Hereditary Angioedema Clinical Trial
— HAE CHAPTER-1Official title:
A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Due to C1-Inhibitor Deficiency (Type I or Type II)
Verified date | February 2024 |
Source | Pharvaris Netherlands B.V. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This study evaluates the safety and efficacy of PHA-022121 administered orally for prophylaxis against angioedema attacks in patients with hereditary angioedema (HAE). The study consists of 2 parts, with patients completing participation in Part 1 prior to initiation of treatment in Part 2. Part 1 of the study has 3 parallel arms and approximately 30 patients will be equally randomized to one of two dose regimens of PHA-022121 or matching placebo. Patients will continue to the single open-label arm in Part 2 of the study after completion of Part 1. The screening period is up to 8 weeks and the treatment periods are 12 weeks (Part 1) and 30 months (Part 2) in duration.
Status | Active, not recruiting |
Enrollment | 34 |
Est. completion date | December 2026 |
Est. primary completion date | October 2, 2023 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 75 Years |
Eligibility | Inclusion Criteria: - Signed and dated informed consent form - Diagnosis of HAE type I or II - Documented history of at least 3 HAE attacks within the last 3 consecutive months prior to screening, or a minimum of 2 HAE attacks during the screening period - Reliable access and experience to use standard of care acute attack medications Exclusion Criteria: - Pregnancy or breast-feeding - Clinically significant abnormal electrocardiogram - Any other systemic disease or significant disease or disorder that would interfere with the patient's safety or ability to participate in the study - Use of C1-esterase inhibitor, oral kallikrein inhibitors, attenuated androgens, anti-fibrinolytics, or monoclonal HAE therapy within a defined period prior to enrolment - Abnormal hepatic function - Abnormal renal function - History of alcohol or drug abuse within defined period, or current evidence of substance dependence or abuse - Participation in any other investigational drug study within defined period |
Country | Name | City | State |
---|---|---|---|
Austria | Study Site | Wien | |
Bulgaria | Study site | Sofia | |
Canada | Study site | Montréal | Quebec |
Canada | Study site | Ottawa | Ontario |
Germany | Study site | Berlin | |
Germany | Study site | Frankfurt | |
Ireland | Study site | Dublin | |
Italy | Study site | Milan | |
Italy | Study site | Padua | PD |
Italy | Study site | Palermo | |
Poland | Study site | Kraków | |
United Kingdom | Study site | Brighton | England |
United Kingdom | Study site | Bristol | England |
United Kingdom | Study site | Cambridge | England |
United Kingdom | Study site | London | England |
United Kingdom | Study site | Southampton | England |
United States | Study site | Birmingham | Alabama |
United States | Study site | Paradise Valley | Arizona |
United States | Study site | Saint Louis | Missouri |
Lead Sponsor | Collaborator |
---|---|
Pharvaris Netherlands B.V. |
United States, Austria, Bulgaria, Canada, Germany, Ireland, Italy, Poland, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of investigator-confirmed HAE attacks | Day 0 to Day 84 | ||
Secondary | Number of investigator-confirmed moderate or severe HAE attacks during the treatment period | Day 0 to Day 84 | ||
Secondary | Number of investigator-confirmed HAE attacks requiring acute treatment during the treatment period | Day 0 to Day 84 | ||
Secondary | Number of patients achieving reduction in attack rate during the treatment period relative to baseline | Day 0 to Day 84 | ||
Secondary | Number of patients that are attack-free during the treatment period | Day 0 to Day 84 | ||
Secondary | Number and proportion of days with angioedema symptoms during the treatment period | Day 0 to Day 84 | ||
Secondary | Time to first investigator-confirmed HAE attack in the treatment period | Day 0 to Day 84 | ||
Secondary | Number of investigator-confirmed HAE attacks resulting in a visit to the emergency department or an admission to hospital | Day 0 to Day 84 | ||
Secondary | Number of investigator-confirmed angioedema attacks during the treatment period in Part 2. | Day 84 to Day 938 | ||
Secondary | Number of investigator-confirmed moderate or severe angioedema attacks during the treatment period in Part 2. | Day 84 to Day 938 | ||
Secondary | Number of investigator-confirmed angioedema attacks requiring acute treatment during the treatment period in Part 2. | Day 84 to Day 938 | ||
Secondary | Incidence of HAE attacks during the treatment period in Part 2 (attack rate trend over time). | Day 84 to Day 938 | ||
Secondary | Number and proportion of days with angioedema symptoms during the treatment period in Part 2. | Day 84 to Day 938 |
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