Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema (APeX-1)

BioCryst

Status and phase: Completed; Phase 2

Conditions: Hereditary Angioedema (HAE)

Treatments: Drug: BCX7353; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02870972

BCX7353-203

Details and patient eligibility

About

This 3-part study will evaluate the safety and efficacy of an oral treatment, BCX7353, in preventing angioedema attacks in subjects with hereditary angioedema (HAE). In Part 1 of the study, eligible subjects will be randomized to receive oral BCX7353 or placebo for 4 weeks. Assuming successful completion of Part 1, additional subjects will be randomized in Part 2 to one of 2 lower doses of BCX7353 or placebo. Part 3 will enroll additional subjects into one of three doses of BCX7353 or placebo. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical and pharmacologic outcomes, and the safety and tolerability of each dose of BCX7353 compared to placebo.

Enrollment: 75 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• A clinical diagnosis of HAE type I or II
• Documented HAE attacks within a defined calendar period
• Access to acute attack medications
• Sexually active women of child-bearing potential and sexually active men must utilize effective contraception

Key Exclusion Criteria:

• Women who are pregnant or breast-feeding
• Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study
• Use of C1INH, androgens or tranexamic acid for prophylaxis of HAE attacks
• History of or current alcohol or drug abuse
• Infection with hepatitis B, hepatitis C or HIV
• Participation in any other investigational drug study currently or within the last 30 days

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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75 participants in 5 patient groups, including a placebo group

Part 1: BCX7353 350 mg once daily

Experimental group

Description:

BCX7353 capsules, 350 mg dose administered once per day for 28 days

Treatment:

Drug: BCX7353

Parts 2 and 3: BCX7353 250 mg once daily

Experimental group

Description:

BCX7353 capsules, 250 mg dose administered once per day for 28 days

Treatment:

Drug: BCX7353

Parts 2 and 3: BCX7353 125 mg once daily

Experimental group

Description:

BCX7353 capsules, 125 mg dose administered once per day for 28 days

Treatment:

Drug: BCX7353

Parts 1, 2 and 3: Placebo

Placebo Comparator group

Description:

Placebo capsules, administered once per day for 28 days

Treatment:

Drug: Placebo

Part 3: BCX7353 62.5 mg once daily

Experimental group

Description:

BCX7353 capsules, 62.5 mg dose administered once per day for 28 days

Treatment:

Drug: BCX7353