12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks

Study Overview
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Brief Summary
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This study will evaluate the safety and efficacy of an oral treatment, BCX4161, in preventing acute attacks in subjects with hereditary angioedema (HAE). Eligible subjects will be randomized to receive one of two doses of BCX4161 or placebo for 12 weeks. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical outcomes, and the safety and tolerability of each dose of BCX4161 compared to placebo.

Official Title
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OPuS-2: A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX4161 for 12 Weeks as an Oral Prophylaxis Treatment for Attacks of Hereditary Angioedema

Conditions
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Hereditary Angioedema, HAE

Intervention / Treatment
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* Drug: BCX4161
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: A clinical diagnosis of HAE type I or II Documented HAE attacks within a defined calendar period; in the absence of documented HAE attacks, subjects will be required to enter a run-in period to document attacks Access to acute attack medications Sexually active women of child-bearing potential and sexually active men must utilize highly effective contraception Key Exclusion Criteria: Women who are pregnant or breast-feeding Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study Use of C1INH or tranexamic acid for prophylaxis of HAE attacks Current participation in any other investigational drug study or within the last 30 days History of or current alcohol or drug abuse Infection with hepatitis B, hepatitis C or HIV

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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BCX4161 300 mg three times daily<br>Three BCX4161 capsules (100 mg) and two placebo capsules to be taken three times daily by mouth | Drug: BCX4161<br> <br> Drug: Placebo<br> <br> |
| Experimental: BCX4161 500 mg three times daily<br>Five BCX4161 capsules (100 mg) to be taken three times daily by mouth | Drug: BCX4161<br> <br> |
| Placebo Comparator: Placebo three times daily<br>Five placebo capsules to be taken three times daily by mouth | Drug: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The mean acute angioedema attack rate |  | 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of attack-free days |  | 12 weeks | 
| Number of subjects who are attack-free |  | 12 weeks | 
| Disease activity, as measured by the 84-day Angioedema Activity Score |  | 12 weeks | 
| Quality of Life, as measured by the Angioedema Quality of Life Questionnaire |  | 12 weeks | 
| Incidence and severity of adverse events and laboratory abnormalities |  | 12 weeks | 
| Quality of Life, as measured by the EuroQoL five-dimensional, 5-level Questionnaire |  | 12 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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BCX4161, Prophylaxis, Prevention, HAE, Hereditary Angioedema