Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE (OPuS-1)

August 14, 2014 updated by: BioCryst Pharmaceuticals

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

24

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: BCX4161
400 mg TID for 28 days
Drug: BCX4161
Placebo Comparator: Placebo
TID for 28 days
Drug: Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of acute angioedema attacks
Time Frame: Over 28 days of treatment
Over 28 days of treatment

Secondary Outcome Measures

Outcome Measure
Time Frame
Incidence and severity of adverse events and laboratory abnormalities
Time Frame: Over 28 days of treatment
Over 28 days of treatment
Number of attack free days
Time Frame: Over 28 days of treatment
Over 28 days of treatment
Angioedema Quality of Life scores
Time Frame: Over 28 days of treatment
Over 28 days of treatment
Angioedema activity scores
Time Frame: Over 28 days of treatment
Over 28 days of treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioCryst Pharmaceuticals

Investigators

  • Principal Investigator: Marcus Maurer, MD, Charite University, Berlin, Germany

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2013

Primary Completion (Actual)

May 1, 2014

Study Completion (Actual)

May 1, 2014

Study Registration Dates

First Submitted

November 5, 2013

First Submitted That Met QC Criteria

November 14, 2013

First Posted (Estimate)

November 15, 2013

Study Record Updates

Last Update Posted (Estimate)

August 18, 2014

Last Update Submitted That Met QC Criteria

August 14, 2014

Last Verified

August 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BCX4161-203

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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