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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
BioCryst Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Hereditary Angioedema focused on measuring Oral Prophylaxis

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

First Posted
November 5, 2013
Last Updated
August 14, 2014
Sponsor
BioCryst Pharmaceuticals
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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

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Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE

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