Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE (APeX-2)

BioCryst

Status and phase: Completed; Phase 3

Conditions: HAE; Hereditary Angioedema

Treatments: Drug: Placebo oral capsule; Drug: BCX7353 capsules

Study type: Interventional

Funder types: Industry

Identifiers

NCT03485911

BCX7353-302

Details and patient eligibility

About

This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE.

Enrollment: 121 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• A clinical diagnosis of hereditary angioedema Type 1 or Type 2, defined as having a C1-INH functional level and a C4 level below the lower limit of the normal (LLN) reference range, as assessed during the Screening period.
• Subject weight of ≥ 40 kg
• Access to and ability to use one or more acute medications approved by the relevant competent authority for the treatment of acute attacks of HAE
• Subjects must be medically appropriate for on-demand treatment as the sole medicinal management for their HAE during the study.
• Subjects must have a specified number of investigator-confirmed attacks during the run-in period of a maximum of 56 days from the Screening visit.
• Acceptable effective contraception
• Written informed consent

Key Exclusion Criteria:

• Pregnancy or breast-feeding
• Any clinically significant medical condition or medical history that, in the opinion of the Investigator or Sponsor, would interfere with the subject's safety or ability to participate in the study
• Any laboratory parameter abnormality that, in the opinion of the Investigator, is clinically significant and relevant for this study
• Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
• Use of C1-INH within 14 days or use of androgens or tranexamic acid within 28 days prior to the Screening visit for prophylaxis of HAE attacks, or initiation of these drugs during the study
• Current participation in any other investigational drug study or received another investigational drug within 30 days of the Screening visit
• Prior enrollment in a BCX7353 study

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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121 participants in 3 patient groups, including a placebo group

BCX7353 110 mg once daily

Experimental group

Description:

BCX7353 administered as oral capsules once daily

Treatment:

Drug: BCX7353 capsules

BCX7353 150 mg once daily

Experimental group

Description:

BCX7353 administered as oral capsules once daily

Treatment:

Drug: BCX7353 capsules

Placebo

Placebo Comparator group

Description:

Matching placebo administered as oral capsules once daily

Treatment:

Drug: Placebo oral capsule