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

Phase 3

Terminated

• Conditions: Hereditary Angioedema; HAE
• Interventions: Drug: avoralstat

• Registration Number: NCT02670720
• Lead Sponsor: BioCryst Pharmaceuticals

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2015-12
• Study First Submitted: 2016-01-20
• Study First Submitted QC: 2016-01-28
• Status Verified: 2016-02
• Primary Completion: 2016-02
• Study Completion: 2016-02
• Study First Posted: 2016-02-02
• Last Update Submitted: 2016-02-15
• Last Update Posted: 2016-02-17

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 6

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
avoralstat	avoralstat	Five avoralstat capsules (100 mg) to be taken three times daily by mouth

Primary Outcome Measures

Name	Time	Method
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	Up to 72 weeks

Secondary Outcome Measures

Name	Time	Method
Durability in response (assessed as subject-reported HAE attacks)	change over time through 72 weeks
Quality of life as determined by the Angioedema Quality of Life Questionnaire	Up to 72 weeks
Angioedema attack rate (subject-reported HAE attacks normalized for time on study)	Up to 72 weeks
Quality of life as determined by the EQ-5D-5L	Up to 72 weeks
Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy	Up to 72 weeks