Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE
OPuS-1
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
1 other identifier
interventional
24
2 countries
6
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Nov 2013
Shorter than P25 for phase_2
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
November 1, 2013
CompletedFirst Submitted
Initial submission to the registry
November 5, 2013
CompletedFirst Posted
Study publicly available on registry
November 15, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2014
CompletedAugust 18, 2014
August 1, 2014
6 months
November 5, 2013
August 14, 2014
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of acute angioedema attacks
Over 28 days of treatment
Secondary Outcomes (4)
Incidence and severity of adverse events and laboratory abnormalities
Over 28 days of treatment
Number of attack free days
Over 28 days of treatment
Angioedema Quality of Life scores
Over 28 days of treatment
Angioedema activity scores
Over 28 days of treatment
Study Arms (2)
BCX4161
ACTIVE COMPARATOR400 mg TID for 28 days
Placebo
PLACEBO COMPARATORTID for 28 days
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- Concurrent use of defined treatments for prophylaxis
- Pregnancy or breast-feeding
- Clinically significant medical condition, laboratory abnormality or medical history
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (6)
Dr Marcus Maurer
Berlin, Germany
Dr Emel Aygoren-Pursun
Frankfurt, Germany
Dr Petra Staubach
Mainz, Germany
Dr Inmaculada Martinez-Saguer
Mörfelden-Walldorf, Germany
Dr Murat Bas
München, Germany
Dr Hilary Longhurst
London, United Kingdom
Related Publications (1)
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.
PMID: 36326435DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Marcus Maurer, MD
Charite University, Berlin, Germany
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- PREVENTION
- Intervention Model
- CROSSOVER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 5, 2013
First Posted
November 15, 2013
Study Start
November 1, 2013
Primary Completion
May 1, 2014
Study Completion
May 1, 2014
Last Updated
August 18, 2014
Record last verified: 2014-08