number and percentage of Serious Adverse Events (SAE)

Number and percentage of patient discontinuations

Number and percentage of patients with adverse events

Trial Design

2Treatment groups

Experimental Treatment

Group I: Placebo BIDExperimental Treatment1 Intervention

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

Group II: ACER-002 (celiprolol) 200 mg BIDExperimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Placebo BID

2021

Completed Phase 3

~640

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Beta-blockers like Celiprolol are commonly used in the treatment of Ehlers-Danlos Syndrome (EDS), particularly vascular EDS, due to their ability to decrease heart rate and blood pressure by blocking beta-adrenergic receptors. This reduction in cardiovascular stress is vital for EDS patients as it helps to minimize the risk of arterial rupture and other severe cardiovascular complications, which are significant concerns in this patient population. By understanding and utilizing these mechanisms, treatment can more effectively target the life-threatening aspects of EDS.