150 Participants Needed

Celiprolol for Ehlers-Danlos Syndrome

(DiSCOVER Trial)

SW
JC
SW
JC
Overseen ByJonathan Cotliar, MD; Ph.D.
Age: < 65
Sex: Any
Trial Phase: Phase 3
Sponsor: Acer Therapeutics Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 3 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

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).

Will I have to stop taking my current medications?

You will need to stop taking β-blockers (a type of heart medication) before joining the trial. The protocol does not specify about other medications.

How does the drug Celiprolol differ from other treatments for Ehlers-Danlos Syndrome?

Celiprolol is unique for Ehlers-Danlos Syndrome because it is a beta-blocker that may help strengthen blood vessels, which is particularly important for this condition that affects connective tissue. Unlike other treatments, Celiprolol specifically targets the cardiovascular complications associated with Ehlers-Danlos Syndrome, offering a novel approach compared to general supportive care.12345

What data supports the effectiveness of the drug celiprolol for treating vascular Ehlers-Danlos syndrome?

Research shows that celiprolol, a type of beta-blocker, significantly reduces the risk of arterial events like dissections and ruptures in patients with vascular Ehlers-Danlos syndrome. This drug helps by lowering heart rate and pressure, which reduces stress on the arteries, and has shown significant benefits in clinical trials.678910

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I can stop taking β-blockers 28 days before joining the study.
I have a confirmed genetic mutation in the COL3A1 gene.
I am 15 years old or older.
See 1 more

Exclusion Criteria

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
I am not taking any medications that are not allowed in the study.
I have not had any major artery, uterus, or intestine ruptures in the last 6 months.
See 4 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either celiprolol or placebo in a double-blind manner, with titration over the first 4 months

40 months
Decentralized visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term with celiprolol

What Are the Treatments Tested in This Trial?

Interventions

  • Celiprolol
Trial Overview 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.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Placebo BIDExperimental Treatment1 Intervention
Group II: ACER-002 (celiprolol) 200 mg BIDExperimental Treatment1 Intervention

Celiprolol is already approved in European Union for the following indications:

🇪🇺
Approved in European Union as Celiprolol for:

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Who Is Running the Clinical Trial?

Acer Therapeutics Inc.

Lead Sponsor

Trials
3
Recruited
220+

Published Research Related to This Trial

In a study involving 53 patients with vascular Ehlers-Danlos syndrome, celiprolol significantly reduced the occurrence of arterial events (ruptures or dissections) compared to a control group, with only 20% of the celiprolol group experiencing these events versus 50% in the control group.
The trial, which lasted an average of 47 months and was stopped early due to positive results, suggests that celiprolol could be an effective preventive treatment for this severe condition, although further research is needed to determine its efficacy in patients without the specific collagen mutation.
Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial.Ong, KT., Perdu, J., De Backer, J., et al.[2022]
Celiprolol is a unique β-blocker that not only blocks β1 receptors but also partially activates β2 receptors, which may provide additional benefits in treating conditions like vascular type Ehlers-Danlos syndrome.
In a significant study, celiprolol showed promising results in reducing arterial events in patients with vascular Ehlers-Danlos syndrome, leading to the premature termination of the trial due to its effectiveness.
Celiprolol: A Unique Selective Adrenoceptor Modulator.Nawarskas, JJ., Cheng-Lai, A., Frishman, WH.[2019]
In a study of 40 Swedish patients with vascular Ehlers-Danlos syndrome (vEDS) treated with the beta blocker celiprolol, 65% were able to reach the target dose of 400 mg daily, indicating good tolerability for most patients.
The annual risk of major vascular events was 4.7%, which is comparable to the protective effect observed in the BBEST trial, suggesting that celiprolol may help reduce the risk of serious complications in vEDS patients.
Celiprolol Treatment in Patients with Vascular Ehlers-Danlos Syndrome.Baderkhan, H., Wanhainen, A., Stenborg, A., et al.[2021]

Citations

Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial. [2022]
Celiprolol: A Unique Selective Adrenoceptor Modulator. [2019]
Celiprolol Treatment in Patients with Vascular Ehlers-Danlos Syndrome. [2021]
DDAVP therapy controls bleeding in Ehlers-Danlos syndrome. [2019]
Celiprolol but not losartan improves the biomechanical integrity of the aorta in a mouse model of vascular Ehlers-Danlos syndrome. [2022]
[Pharmacotherapy of osteoporosis: evidence-based clinical practice]. [2007]
Long term effects of Tibolone on postmenopausal women with uterine myomas. [2019]
8.Czech Republicpubmed.ncbi.nlm.nih.gov
[Raloxifene in clinical practice. Results of the non-interventional study CORAL (COmpliance with RALoxifene)]. [2015]
Optimized semisolid self-nanoemulsifying system based on glyceryl behenate: A potential nanoplatform for enhancing antitumor activity of raloxifene hydrochloride in MCF-7 human breast cancer cells. [2021]
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Clinically favorable effects of raloxifene on bone mineral density and serum lipids: population assessment via bivariate analysis. [2020]
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