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

Hereditary Angioedema Clinical Trial

— OPuS-1  

Official title:

A Phase 2a Double-Blind Placebo-Controlled 2-Period Crossover Study to Evaluate the Safety and Efficacy of BCX4161 as a Prophylactic Treatment to Reduce the Frequency of Attacks in Subjects With Hereditary Angioedema

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01984788
• Other study ID #: BCX4161-203
• Status: Completed
• Phase: Phase 2
• First received: November 5, 2013
• Last updated: August 14, 2014
• Start date: November 2013
• Est. completion date: May 2014

Study information

• Verified date: August 2014
• Source: BioCryst Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Bundesinstitut fur Arzneimittel und Medizinprodukte (BfArM)
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: May 2014
• Est. primary completion date: May 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

Key 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 Exclusion Criteria:

• Concurrent use of defined treatments for prophylaxis

• Pregnancy or breast-feeding

• Clinically significant medical condition, laboratory abnormality or medical history

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Prevention

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema

Intervention

Drug:
• BCX4161

• Placebo

Locations

Country	Name	City	State
Germany	Dr Marcus Maurer	Berlin
Germany	Dr Emel Aygoren-Pursun	Frankfurt
Germany	Dr Petra Staubach	Mainz
Germany	Dr Inmaculada Martinez-Saguer	Morfelden-Walldorf
Germany	Dr Murat Bas	Munchen
United Kingdom	Dr Hilary Longhurst	London

Sponsors (1)

Lead Sponsor	Collaborator
BioCryst Pharmaceuticals

Countries where clinical trial is conducted

Germany,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of acute angioedema attacks		Over 28 days of treatment	No
Secondary	Incidence and severity of adverse events and laboratory abnormalities		Over 28 days of treatment	Yes
Secondary	Number of attack free days		Over 28 days of treatment	No
Secondary	Angioedema Quality of Life scores		Over 28 days of treatment	No
Secondary	Angioedema activity scores		Over 28 days of treatment	No