ClinicalTrials.gov record NCT05432466

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age15 years old to 64 years old
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 3 randomized, double-blind trial enrolls people with genetically confirmed COL3A1-positive vascular Ehlers-Danlos syndrome, aged 15–64. About 150 participants will be randomized 2:1 to receive oral celiprolol (titrated dosing) or matching placebo during a double-blind period estimated at about 40 months (it may end earlier or run longer depending on when enough vEDS-related events occur); an optional open-label extension is available after the double-blind period or a qualifying vEDS event. The main outcome is time to the first vEDS-related clinical event requiring medical attention.

What it is studying. The study is testing celiprolol given by mouth (with stepwise dose increases) versus placebo, comparing time to first vEDS-related clinical event requiring medical attention in people with COL3A1-positive vEDS.

What the researchers hope to learn. To evaluate the effect of celiprolol versus placebo on time to first vEDS-related clinical event requiring medical attention; and to collect safety data (adverse events, serious adverse events, deaths, and discontinuations).

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

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

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are randomized (2:1) to take either celiprolol (ACER-002) or matching placebo in a double-blind schedule with stepwise dose increases up to 400 mg daily. They must stop other β‑blockers before randomization, have an MRA at a local imaging facility, and attend follow-up visits for safety monitoring and reporting of vEDS‑related clinical events over the double-blind period (estimated up to 40 months). After the double-blind period or after a vEDS-related clinical event, participants may be offered enrollment in an open-label extension. Safety checks and adverse-event reporting are part of study participation.

How the study is structured. Phase 3, randomized, double-blind, placebo-controlled trial with parallel groups. Participants are assigned 2:1 to celiprolol or placebo, take study drug orally with planned dose titration, and are followed during a double-blind period (an interim analysis is planned); after the double-blind period or a vEDS event, participants may opt into an open-label extension.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on vascular Ehlers-Danlos syndrome

Clinical trials6 trials listed for vascular Ehlers-Danlos syndromeEvery trial the registry lists for vascular Ehlers-Danlos syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in vascular Ehlers-Danlos syndrome researchLatest monthly update: September 2026. Includes: Care coordination. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Nov 7, 2022
Primary completion
Mar 1, 2029 (estimated)
Study completion
Apr 1, 2029 (estimated)
Target enrollment
150 participants (estimated)
Last updated on registry
Aug 24, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Acer Therapeutics Inc.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
double-blind
Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
placebo-controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
prohibited medications
A medicine that participants may not use during the study, usually because it could interfere with the study treatment or its measurement.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
2:1
How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
open label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
extension period
A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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