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

BioCryst Pharmaceuticals110 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

QUADRUPLE

Enrollment

110

Conditions

Hereditary Angioedema HAE

Interventions

BCX4161DRUG

PlaceboDRUG

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (40)

Outcomes

Primary Outcomes

The Mean Acute Angioedema Attack Rate

An angioedema attack was defined as swelling at any location reported by participant, which had no swelling earlier. Total number of confirmed attacks during the treatment period standardized to a weekly attack-rate to adjust for the total duration of treatment. The attack rate was derived for each participant by treatment period. The weekly attack rate was equal to the total number of confirmed attacks during a treatment period divided by the duration of the treatment (in days) times 7 days.

Time frame: 12 weeks

Secondary Outcomes

Number of Attack-free Days

The number of attack free days was the sum of the days during the treatment period for which participants reported no attacks.

Time frame: 12 weeks

Number of Participants Who Are Attack-free

The number of participants who reported no attacks during the 12-week treatment period.

Time frame: 12 weeks

Disease Activity, as Measured by the 84-day Angioedema Activity Score

Angioedema Activity Score (AAS) consists of 5 questions (period in which swelling occurred, physical discomfort, daily activity restriction, cosmetic disfigurement, and overall severity) to be answered for each day during which a subject experiences an HAE attack. A score between 0 (no symptoms) and 3 (Severe symptoms) was assigned to the responses for each question and the total daily score will be derived as the sum of the 5 question scores for each day during which the subject experiences a confirmed attack. Daily AAS ranged from 0 to 15 with higher scores indicating the greater disease severity. For participants who completed the study, the 84-day AAS was obtained by sum of AAS score during the treatment period and ranged from 0 (best) to 1,260 (worst).For participants who discontinued the study prematurely the 84-day AAS was obtained using the formula: Sum of AAS score of each day on treatment ∗ 84/Number of days on treatment.

Time frame: 12 weeks

Data from ClinicalTrials.gov

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Birmingham, Alabama, United States

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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

...and 30 more locations

Number of Participants With Treatment Emergent Adverse Events

An adverse event (AE) is any untoward medical occurrence in a clinical study participant. No causal relationship with study drug or with the clinical study itself is implied. An AE could be an unfavorable and unintended sign, symptom (including an abnormal laboratory finding), syndrome, or illness that developed or worsened during the clinical study. A serious adverse event (SAE) is any untoward medical occurrence resulting in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or other medically important event. Any graded abnormality that occurred following the initiation of study drug and up to 30 days after the last dose of study drug, and represented at least a 1-grade increase from the baseline assessment, was defined as treatment emergent.

Time frame: From first dose up to 14 weeks