12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks (OPuS-2)

February 15, 2016 updated by: BioCryst Pharmaceuticals

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

110

Phase

  • Phase 2
  • Phase 3

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

  • Belgium
      • Leuven, Belgium
  • Canada
      • Ottawa, Canada
  • France
      • Grenoble, France
      • Lille, France
      • Paris, France
  • Germany
      • Berlin, Germany
      • Frankfurt, Germany
      • Morfelden-Walldorf, Germany
  • Hungary
      • Budapest, Hungary
  • Italy
      • Milano, Italy
  • United Kingdom
      • Birmingham, United Kingdom
      • Bristol, United Kingdom
      • Cardiff, United Kingdom
      • London, United Kingdom
      • Manchester, United Kingdom
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35209
    • Arizona
      • Scottsdale, Arizona, United States, 85251
    • California
      • Granada Hills, California, United States, 91344
      • San Diego, California, United States, 92093
      • San Diego, California, United States, 92112
      • Walnut Creek, California, United States, 94598
    • Colorado
      • Colorado Springs, Colorado, United States, 80907
    • Louisiana
      • Shreveport, Louisiana, United States, 71106
    • Maryland
      • Chevy Chase, Maryland, United States, 20815
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
    • Minnesota
      • Plymouth, Minnesota, United States, 55446
    • Missouri
      • St Louis, Missouri, United States, 63141
    • New York
      • New York, New York, United States, 10029
    • North Carolina
      • Charlotte, North Carolina, United States, 28277
    • Ohio
      • Cincinnati, Ohio, United States, 45267
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73131
      • Tulsa, Oklahoma, United States, 74133
    • Oregon
      • Lake Oswego, Oregon, United States, 97035
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
      • Pittsburg, Pennsylvania, United States, 15241
    • Texas
      • Dallas, Texas, United States, 75231
      • Galveston, Texas, United States, 77555
    • Virginia
      • Fairfax, Virginia, United States, 63141
    • Washington
      • Spokane, Washington, United States, 99204
      • Tacoma, Washington, United States, 98405

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 and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Key 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 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

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: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
The mean acute angioedema attack rate
Time Frame: 12 weeks
12 weeks

Secondary Outcome Measures

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

Collaborators and Investigators

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

Sponsor

BioCryst Pharmaceuticals

Investigators

  • Principal Investigator: Marc Riedl, MD MS, UCSD

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, 2014

Primary Completion (Actual)

January 1, 2016

Study Completion (Actual)

January 1, 2016

Study Registration Dates

First Submitted

November 24, 2014

First Submitted That Met QC Criteria

November 25, 2014

First Posted (Estimate)

December 1, 2014

Study Record Updates

Last Update Posted (Estimate)

February 17, 2016

Last Update Submitted That Met QC Criteria

February 15, 2016

Last Verified

February 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BCX4161-301

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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