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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. Identifier: NCT02303626
Recruitment Status : Completed
First Posted : December 1, 2014
Last Update Posted : February 17, 2016
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

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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

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

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 identifier (NCT number): NCT02303626

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Sponsors and Collaborators
BioCryst Pharmaceuticals
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Principal Investigator: Marc Riedl, MD MS UCSD

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Responsible Party: BioCryst Pharmaceuticals Identifier: NCT02303626     History of Changes
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:
Hereditary Angioedema
Additional relevant MeSH terms:
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Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Immune System Diseases
Genetic Diseases, Inborn