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

NCT07743671 | Not Yet Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: HHT-3012025-524490-17-00
• Study Type: interventional
• Target: 240
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Hereditary Hemorrhagic Telangiectasia

Keywords

Hereditary Hemorrhagic Telangiectasia; HHT; Engasertib; VAD044

Outcome Measures

Primary Outcomes (1)

• Total Number of Epistaxis Events Through Week 28

  Day 1 through Week 28

Secondary Outcomes (7)

• Absolute Change from Baseline in Total Duration of Epistaxis at Week 28

  Baseline and Week 28

• Absolute Change from Baseline in Epistaxis Severity Score (ESS) at Week 28

  Baseline and Week 28

• Absolute Change from Baseline in the Nasal Outcome Score for Epistaxis in HHT (NOSE HHT) Score at Week 28

  Baseline and Week 28

• Absolute Change in Red Blood Cell (RBC) Unit Equivalents (RUEs) Received at Week 28

  Baseline and Week 28

• Patient Global Impression of Change (PGIC) Nosebleeds Sub-score at Week 28

  Week 28

Study Arms (2)

Engasertib 40 mg

EXPERIMENTAL

Participants will receive engasertib as an oral capsule QD for 28 weeks.

Drug: Engasertib

Placebo

PLACEBO COMPARATOR

Participants will receive matching placebo as an oral capsule QD for 28 weeks.

Drug: Placebo

Interventions

Engasertib DRUG

Engasertib will be administered as oral capsules.

Also known as: VAD044

Engasertib 40 mg

Placebo DRUG

Placebo will be administered as oral capsules.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Vaderis Therapeutics AG: lead

MeSH Terms

Conditions

Telangiectasia, Hereditary Hemorrhagic

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Telangiectasis; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Vascular Malformations; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Vaderis Therapeutics AG

CONTACT

844-823-3747; info@vaderis.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 29, 2026
• First Posted: August 4, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2028
• Last Updated: August 4, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share