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

BioCryst

Status and phase: Completed; Phase 3; Phase 2

Conditions: HAE; Hereditary Angioedema

Treatments: Drug: BCX4161; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02303626

BCX4161-301

Details and patient eligibility

About

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.

Enrollment: 110 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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, 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

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

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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110 participants in 3 patient groups, including a placebo group

BCX4161 300 mg three times daily

Experimental group

Description:

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

Treatment:

Drug: Placebo

Drug: BCX4161

BCX4161 500 mg three times daily

Experimental group

Description:

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

Treatment:

Drug: BCX4161

Placebo three times daily

Placebo Comparator group

Description:

Five placebo capsules to be taken three times daily by mouth

Treatment:

Drug: Placebo