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A Long Term Safety Study of BCX7353 in Hereditary Angioedema (APeX-S)

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ClinicalTrials.gov Identifier: NCT03472040
Recruitment Status : Recruiting
First Posted : March 21, 2018
Last Update Posted : July 10, 2019
Sponsor:
Information provided by (Responsible Party):
BioCryst Pharmaceuticals

Brief Summary:
This is an open-label study to evaluate the long term safety and effectiveness of oral treatment with BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema HAE Prophylaxis Drug: BCX7353 Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 275 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: An Open-label Study to Evaluate the Long-term Safety of Daily Oral BCX7353 in Subjects With Type I and II Hereditary Angioedema
Actual Study Start Date : February 16, 2018
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : February 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: BCX7353 110 mg once daily Drug: BCX7353
BCX7353 mg oral capsules administered once daily

Experimental: BCX7353 150 mg once daily Drug: BCX7353
BCX7353 mg oral capsules administered once daily




Primary Outcome Measures :
  1. The long term safety of oral BCX7353 capsules [ Time Frame: 96 weeks ]
    The number and percentage of subjects with treatment-emergent adverse events


Secondary Outcome Measures :
  1. The rate of acute attacks of angioedema during treatment [ Time Frame: 96 weeks ]
  2. The durability of response to treatment [ Time Frame: 96 weeks ]
    To evaluate if the rate of attacks remains consistent (durable) over time

  3. Patient reported quality of life (QoL) during treatment [ Time Frame: 96 weeks ]
    To measure changes in quality of life over time from baseline, assessed by use of an HAE-specific Angioedema QoL (AE-QoL) questionnaire

  4. Patient's satisfaction with medication during long term administration of BCX7353 [ Time Frame: 96 weeks ]
    To measure satisfaction with study medication using the Treatment Satisfaction Questionnaire for Medication (TSQM)



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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Subjects with HAE Type I or II who either have participated in a previous BCX7353 study or, in selected countries, in the opinion of the Investigator are expected to derive benefit from an oral treatment for the prevention of angioedema attacks.
  • Access to appropriate medication for treatment of acute attacks
  • Acceptable effective contraception
  • Written informed consent

Key Exclusion Criteria:

  • Pregnancy or breast-feeding
  • Any clinically significant medical condition or medical history that, in the opinion of the Investigator or Sponsor, would interfere with the subject's safety or ability to participate in the study
  • Any laboratory parameter abnormality that, in the opinion of the Investigator, is clinically significant and relevant for this study
  • Discontinuation of study drug due to a hypersensitivity reaction BCX7353 in a prior study
  • Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
  • Unacceptable noncompliance in a previous BCX7353 study (if applicable) as assessed by the Sponsor or Investigator
  • Investigational drug exposure, other than BCX7353, within 30 days prior to the screening visit (or baseline if no screening visit)

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): NCT03472040


Contacts
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Contact: Study director +1 919 859 1302 clinicaltrials@biocryst.com

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Locations
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United States, Alabama
Study Center Recruiting
Birmingham, Alabama, United States, 35209
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United States, Maryland
Study Center Recruiting
Chevy Chase, Maryland, United States, 20815
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United States, Missouri
Study Center Recruiting
Saint Louis, Missouri, United States, 63141
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United States, Nebraska
Study Center Recruiting
Lincoln, Nebraska, United States, 68505
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United States, Ohio
Study Center Recruiting
Cincinnati, Ohio, United States, 45231
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United States, Washington
Study Center Recruiting
Spokane, Washington, United States, 99202
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Australia
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Adelaide, Australia
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Campbelltown, Australia
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Camperdown, Australia
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Melbourne, Australia
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Murdoch, Australia
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Nedlands, Australia
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Austria
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Graz, Austria
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Vienna, Austria
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Denmark
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Odense, Denmark
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France
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Grenoble, France
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Lille, France
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Paris, France
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Germany
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Berlin, Germany
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Frankfurt, Germany
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Ulm, Germany
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Hong Kong
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Central, Hong Kong
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Hungary
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Budapest, Hungary
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Israel
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Ashkelon, Israel
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Haifa, Israel
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Tel Aviv, Israel
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Tel HaShomer, Israel
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Italy
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Milan, Italy
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Padova, Italy
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Salerno, Italy
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Korea, Republic of
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Daegu, Korea, Republic of
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Donggu, Korea, Republic of
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Gyeonggi-do, Korea, Republic of
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Seoul, Korea, Republic of
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New Zealand
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Auckland, New Zealand
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Wellington, New Zealand
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North Macedonia
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Skopje, North Macedonia
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Poland
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Kraków, Poland
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Serbia
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Belgrade, Serbia
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Niš, Serbia
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Slovakia
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Martin, Slovakia
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South Africa
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Cape Town, South Africa
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Spain
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Barcelona, Spain
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Madrid, Spain
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Switzerland
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Zürich, Switzerland
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United Kingdom
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Birmingham, United Kingdom
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Bristol, United Kingdom
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Cambridge, United Kingdom
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London, United Kingdom
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Plymouth, United Kingdom
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Study Center Recruiting
Southampton, United Kingdom
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
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Principal Investigator: Henriette Farkas, MD Semmelweis University, Budapest, Hungary

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Responsible Party: BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03472040     History of Changes
Other Study ID Numbers: BCX7353-204
First Posted: March 21, 2018    Key Record Dates
Last Update Posted: July 10, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes

Keywords provided by BioCryst Pharmaceuticals:
BCX7353
Hereditary Angioedema
HAE

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