Vascular Ehlers-Danlos SyndromePhase 3DiSCOVERNCT05432466
Clinical Trial to Compare the Efficacy of Celiprolol to Placebo in Patients With Vascular Ehlers-Danlos Syndrome
A Phase 3 study testing ACER-002 (celiprolol) 200 mg BID in people with Vascular Ehlers-Danlos Syndrome, run by Acer Therapeutics Inc. at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 15 to 64 years
- Conditions the study is for
- Vascular Ehlers-Danlos Syndrome
- What is being tested?
- ACER-002 (celiprolol) 200 mg BID
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 150 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Nov 2022to 1 Apr 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 15 to 64 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Science 37
Culver City, California, 90230
Sheila Woodhouse, MD, 984-377-3737, sheila.woodhouse@science37.com
Johnathan Cotliar, MD, 984-377-3737, jonathan@science37.com
About this study
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
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.
Read the detailed 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.
- Conditions
- Vascular Ehlers-Danlos Syndrome
- Being tested
- ACER-002 (celiprolol) 200 mg BID, Placebo BID
- Medical subject headings
- Ehlers-Danlos Syndrome, Type IV
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ACER-002 (celiprolol) 200 mg BIDDrug
Also called ACER-002, celiprolol
ACER-002 (celiprolol) 200 mg BID
Placebo BIDDrug
Also called placebo
placebo for ACER-002
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: ACER-002 (celiprolol) 200 mg BID |
| Placebo BID | Experimental | Placebo twice daily (BID) Placebo given orally to mimic ACER-002 (celiprolol) administration Receives: Drug: Placebo BID |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: Over the double-blind period (estimated to be 40 months)
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.
Measured: Over the double-blind period (estimated to be 40 months)
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.
Measured: Over the double-blind period (estimated to be 40 months)
Number and percentage of patient deaths
Measured: Over the double-blind period (estimated to be 40 months)
Number and percentage of patient discontinuations
Discontinuation or withdrawal from the study
Measured: Over the double-blind period (estimated to be 40 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2022
- Study start7 Nov 2022
- Primary completion1 Mar 2029 (estimated)
- Study completion1 Apr 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Vascular Ehlers-Danlos SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model 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 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.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Acer Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05432466
- Sponsor's protocol ID
- ACER-002-301
- Acronym
- DiSCOVER
- Status last verified
- Aug 2025
- First submitted
- 21 Jun 2022
- Last updated
- 24 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05432466
- Acronym / sponsor ID
- DiSCOVER / ACER-002-301
- Conditions
- Vascular Ehlers-Danlos Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Acer Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 7 Nov 2022 (actual)
- Primary completion
- 1 Mar 2029 (estimated)
- Study completion
- 1 Apr 2029 (estimated)
- First posted
- 27 Jun 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model 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 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.
| Arm | Type | Receives |
|---|---|---|
| ACER-002 (celiprolol) 200 mg BID | Experimental | Drug: ACER-002 (celiprolol) 200 mg BIDACER-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 |
| Placebo BID | Experimental | Drug: Placebo BIDPlacebo twice daily (BID) Placebo given orally to mimic ACER-002 (celiprolol) administration |
Who may qualify
- Ages
- 15 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ACER-002 (celiprolol) 200 mg BIDAlso: ACER-002, celiprololACER-002 (celiprolol) 200 mg BID | Drug | ACER-002 (celiprolol) 200 mg BID |
| Placebo BIDAlso: placeboplacebo for ACER-002 | Drug | Placebo BID |
Primary outcome
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)
5 secondary outcomes
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
Time frame: Over the double-blind period (estimated to be 40 months)
Number and percentage of patients with adverse events
Time frame: Over the double-blind period (estimated to be 40 months)
Number and percentage of Serious Adverse Events (SAE)
Time frame: Over the double-blind period (estimated to be 40 months)
Number and percentage of patient deaths
Time frame: Over the double-blind period (estimated to be 40 months)
Number and percentage of patient discontinuations
Time frame: Over the double-blind period (estimated to be 40 months)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ACER-002 (celiprolol) 200 mg BID in people with Vascular Ehlers-Danlos Syndrome, run by Acer Therapeutics Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sheila Woodhouse, MD; Ph.D. (984-377-3737, sheila.woodhouse@science37.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2025.