Menu
Not yet recruiting NCT07743671

A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)

Phase III Interventional Hereditary Hemorrhagic Telangiectasia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Engasertib, Placebo.
Who it may be relevant to
Registry conditions: Hereditary Hemorrhagic Telangiectasia. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 3, Randomized, Double-blind, Placebo-Controlled Study to Assess the Efficacy and Safety of Engasertib in Subjects With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)

Overview

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.

Interventions

  • Drug Engasertib
    Engasertib will be administered as oral capsules.
  • Drug Placebo
    Placebo will be administered as oral capsules.

Primary outcome measures

  • Total Number of Epistaxis Events Through Week 28 [Time frame: Day 1 through Week 28]
Secondary outcome measures (7)
  • Absolute Change from Baseline in Total Duration of Epistaxis at Week 28 [Time frame: Baseline and Week 28]
  • Absolute Change from Baseline in Epistaxis Severity Score (ESS) at Week 28 [Time frame: Baseline and Week 28]
  • Absolute Change from Baseline in the Nasal Outcome Score for Epistaxis in HHT (NOSE HHT) Score at Week 28 [Time frame: Baseline and Week 28]
  • Absolute Change in Red Blood Cell (RBC) Unit Equivalents (RUEs) Received at Week 28 [Time frame: Baseline and Week 28]
  • Patient Global Impression of Change (PGIC) Nosebleeds Sub-score at Week 28 [Time frame: Week 28]
  • HHT-specific Quality of Life (HHT-QoL) Score at Week 28 [Time frame: Week 28]
  • Absolute Change from Baseline in the Intensity-adjusted Duration of Epistaxis per Month at Week 28 [Time frame: Baseline and Week 28]

Eligibility criteria

Inclusion criteria

  • Participants are ≥18 years of age at the Screening Visit.
  • Participants have a definite diagnosis of HHT by the Curaçao criteria, defined as spontaneous and recurrent epistaxis and having at least 2 of the following criteria:
  • Multiple telangiectases at characteristic sites: lips, oral cavity, fingers, or nose;
  • Visceral lesions: gastrointestinal telangiectasia and/or pulmonary, hepatic, cerebral, or spinal arteriovenous malformations (AVMs); or
  • A first degree relative with HHT according to these criteria.
  • Participants must have an ESS >4 at screening, and, in the judgement of the Investigator, participants are expected to have regular epistaxis that typically lasts for several minutes. This criterion is assessed at screening only and does not require reconfirmation prior to randomization on Day 1.
  • Participants have anemia OR in the prior 6 months have received a parenteral infusion of at least 250 mg of iron OR in the prior 6 months have received a red cell or whole blood transfusion.

Exclusion criteria

  • History or current diagnosis of clinically significant electrocardiogram (ECG) abnormalities.
  • History of significant or uncontrolled skin disorders per Investigator's judgement.
  • Local ablative (eg, cauterization) or surgical procedures on nasal telangiectases <6 weeks before the Screening Visit.
  • Use of drugs with anti-angiogenic properties, including, but not limited to, bevacizumab, pazopanib, thalidomide, lenalidomide, pomalidomide, tacrolimus, sirolimus, or selective estrogen response modulators (tamoxifen, raloxifene, or bazedoxifene) < 6 weeks before the Screening Visit.
  • Use of oral tranexamic or epsilon-aminocaproic acid unless they are on a stable dose for at least 4 weeks before the Screening Visit, which will need to be continued during the entire duration of the double-blind Treatment Period.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Double blind
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07743671 · HHT-301 · 2025-524490-17-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗