Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE (OPuS-1)
Primary Purpose
Hereditary Angioedema
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
BCX4161
Placebo
Sponsored by
About this trial
This is an interventional prevention trial for Hereditary Angioedema focused on measuring Oral Prophylaxis
Eligibility Criteria
Key Inclusion Criteria:
- Written informed consent
- Clinical and laboratory diagnosis of Hereditary Angioedema Type I or II
- An average angioedema attack frequency of 1/week
- Acceptable birth control measures
Key Exclusion Criteria:
- Concurrent use of defined treatments for prophylaxis
- Pregnancy or breast-feeding
- Clinically significant medical condition, laboratory abnormality or medical history
Sites / Locations
- Dr Marcus Maurer
- Dr Emel Aygoren-Pursun
- Dr Petra Staubach
- Dr Inmaculada Martinez-Saguer
- Dr Murat Bas
- Dr Hilary Longhurst
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Placebo Comparator
Arm Label
BCX4161
Placebo
Arm Description
400 mg TID for 28 days
TID for 28 days
Outcomes
Primary Outcome Measures
Number of acute angioedema attacks
Secondary Outcome Measures
Incidence and severity of adverse events and laboratory abnormalities
Number of attack free days
Angioedema Quality of Life scores
Angioedema activity scores
Full Information
NCT ID
NCT01984788
First Posted
November 5, 2013
Last Updated
August 14, 2014
Sponsor
BioCryst Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT01984788
Brief Title
Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE
Acronym
OPuS-1
Official Title
A Phase 2a Double-Blind Placebo-Controlled 2-Period Crossover Study to Evaluate the Safety and Efficacy of BCX4161 as a Prophylactic Treatment to Reduce the Frequency of Attacks in Subjects With Hereditary Angioedema
Study Type
Interventional
2. Study Status
Record Verification Date
August 2014
Overall Recruitment Status
Completed
Study Start Date
November 2013 (undefined)
Primary Completion Date
May 2014 (Actual)
Study Completion Date
May 2014 (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
The purpose of this study is to determine whether BCX4161 given as a daily oral prophylactic treatment is safe and effective in reducing the number of acute attacks in patients with hereditary angioedema.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema
Keywords
Oral Prophylaxis
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
24 (Actual)
8. Arms, Groups, and Interventions
Arm Title
BCX4161
Arm Type
Active Comparator
Arm Description
400 mg TID for 28 days
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
TID for 28 days
Intervention Type
Drug
Intervention Name(s)
BCX4161
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Number of acute angioedema attacks
Time Frame
Over 28 days of treatment
Secondary Outcome Measure Information:
Title
Incidence and severity of adverse events and laboratory abnormalities
Time Frame
Over 28 days of treatment
Title
Number of attack free days
Time Frame
Over 28 days of treatment
Title
Angioedema Quality of Life scores
Time Frame
Over 28 days of treatment
Title
Angioedema activity scores
Time Frame
Over 28 days of treatment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Written informed consent
Clinical and laboratory diagnosis of Hereditary Angioedema Type I or II
An average angioedema attack frequency of 1/week
Acceptable birth control measures
Key Exclusion Criteria:
Concurrent use of defined treatments for prophylaxis
Pregnancy or breast-feeding
Clinically significant medical condition, laboratory abnormality or medical history
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Marcus Maurer, MD
Organizational Affiliation
Charite University, Berlin, Germany
Official's Role
Principal Investigator
Facility Information:
Facility Name
Dr Marcus Maurer
City
Berlin
Country
Germany
Facility Name
Dr Emel Aygoren-Pursun
City
Frankfurt
Country
Germany
Facility Name
Dr Petra Staubach
City
Mainz
Country
Germany
Facility Name
Dr Inmaculada Martinez-Saguer
City
Morfelden-Walldorf
Country
Germany
Facility Name
Dr Murat Bas
City
Munchen
Country
Germany
Facility Name
Dr Hilary Longhurst
City
London
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
36326435
Citation
Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
Results Reference
derived
Learn more about this trial
Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE
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