Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema

Phase 3TerminatedNCT02670720

BioCryst Pharmaceuticals6 enrolled

Overview

This is an open-label study designed to evaluate the long-term safety of prophylactic avoralstat (500 mg three times daily) when given to approximately 150 patients with hereditary angioedema (HAE) for a duration of up to 72 weeks. The study will also evaluate the long-term efficacy and impact on quality of life of avoralstat prophylactic treatment.

Study Type

INTERVENTIONAL

Allocation

Purpose

PREVENTION

Masking

NONE

Enrollment

Conditions

Hereditary Angioedema HAE

Interventions

avoralstatDRUG

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Males and non-pregnant, non-lactating females age ≥ 18 years. * Provide written, informed consent. * Prior completion of an avoralstat therapeutic study OR a confirmed diagnosis of HAE Type 1 or HAE Type 2. * Access to appropriate medication for the treatment of acute HAE attacks. * Adequate contraception. Exclusion Criteria: * Females who are pregnant or breast feeding. * Clinically significant medical condition or medical history. * Abnormal screening ECG, laboratory or urinalysis finding that is clinically significant. * Investigational drug exposure within 30 days (except avoralstat). * History of or current alcohol or drug abuse. * HIV or active HBV or HCV infection.

Locations (3)

Unnamed facility

Leuven, Belgium

Unnamed facility

Grenoble, France

Unnamed facility

Lille, France

Outcomes

Primary Outcomes

Proportion of subjects who discontinue due to a treatment-emergent adverse event, who experience a treatment-emergent serious AE, who experience a Grade 3 or 4 AE and who experience Grade 3 or 4 laboratory abnormalities

Time frame: Up to 72 weeks

Secondary Outcomes

Angioedema attack rate (subject-reported HAE attacks normalized for time on study)

Time frame: Up to 72 weeks

Durability in response (assessed as subject-reported HAE attacks)

Time frame: change over time through 72 weeks

Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy

Time frame: Up to 72 weeks

Quality of life as determined by the EQ-5D-5L

Time frame: Up to 72 weeks

Quality of life as determined by the Angioedema Quality of Life Questionnaire

Time frame: Up to 72 weeks

Data from ClinicalTrials.gov

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