240 Participants NeededMy employer runs this trial

Engasertib for Osler-Weber-Rendu Syndrome

Recruiting at 3 trial locations
VT
Overseen ByVaderis Therapeutics AG
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: Vaderis Therapeutics AG
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

What You Need to Know Before You Apply

What is the purpose of this trial?

The primary objective of this trial is to assess the efficacy of engasertib 40 mg once daily (QD) in reducing the frequency of epistaxis compared to placebo QD during 28 weeks of double-blind treatment in participants with moderate to severe HHT.

Are You a Good Fit for This Trial?

This trial is for adults (18+) with moderate to severe hereditary hemorrhagic telangiectasia (HHT), who have frequent nosebleeds, visible blood vessel spots on the skin or mouth, possible organ involvement, and either anemia or recent iron/blood transfusions.

Inclusion Criteria

I am 18 years old or older.
I have multiple red spots on my lips, mouth, fingers, or nose.
I have frequent nosebleeds that last several minutes and scored over 4 on the ESS test.
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What Are the Treatments Tested in This Trial?

Interventions

  • Engasertib

Trial Overview

The study compares a daily dose of engasertib versus a placebo over 28 weeks to see if engasertib can reduce how often participants get nosebleeds. Participants are randomly assigned to one of the two groups without knowing which they receive.

How Is the Trial Designed?

2

Treatment groups

Experimental Treatment

Placebo Group

Group I: Engasertib 40 mgExperimental Treatment1 Intervention
Group II: PlaceboPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Vaderis Therapeutics AG

Lead Sponsor

Trials
1
Recruited
80+