Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema

Study Overview
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Brief Summary
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This is an open-label study designed to evaluate the long-term safety of prophylactic avoralstat (500 mg three times daily) when given to approximately 150 patients with hereditary angioedema (HAE) for a duration of up to 72 weeks. The study will also evaluate the long-term efficacy and impact on quality of life of avoralstat prophylactic treatment.

Official Title
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OPuS-4: An Open-label Study to Evaluate the Long-term Safety of Avoralstat in Subjects With Hereditary Angioedema

Conditions
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Hereditary Angioedema, HAE

Intervention / Treatment
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* Drug: avoralstat


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males and non-pregnant, non-lactating females age ≥ 18 years. Provide written, informed consent. Prior completion of an avoralstat therapeutic study OR a confirmed diagnosis of HAE Type 1 or HAE Type 2. Access to appropriate medication for the treatment of acute HAE attacks. Adequate contraception. Exclusion Criteria: Females who are pregnant or breast feeding. Clinically significant medical condition or medical history. Abnormal screening ECG, laboratory or urinalysis finding that is clinically significant. Investigational drug exposure within 30 days (except avoralstat). History of or current alcohol or drug abuse. HIV or active HBV or HCV infection.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: avoralstat<br>Five avoralstat capsules (100 mg) to be taken three times daily by mouth | Drug: avoralstat<br> <br> * Other names: BCX4161;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of subjects who discontinue due to a treatment-emergent adverse event, who experience a treatment-emergent serious AE, who experience a Grade 3 or 4 AE and who experience Grade 3 or 4 laboratory abnormalities |  | Up to 72 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Angioedema attack rate (subject-reported HAE attacks normalized for time on study) |  | Up to 72 weeks | 
| Durability in response (assessed as subject-reported HAE attacks) |  | change over time through 72 weeks | 
| Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy |  | Up to 72 weeks | 
| Quality of life as determined by the EQ-5D-5L |  | Up to 72 weeks | 
| Quality of life as determined by the Angioedema Quality of Life Questionnaire |  | Up to 72 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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BCX4161, avoralstat, HAE, hereditary angioedema, prophylaxis