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Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE (OPuS-1)

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ClinicalTrials.gov Identifier: NCT01984788
Recruitment Status : Completed
First Posted : November 15, 2013
Last Update Posted : August 18, 2014
Sponsor:
Information provided by (Responsible Party):
BioCryst Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema Drug: BCX4161 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Prevention
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 Start Date : November 2013
Actual Primary Completion Date : May 2014
Actual Study Completion Date : May 2014

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Active Comparator: BCX4161
400 mg TID for 28 days
Drug: BCX4161
Placebo Comparator: Placebo
TID for 28 days
Drug: Placebo



Primary Outcome Measures :
  1. Number of acute angioedema attacks [ Time Frame: Over 28 days of treatment ]

Secondary Outcome Measures :
  1. Incidence and severity of adverse events and laboratory abnormalities [ Time Frame: Over 28 days of treatment ]
  2. Number of attack free days [ Time Frame: Over 28 days of treatment ]
  3. Angioedema Quality of Life scores [ Time Frame: Over 28 days of treatment ]
  4. Angioedema activity scores [ Time Frame: Over 28 days of treatment ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01984788


Locations
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Germany
Dr Marcus Maurer
Berlin, Germany
Dr Emel Aygoren-Pursun
Frankfurt, Germany
Dr Petra Staubach
Mainz, Germany
Dr Inmaculada Martinez-Saguer
Morfelden-Walldorf, Germany
Dr Murat Bas
Munchen, Germany
United Kingdom
Dr Hilary Longhurst
London, United Kingdom
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
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Principal Investigator: Marcus Maurer, MD Charite University, Berlin, Germany

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Responsible Party: BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01984788     History of Changes
Other Study ID Numbers: BCX4161-203
First Posted: November 15, 2013    Key Record Dates
Last Update Posted: August 18, 2014
Last Verified: August 2014

Keywords provided by BioCryst Pharmaceuticals:
Oral Prophylaxis

Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn