Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE

Phase 2

Completed

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

• Registration Number: NCT01984788
• Lead Sponsor: BioCryst Pharmaceuticals

Brief Summary

The purpose of this study is to determine whether BCX4161 given as a daily oral prophylactic treatment is safe and effective in reducing the number of acute attacks in patients with hereditary angioedema.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-11
• Study First Submitted: 2013-11-05
• Study First Submitted QC: 2013-11-14
• Study First Posted: 2013-11-15
• Primary Completion: 2014-05
• Study Completion: 2014-05
• Status Verified: 2014-08
• Disposition First Submitted: 2014-08-14
• Disposition First Submitted QC: 2014-08-14
• Last Update Submitted: 2014-08-14
• Disposition First Posted: 2014-08-18
• Last Update Posted: 2014-08-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

• Written informed consent
• Clinical and laboratory diagnosis of Hereditary Angioedema Type I or II
• An average angioedema attack frequency of 1/week
• Acceptable birth control measures

Key

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Exclusion Criteria

• Concurrent use of defined treatments for prophylaxis
• Pregnancy or breast-feeding
• Clinically significant medical condition, laboratory abnormality or medical history

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	TID for 28 days
BCX4161	BCX4161	400 mg TID for 28 days

Primary Outcome Measures

Name	Time	Method
Number of acute angioedema attacks	Over 28 days of treatment

Secondary Outcome Measures

Name	Time	Method
Number of attack free days	Over 28 days of treatment
Incidence and severity of adverse events and laboratory abnormalities	Over 28 days of treatment
Angioedema activity scores	Over 28 days of treatment
Angioedema Quality of Life scores	Over 28 days of treatment

Trial Locations

Locations (6)

Dr Inmaculada Martinez-Saguer; 🇩🇪 Morfelden-Walldorf, Germany

Dr Petra Staubach; 🇩🇪 Mainz, Germany

Dr Marcus Maurer; 🇩🇪 Berlin, Germany

Dr Hilary Longhurst; 🇬🇧 London, United Kingdom

Dr Murat Bas; 🇩🇪 Munchen, Germany

Dr Emel Aygoren-Pursun; 🇩🇪 Frankfurt, Germany