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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
240
Engasertib will be administered as oral capsules.
Placebo will be administered as oral capsules.
The University of Chicago Medical Center (UCMC)
Chicago, Illinois, United States
RECRUITINGInnovative Hematology, Inc.,
Indianapolis, Indiana, United States
RECRUITINGMayo Clinic
Rochester, Minnesota, United States
RECRUITINGTotal Number of Epistaxis Events Through Week 28
Time frame: Day 1 through Week 28
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
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Washington University School of Medicine
St Louis, Missouri, United States
RECRUITING