Hereditary Hemorrhagic Telangiectasia (HHT)
Conditions
Brief summary
Total number of epistaxis events through Week 28 as assessed by the HHT eDiary
Detailed description
Absolute change from baseline in total duration of epistaxis at Week 28 as assessed by the HHTeDiary, Absolute change from baseline in ESS at Week 28, based on symptoms as they have occurred over the last 4 weeks as assessed by the ESS questionnaire, Absolute change from baseline in the NOSE HHT score at Week 28, based on symptoms as they have occurred over the past 2 weeks as assessed by the NOSE HHT questionnaire, Absolute change in red blood cell (RBC) unit equivalents (RUEs) received at Week 28
Interventions
DRUGEngasertib
DRUGEngasertib Placebo
Sponsors
Vaderis Therapeutics AG
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Total number of epistaxis events through Week 28 as assessed by the HHT eDiary | — |
Secondary
| Measure | Time frame |
|---|---|
| Absolute change from baseline in total duration of epistaxis at Week 28 as assessed by the HHTeDiary, Absolute change from baseline in ESS at Week 28, based on symptoms as they have occurred over the last 4 weeks as assessed by the ESS questionnaire, Absolute change from baseline in the NOSE HHT score at Week 28, based on symptoms as they have occurred over the past 2 weeks as assessed by the NOSE HHT questionnaire, Absolute change in red blood cell (RBC) unit equivalents (RUEs) received at Week 28 | — |
Outcome results
None listed