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

Phase 2

Completed

• Conditions: Hereditary Angioedema; HAE
• Interventions: Drug: BCX4161; Drug: Placebo

• Registration Number: NCT02303626
• Lead Sponsor: BioCryst Pharmaceuticals

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-11
• Study First Submitted: 2014-11-24
• Study First Submitted QC: 2014-11-25
• Study First Posted: 2014-12-01
• Primary Completion: 2016-01
• Study Completion: 2016-01
• Status Verified: 2016-02
• Last Update Submitted: 2016-02-15
• Last Update Posted: 2016-02-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 110

Inclusion Criteria

1. A clinical diagnosis of HAE type I or II
2. 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
3. Access to acute attack medications
4. Sexually active women of child-bearing potential and sexually active men must utilize highly effective contraception

Key

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Exclusion Criteria

1. Women who are pregnant or breast-feeding
2. Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study
3. Use of C1INH or tranexamic acid for prophylaxis of HAE attacks
4. Current participation in any other investigational drug study or within the last 30 days
5. History of or current alcohol or drug abuse
6. Infection with hepatitis B, hepatitis C or HIV

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
BCX4161 300 mg three times daily	Placebo	Three BCX4161 capsules (100 mg) and two placebo capsules to be taken three times daily by mouth
Placebo three times daily	Placebo	Five placebo capsules to be taken three times daily by mouth
BCX4161 300 mg three times daily	BCX4161	Three BCX4161 capsules (100 mg) and two placebo capsules to be taken three times daily by mouth
BCX4161 500 mg three times daily	BCX4161	Five BCX4161 capsules (100 mg) to be taken three times daily by mouth

Primary Outcome Measures

Name	Time	Method
The mean acute angioedema attack rate	12 weeks

Secondary Outcome Measures

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