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This is a Phase 3, multicenter, long-term, open-label study to evaluate the safety and efficacy of once-daily orally administered deucrictibant extended-release tablet for prophylaxis to prevent angioedema attacks in participants aged ≥12 years with Hereditary Angioedema
The study consists of a Screening Period during which eligibility is confirmed (only for participants not rolling over within 28 days from a previous deucrictibant prophylactic study), a Treatment Period in which participants will receive open-label deucrictibant extended-release tablet once daily for approximately 130 weeks, followed by an End of Study visit after maximum 4 weeks. Participants will undergo regular safety (e.g. lab draws) and efficacy assessments, will complete an electronic diary daily, and also complete questionnaires at predefined timepoints during the study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Deucrictibant | Experimental | Deucrictibant |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deucrictibant | Drug | Deucrictibant extended-release tablet for once daily oral use |
|
| Measure | Description | Time Frame |
|---|---|---|
| Treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation | 130 weeks | |
| Change in heart rate | 130 weeks | |
| Change in blood pressure | 130 weeks | |
| Change in body temperature | 130 weeks | |
| Change in clinical laboratory tests from baseline | Hematology, blood chemistry, and urinalysis. Descriptive in nature, no formal statistical hypothesis testing will be performed. | 130 weeks |
| Change in electrocardiograms (ECGs) from baseline | Digital triplicate 12-lead ECG. Descriptive in nature, no formal statistical hypothesis testing will be performed. | 130 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Time-normalized number of Investigator-confirmed HAE attacks during the Treatment Period | 130 weeks | |
| Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the Treatment Period | 130 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Pharvaris Clinical Team | Contact | 0031 712-036-410 | clinicaltrials@pharvaris.com |
| Name | Affiliation | Role |
|---|---|---|
| Study Director, Pharvaris | Pharvaris Netherlands B.V. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Study Site | Recruiting | Birmingham | Alabama | 35209 | United States | |
| Study Site |
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| Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during Treatment Period | 130 weeks |
| Time-normalized number of Investigator-confirmed severe HAE attacks during the Treatment Period | 130 weeks |
| Proportion of time without angioedema symptoms during the Treatment Period | 130 weeks |
| Patient reported outcome: Angioedema Quality of Life (AE-QoL) questionnaire | The AE-QoL is a short 17-item questionnaire designed to retrospectively assess HRQoL, with a recall period of 4 weeks. Its results can be displayed as a total score or as 4 domain scores. The scores range from 0 to 100, after linear transformation of raw values, with higher scores indicating higher HRQoL impairment. | 130 weeks |
| Patient reported outcome: Patient Global Assessment of Change (PGA-Change) | PGA-Change assesses on a 5-point scale how the participant's QoL has been impacted by HAE since start taking the study drug. | 130 weeks |
| Patient reported outcome: Angioedema Control Test 4-week version (AECT-4wk) | AECT-4wk measures disease control retrospectively, it comprises 4 questions over a 4-week recall period. Scores for the responses in the AECT range from 0 to 16, with higher scores indicating better disease control (≤ 9 poorly controlled; ≥ 10 well controlled). | 130 weeks |
| Patient reported outcome: Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP) | WPAI-SHP is a questionnaire assessing how a health condition impacts a person's ability to work and do regular activities and it includes 4 domains. Scores indicate the percentage of time the patient missed work or was less productive owing to HAE-related complications. | 130 weeks |
| Patient reported outcome: Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9) | TSQM-9 is a 9-item questionnaire evaluating patient treatment satisfaction and it includes 3 domains. Scoring is by domain and each domain score is computed by summing the individual TSQM items in each domain and then transforming the composite score into a value ranging from 0 to 100, with higher scores indicating higher satisfaction. | 130 weeks |
| Pharmacokinetics [PK]: Pre-dose plasma concentration of deucrictibant and deucrictibant metabolites (Ctrough) | 130 weeks |
| Pharmacokinetics [PK]: Urine concentration of deucrictibant and deucrictibant metabolites | 130 weeks |
| Recruiting |
| Scottsdale |
| Arizona |
| 85251 |
| United States |
| Study Site | Recruiting | Little Rock | Arkansas | 72205 | United States |
| Study Site | Recruiting | San Diego | California | 92122 | United States |
| Study Site | Recruiting | Santa Monica | California | 90404 | United States |
| Study Site | Recruiting | Walnut Creek | California | 94598 | United States |
| Study Site | Recruiting | Wheaton | Maryland | 20902 | United States |
| Study Site | Recruiting | St Louis | Missouri | 63141 | United States |
| Study Site | Recruiting | Las Vegas | Nevada | 89128-0450 | United States |
| Study Site | Recruiting | Hershey | Pennsylvania | 17033 | United States |
| Study Site | Recruiting | Dallas | Texas | 75231 | United States |
| Study Site | Recruiting | Milwaukee | Wisconsin | 53226-3067 | United States |
| Study Site | Recruiting | Corrientes | Argentina |
| Study Site | Recruiting | Pilar | Argentina |
| Study Site | Recruiting | Salta | Argentina |
| Study Site | Recruiting | Melbourne | 3052 | Australia |
| Study Site | Recruiting | Melbourne | Australia |
| Study Site | Recruiting | Perth | Australia |
| Study Site | Recruiting | Sydney | Australia |
| Study Site | Recruiting | Vienna | Austria |
| Study Site | Recruiting | Ribeirão Preto | Brazil |
| Study Site | Recruiting | Salvador | Brazil |
| Study Site | Recruiting | Santo André | Brazil |
| Study Site | Recruiting | São Paulo | 01454010 | Brazil |
| Study Site | Recruiting | São Paulo | 14051-140 | Brazil |
| Study Site | Recruiting | Sofia | Bulgaria |
| Study Site | Recruiting | Edmonton | Canada |
| Study Site | Recruiting | Montreal | Canada |
| Study Site | Recruiting | Beijing | China |
| Study Site | Recruiting | Grenoble | France |
| Study Site | Recruiting | Berlin | Germany |
| Study Site | Recruiting | Frankfurt | Germany |
| Study Site | Recruiting | Munich | Germany |
| Study Site | Recruiting | Hong Kong | Hong Kong |
| Study Site | Recruiting | Budapest | Hungary |
| Study Site | Recruiting | Dublin | Ireland |
| Study Site | Recruiting | Padova | Italy |
| Study Site | Recruiting | Palermo | Italy |
| Study Site | Recruiting | Roma | Italy |
| Study Site | Recruiting | Hiroshima | Japan |
| Study Site | Recruiting | Kanagawa | Japan |
| Study Site | Recruiting | Osaka | Japan |
| Study Site | Recruiting | Tokyo | Japan |
| Study Site | Recruiting | Krakow | Poland |
| Study Site | Recruiting | Martin | Slovakia |
| Study Site | Recruiting | Cape Town | South Africa |
| Study Site | Recruiting | Daegu | South Korea |
| Study Site | Recruiting | Seoul | South Korea |
| Study Site | Recruiting | Barcelona | 08907 | Spain |
| Study Site | Recruiting | Ankara | Turkey (Türkiye) |
| Study Site | Recruiting | Istanbul | Turkey (Türkiye) |
| Study Site | Recruiting | Izmir | Turkey (Türkiye) |
| Study Site | Recruiting | Birmingham | B18 7QH | United Kingdom |
| Study Site | Recruiting | Birmingham | B9 5SS | United Kingdom |
| Study Site | Recruiting | Brighton | United Kingdom |
| Study Site | Recruiting | Bristol | United Kingdom |
| Study Site | Recruiting | Cambridge | United Kingdom |
| Study Site | Recruiting | London | E1 1FR | United Kingdom |
| Study Site | Recruiting | London | United Kingdom |
| Study Site | Recruiting | Oxford | United Kingdom |
| Study Site | Recruiting | Plymouth | United Kingdom |
| Study Site | Recruiting | Southampton | United Kingdom |
| ID | Term |
|---|---|
| D054179 | Angioedemas, Hereditary |
| ID | Term |
|---|---|
| D000799 | Angioedema |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D000081208 | Hereditary Complement Deficiency Diseases |
| D000081207 | Primary Immunodeficiency Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D014581 | Urticaria |
| D017445 | Skin Diseases, Vascular |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D007153 | Immunologic Deficiency Syndromes |
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