Hereditary Angioedema
Conditions
Brief summary
Time to onset of symptom relief, defined as Patient Global Impression of Change (PGI-C) rating of at least “a little better” for 2 consecutive timepoints within 12 hours post-treatment.
Detailed description
1. Proportion of study drug-treated attacks achieving PGI-C rating of at least “a little better” at 4 hours post-treatment, 2. Time to substantial symptom relief, defined as achieving PGI-C rating of at least “better” for 2 consecutive timepoints within 12 hours post-treatment., 3. Time to substantial symptom relief by Patient Global Impression of Severity (PGI-S), defined as achieving ≥1 point reduction in PGI-S from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment., 4. Time to complete symptom resolution, defined as achieving PGI-S rating of “none” within 48 hours post-treatment., 5. Time to End of Progression (EoP) in attack symptoms within 12 hours, with EoP time defined as the earliest post-treatment timepoint after which all subsequent PGI-C ratings are stable or improved., 6. Proportion of study drug-treated attacks requiring rescue medication within 24 hours post treatment., 7. Proportion of attacks achieving symptom resolution, defined as achieving PGI-S rating of “none” with one dose of study drug at 24 hours post-treatment., 8. Time to substantial symptom relief by Angioedema SyMptom Rating scAle (AMRA), defined as a ≥50% reduction in AMRA composite score from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment., 9. Time to almost complete or complete symptom relief by AMRA, defined as all item scores in AMRA having a value ≤10 for 2 consecutive timepoints within 24 hours post-treatment., 10. Proportion of study drug-treated attacks reaching almost complete or complete symptom relief by AMRA, defined as all item scores in AMRA having a value ≤10 at 24 hours post treatment., 11. Time to EoP in attack symptoms within 12 hours, with EoP time defined as the earliest post-treatment timepoint after which every individual AMRA item is stable or improved at all subsequent timepoints
Interventions
DRUGICATIBANT
DRUGPlacebo matches Deucritibant soft capsules with exception of active substance.
Sponsors
Pharvaris Netherlands B.V.
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time to onset of symptom relief, defined as Patient Global Impression of Change (PGI-C) rating of at least “a little better” for 2 consecutive timepoints within 12 hours post-treatment. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Proportion of study drug-treated attacks achieving PGI-C rating of at least “a little better” at 4 hours post-treatment, 2. Time to substantial symptom relief, defined as achieving PGI-C rating of at least “better” for 2 consecutive timepoints within 12 hours post-treatment., 3. Time to substantial symptom relief by Patient Global Impression of Severity (PGI-S), defined as achieving ≥1 point reduction in PGI-S from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment., 4. Time to complete symptom resolution, defined as achieving PGI-S rating of “none” within 48 hours post-treatment., 5. Time to End of Progression (EoP) in attack symptoms within 12 hours, with EoP time defined as the earliest post-treatment timepoint after which all subsequent PGI-C ratings are stable or improved., 6. Proportion of study drug-treated attacks requiring rescue medication within 24 hours post treatment., 7. Proportion of attacks achieving symptom resolution, defined as achieving PGI | — |
Countries
Austria, Bulgaria, Czechia, France, Germany, Hungary, Ireland, Italy, Netherlands, Poland, Romania, Slovakia, Spain, Sweden
Outcome results
None listed