Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema (OPuS-4)

February 15, 2016 updated by: BioCryst Pharmaceuticals

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

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 Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

6

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: avoralstat
Five avoralstat capsules (100 mg) to be taken three times daily by mouth
Drug: avoralstat
Other Names:
  • BCX4161

What is the study measuring?

Primary Outcome Measures

Outcome 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
Time Frame: Up to 72 weeks
Up to 72 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
Angioedema attack rate (subject-reported HAE attacks normalized for time on study)
Time Frame: Up to 72 weeks
Up to 72 weeks
Durability in response (assessed as subject-reported HAE attacks)
Time Frame: change over time through 72 weeks
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
Up to 72 weeks
Quality of life as determined by the EQ-5D-5L
Time Frame: Up to 72 weeks
Up to 72 weeks
Quality of life as determined by the Angioedema Quality of Life Questionnaire
Time Frame: Up to 72 weeks
Up to 72 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioCryst Pharmaceuticals

Investigators

  • Principal Investigator: Henriette Farkas, MD, PhD, DSc, Semmelweis University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2015

Primary Completion (Actual)

February 1, 2016

Study Completion (Actual)

February 1, 2016

Study Registration Dates

First Submitted

January 20, 2016

First Submitted That Met QC Criteria

January 28, 2016

First Posted (Estimate)

February 2, 2016

Study Record Updates

Last Update Posted (Estimate)

February 17, 2016

Last Update Submitted That Met QC Criteria

February 15, 2016

Last Verified

February 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BCX4161-303

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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