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Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema (APeX-1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02870972
Recruitment Status : Completed
First Posted : August 18, 2016
Last Update Posted : October 26, 2017
Sponsor:
Information provided by (Responsible Party):
BioCryst Pharmaceuticals

Study Description
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Brief Summary:
This 3-part study will evaluate the safety and efficacy of an oral treatment, BCX7353, in preventing angioedema attacks in subjects with hereditary angioedema (HAE). In Part 1 of the study, eligible subjects will be randomized to receive oral BCX7353 or placebo for 4 weeks. Assuming successful completion of Part 1, additional subjects will be randomized in Part 2 to one of 2 lower doses of BCX7353 or placebo. Part 3 will enroll additional subjects into one of three doses of BCX7353 or placebo. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical and pharmacologic outcomes, and the safety and tolerability of each dose of BCX7353 compared to placebo.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema (HAE) Drug: BCX7353 Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 75 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Prevention
Official Title: A Randomized, Double-blind, Placebo-controlled, Dose-ranging, Parallel-group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BCX7353 as a Preventative Treatment to Reduce the Frequency of Attacks in Subjects With Hereditary Angioedema
Actual Study Start Date : August 2016
Actual Primary Completion Date : August 2017
Actual Study Completion Date : August 2017

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Part 1: BCX7353 350 mg once daily
BCX7353 capsules, 350 mg dose administered once per day for 28 days
 Drug: BCX7353
Plasma kallikrein inhibitor
Experimental: Parts 2 and 3: BCX7353 250 mg once daily
BCX7353 capsules, 250 mg dose administered once per day for 28 days
 Drug: BCX7353
Plasma kallikrein inhibitor
Experimental: Parts 2 and 3: BCX7353 125 mg once daily
BCX7353 capsules, 125 mg dose administered once per day for 28 days
 Drug: BCX7353
Plasma kallikrein inhibitor
Placebo Comparator: Parts 1, 2 and 3: Placebo
Placebo capsules, administered once per day for 28 days
 Drug: Placebo
Experimental: Part 3: BCX7353 62.5 mg once daily
BCX7353 capsules, 62.5 mg dose administered once per day for 28 days
 Drug: BCX7353
Plasma kallikrein inhibitor


Outcome Measures
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Primary Outcome Measures :
  1. Number of confirmed HAE attacks [ Time Frame: 28 days ]

Secondary Outcome Measures :
  1. Incidence and severity of adverse events and laboratory abnormalities [ Time Frame: 28 days ]
  2. Quality of Life, as measured by the Angioedema Quality of Life Questionnaire [ Time Frame: 28 days ]
  3. Quality of Life, as measured by the Depression, Anxiety, Stress Scales (DASS) questionnaire [ Time Frame: 28 days ]
  4. Plasma concentrations of BCX7353 at steady state [ Time Frame: 28 days ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • A clinical diagnosis of HAE type I or II
  • Documented HAE attacks within a defined calendar period
  • Access to acute attack medications
  • Sexually active women of child-bearing potential and sexually active men must utilize effective contraception

Key Exclusion Criteria:

  • Women who are pregnant or breast-feeding
  • Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study
  • Use of C1INH, androgens or tranexamic acid for prophylaxis of HAE attacks
  • History of or current alcohol or drug abuse
  • Infection with hepatitis B, hepatitis C or HIV
  • Participation in any other investigational drug study currently or within the last 30 days
Contacts and Locations
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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): NCT02870972


Locations
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Australia
Adelaide, Australia
Campbelltown, Australia
Austria
Graz, Austria
Vienna, Austria
Canada
Quebec City, Canada
Toronto, Canada
Denmark
Odense, Denmark
Germany
Berlin, Germany
Frankfurt, Germany
Ulm, Germany
Hungary
Budapest, Hungary
Italy
Milano, Italy
Padova, Italy
Salerno, Italy
Macedonia, The Former Yugoslav Republic of
Skopje, Macedonia, The Former Yugoslav Republic of
Spain
Barcelona, Spain
Madrid, Spain
Sevilla, Spain
Switzerland
Zürich, Switzerland
United Kingdom
Brimingham, United Kingdom
Bristol, United Kingdom
London, United Kingdom
Oxford, United Kingdom
Southampton, United Kingdom
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
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Principal Investigator: Emel Aygören-Pürsün, MD University Hospital Frankfurt Goethe University
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02870972    
Other Study ID Numbers: BCX7353-203
First Posted: August 18, 2016    Key Record Dates
Last Update Posted: October 26, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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