Back to resultsOpen-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema (OPuS-4)
Primary Purpose
Hereditary Angioedema, HAE
Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
avoralstat
Sponsored by
About this trial
This is an interventional prevention trial for Hereditary Angioedema focused on measuring BCX4161, avoralstat, HAE, hereditary angioedema, prophylaxis
Eligibility Criteria
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.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
avoralstat
Arm Description
Five avoralstat capsules (100 mg) to be taken three times daily by mouth
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
Angioedema attack rate (subject-reported HAE attacks normalized for time on study)
Durability in response (assessed as subject-reported HAE attacks)
Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy
Quality of life as determined by the EQ-5D-5L
Quality of life as determined by the Angioedema Quality of Life Questionnaire
Full Information
NCT ID
NCT02670720
First Posted
January 20, 2016
Last Updated
February 15, 2016
Sponsor
BioCryst Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT02670720
Brief Title
Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema
Acronym
OPuS-4
Official Title
OPuS-4: An Open-label Study to Evaluate the Long-term Safety of Avoralstat in Subjects With Hereditary Angioedema
Study Type
Interventional
2. Study Status
Record Verification Date
February 2016
Overall Recruitment Status
Terminated
Why Stopped
preceding efficacy study of avoralstat in its current formulation was not significant and does not support continued development
Study Start Date
December 2015 (undefined)
Primary Completion Date
February 2016 (Actual)
Study Completion Date
February 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
BioCryst Pharmaceuticals
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema, HAE
Keywords
BCX4161, avoralstat, HAE, hereditary angioedema, prophylaxis
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
avoralstat
Arm Type
Experimental
Arm Description
Five avoralstat capsules (100 mg) to be taken three times daily by mouth
Intervention Type
Drug
Intervention Name(s)
avoralstat
Other Intervention Name(s)
BCX4161
Primary Outcome Measure Information:
Title
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 Outcome Measure Information:
Title
Angioedema attack rate (subject-reported HAE attacks normalized for time on study)
Time Frame
Up to 72 weeks
Title
Durability in response (assessed as subject-reported HAE attacks)
Time Frame
change over time through 72 weeks
Title
Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy
Time Frame
Up to 72 weeks
Title
Quality of life as determined by the EQ-5D-5L
Time Frame
Up to 72 weeks
Title
Quality of life as determined by the Angioedema Quality of Life Questionnaire
Time Frame
Up to 72 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Henriette Farkas, MD, PhD, DSc
Organizational Affiliation
Semmelweis University
Official's Role
Principal Investigator
Facility Information:
City
Leuven
Country
Belgium
City
Grenoble
Country
France
City
Lille
Country
France
12. IPD Sharing Statement
Learn more about this trial
Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema
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