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

OPuS-4: An Open-label Study to Evaluate the Long-term Safety of Avoralstat in Subjects With Hereditary Angioedema

Status

Terminated

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02670720

Acronym

OPuS-4

Enrollment

Registered

2016-02-02

Start date

2015-12-31

Completion date

2016-02-29

Last updated

2016-02-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

HAE, Hereditary Angioedema

Keywords

BCX4161, avoralstat, HAE, hereditary angioedema, prophylaxis

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.

Interventions

DRUGavoralstat

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

PREVENTION

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame
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

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

Countries

Belgium, France

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026