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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

Tracking Information
First Submitted Date  ICMJE February 27, 2018
First Posted Date  ICMJE March 21, 2018
Last Update Posted Date July 10, 2019
Actual Study Start Date  ICMJE February 16, 2018
Estimated Primary Completion Date February 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 5, 2019)
The long term safety of oral BCX7353 capsules [ Time Frame: 96 weeks ]
The number and percentage of subjects with treatment-emergent adverse events
Original Primary Outcome Measures  ICMJE
 (submitted: March 14, 2018)
The long term safety of oral BCX7353 capsules based on analysis of treatment emergent adverse events and laboratory abnormalities [ Time Frame: 48 weeks ]
Change History Complete list of historical versions of study NCT03472040 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 5, 2019)
  • The rate of acute attacks of angioedema during treatment [ Time Frame: 96 weeks ]
  • The durability of response to treatment [ Time Frame: 96 weeks ]
    To evaluate if the rate of attacks remains consistent (durable) over time
  • 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
  • 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)
Original Secondary Outcome Measures  ICMJE
 (submitted: March 14, 2018)
  • The rate of acute attacks of angioedema during treatment [ Time Frame: 48 weeks ]
  • The durability of response to treatment [ Time Frame: 48 weeks ]
    To evaluate if the rate of attacks remains consistent (durable) over time
  • Patient reported quality of life (QoL) during treatment [ Time Frame: 48 weeks ]
    To measure changes in quality of life over time from baseline, assessed by use of an HAE-specific Angioedema QoL (AE-QoL) questionnaire
  • Patient's satisfaction with medication during long term administration of BCX7353 [ Time Frame: 48 weeks ]
    To measure satisfaction with study medication using the Treatment Satisfaction Questionnaire for Medication (TSQM)
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Long Term Safety Study of BCX7353 in Hereditary Angioedema
Official Title  ICMJE An Open-label Study to Evaluate the Long-term Safety of Daily Oral BCX7353 in Subjects With Type I and II Hereditary Angioedema
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Condition  ICMJE
  • Hereditary Angioedema
  • HAE
  • Prophylaxis
Intervention  ICMJE Drug: BCX7353
BCX7353 mg oral capsules administered once daily
Study Arms  ICMJE
  • Experimental: BCX7353 110 mg once daily
    Intervention: Drug: BCX7353
  • Experimental: BCX7353 150 mg once daily
    Intervention: Drug: BCX7353
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 9, 2019)
275
Original Estimated Enrollment  ICMJE
 (submitted: March 14, 2018)
160
Estimated Study Completion Date  ICMJE February 2021
Estimated Primary Completion Date February 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Gender Based Eligibility: Yes
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Study director +1 919 859 1302 clinicaltrials@biocryst.com
Listed Location Countries  ICMJE Australia,   Austria,   Denmark,   France,   Germany,   Hong Kong,   Hungary,   Israel,   Italy,   Korea, Republic of,   New Zealand,   North Macedonia,   Poland,   Serbia,   Slovakia,   South Africa,   Spain,   Switzerland,   United Kingdom,   United States
Removed Location Countries Macedonia, The Former Yugoslav Republic of
 
Administrative Information
NCT Number  ICMJE NCT03472040
Other Study ID Numbers  ICMJE BCX7353-204
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party BioCryst Pharmaceuticals
Study Sponsor  ICMJE BioCryst Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Henriette Farkas, MD Semmelweis University, Budapest, Hungary
PRS Account BioCryst Pharmaceuticals
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP