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

NCT ID: NCT02303626

Last Updated: 2025-11-17

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

110 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-12-17

Study Completion Date

2016-01-31

Brief Summary

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This study will evaluate the safety and efficacy of an oral treatment, BCX4161, in preventing acute attacks in participants with hereditary angioedema (HAE). Eligible participants 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.

Detailed Description

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Conditions

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Hereditary Angioedema HAE

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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BCX4161 300 mg three times daily

Three BCX4161 capsules (100 mg) and two placebo capsules to be taken three times daily by mouth

Group Type EXPERIMENTAL

BCX4161

Intervention Type DRUG

Placebo

Intervention Type DRUG

BCX4161 500 mg three times daily

Five BCX4161 capsules (100 mg) to be taken three times daily by mouth

Group Type EXPERIMENTAL

BCX4161

Intervention Type DRUG

Placebo three times daily

Five placebo capsules to be taken three times daily by mouth

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Interventions

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BCX4161

Intervention Type DRUG

Placebo

Intervention Type DRUG

Eligibility Criteria

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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, participants 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

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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BioCryst Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Marc Riedl, MD MS

Role: PRINCIPAL_INVESTIGATOR

UCSD

Locations

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Birmingham, Alabama, United States

Site Status

Scottsdale, Arizona, United States

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Granada Hills, California, United States

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San Diego, California, United States

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San Diego, California, United States

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Walnut Creek, California, United States

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Colorado Springs, Colorado, United States

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Shreveport, Louisiana, United States

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Chevy Chase, Maryland, United States

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Boston, Massachusetts, United States

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Plymouth, Minnesota, United States

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St Louis, Missouri, United States

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New York, New York, United States

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Charlotte, North Carolina, United States

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Cincinnati, Ohio, United States

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Oklahoma City, Oklahoma, United States

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Tulsa, Oklahoma, United States

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Lake Oswego, Oregon, United States

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Hershey, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Dallas, Texas, United States

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Galveston, Texas, United States

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Fairfax, Virginia, United States

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Spokane, Washington, United States

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Tacoma, Washington, United States

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Leuven, , Belgium

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Ottawa, , Canada

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Grenoble, , France

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Lille, , France

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Paris, , France

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Berlin, , Germany

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Frankfurt, , Germany

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Mörfelden-Walldorf, , Germany

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Budapest, , Hungary

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Milan, , Italy

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Birmingham, , United Kingdom

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Bristol, , United Kingdom

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Cardiff, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

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Countries

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United States Belgium Canada France Germany Hungary Italy United Kingdom

References

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Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.

Reference Type DERIVED
PMID: 36326435 (View on PubMed)

Other Identifiers

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BCX4161-301

Identifier Type: -

Identifier Source: org_study_id

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