Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02303626
Recruitment Status : Completed
First Posted : December 1, 2014
Last Update Posted : February 17, 2016
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema HAE Drug: BCX4161 Drug: Placebo Phase 2 Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 110 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Official Title: 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
Study Start Date : November 2014
Actual Primary Completion Date : January 2016
Actual Study Completion Date : January 2016

Resource links provided by the National Library of Medicine


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: BCX4161
Drug: Placebo
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



Primary Outcome Measures :
  1. The mean acute angioedema attack rate [ Time Frame: 12 weeks ]

Secondary Outcome Measures :
  1. Number of attack-free days [ Time Frame: 12 weeks ]
  2. Number of subjects who are attack-free [ Time Frame: 12 weeks ]
  3. Disease activity, as measured by the 84-day Angioedema Activity Score [ Time Frame: 12 weeks ]
  4. Quality of Life, as measured by the Angioedema Quality of Life Questionnaire [ Time Frame: 12 weeks ]
  5. Incidence and severity of adverse events and laboratory abnormalities [ Time Frame: 12 weeks ]
  6. Quality of Life, as measured by the EuroQoL five-dimensional, 5-level Questionnaire [ Time Frame: 12 weeks ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02303626


Locations
Layout table for location information
United States, Alabama
Birmingham, Alabama, United States, 35209
United States, Arizona
Scottsdale, Arizona, United States, 85251
United States, 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
United States, Colorado
Colorado Springs, Colorado, United States, 80907
United States, Louisiana
Shreveport, Louisiana, United States, 71106
United States, Maryland
Chevy Chase, Maryland, United States, 20815
United States, Massachusetts
Boston, Massachusetts, United States, 02114
United States, Minnesota
Plymouth, Minnesota, United States, 55446
United States, Missouri
St Louis, Missouri, United States, 63141
United States, New York
New York, New York, United States, 10029
United States, North Carolina
Charlotte, North Carolina, United States, 28277
United States, Ohio
Cincinnati, Ohio, United States, 45267
United States, Oklahoma
Oklahoma City, Oklahoma, United States, 73131
Tulsa, Oklahoma, United States, 74133
United States, Oregon
Lake Oswego, Oregon, United States, 97035
United States, Pennsylvania
Hershey, Pennsylvania, United States, 17033
Pittsburg, Pennsylvania, United States, 15241
United States, Texas
Dallas, Texas, United States, 75231
Galveston, Texas, United States, 77555
United States, Virginia
Fairfax, Virginia, United States, 63141
United States, Washington
Spokane, Washington, United States, 99204
Tacoma, Washington, United States, 98405
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
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
Layout table for investigator information
Principal Investigator: Marc Riedl, MD MS UCSD

Layout table for additonal information
Responsible Party: BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02303626    
Other Study ID Numbers: BCX4161-301
First Posted: December 1, 2014    Key Record Dates
Last Update Posted: February 17, 2016
Last Verified: February 2016
Keywords provided by BioCryst Pharmaceuticals:
BCX4161
Prophylaxis
Prevention
HAE
Hereditary Angioedema
Additional relevant MeSH terms:
Layout table for MeSH terms
Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn