Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE (OPuS-1)

BioCryst

Status and phase: Completed; Phase 2

Conditions: Hereditary Angioedema

Treatments: Drug: BCX4161; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT01984788

BCX4161-203

Details and patient eligibility

About

The purpose of this study is to determine whether BCX4161 given as a daily oral prophylactic treatment is safe and effective in reducing the number of acute attacks in patients with hereditary angioedema.

Enrollment: 24 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Written informed consent
• Clinical and laboratory diagnosis of Hereditary Angioedema Type I or II
• An average angioedema attack frequency of 1/week
• Acceptable birth control measures

Key Exclusion Criteria:

• Concurrent use of defined treatments for prophylaxis
• Pregnancy or breast-feeding
• Clinically significant medical condition, laboratory abnormality or medical history

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Double Blind

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24 participants in 2 patient groups, including a placebo group

BCX4161

Active Comparator group

Description:

400 mg TID for 28 days

Treatment:

Drug: BCX4161

Placebo

Placebo Comparator group

Description:

TID for 28 days

Treatment:

Drug: Placebo