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Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan (APeX-J)

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: NCT03873116
Recruitment Status : Active, not recruiting
First Posted : March 13, 2019
Last Update Posted : August 7, 2019
Sponsor:
Information provided by (Responsible Party):
BioCryst Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE March 11, 2019
First Posted Date  ICMJE March 13, 2019
Last Update Posted Date August 7, 2019
Actual Study Start Date  ICMJE February 28, 2019
Estimated Primary Completion Date November 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 11, 2019)
Part 1: The rate of expert-confirmed HAE attacks during dosing in the entire 24-week treatment period (Day 1 to Day 168) [ Time Frame: 24 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03873116 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 20, 2019)
  • Part 1: Change from baseline in Angioedema Quality of Life (AE- QoL) questionnaire at Week 24 (total score) [ Time Frame: 24 weeks ]
    Change from Quality of Life, on a 1-100 scale, where higher scores indicate more impairment
  • Part 1: Proportion of days with angioedema symptoms through 24 weeks [ Time Frame: 24 weeks ]
  • Part 1: Rate of expert-confirmed HAE attacks during dosing in the effective treatment period (beginning on Day 8 through 24 weeks) [ Time Frame: 24 weeks ]
Original Secondary Outcome Measures  ICMJE
 (submitted: March 11, 2019)
  • Part 1: Change from baseline in Angioedema Quality of Life (AE- QoL) questionnaire at Week 24 (total score) [ Time Frame: 24 weeks ]
  • Part 1: Proportion of days with angioedema symptoms through 24 weeks [ Time Frame: 24 weeks ]
  • Part 1: Rate of expert-confirmed HAE attacks during dosing in the effective treatment period (beginning on Day 8 through 24 weeks) [ Time Frame: 24 weeks ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan
Official Title  ICMJE A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX7353 as an Oral Treatment for the Prevention of Attacks in Subjects With Hereditary Angioedema
Brief Summary This is a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE who live in Japan.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Prevention
Condition  ICMJE Hereditary Angioedema, HAE
Intervention  ICMJE
  • Drug: BCX7353 capsules
    BCX7353 capsules administered orally once daily
  • Drug: Placebo oral capsule
    Matching placebo capsules administered orally once daily
Study Arms  ICMJE
  • Experimental: BCX7353 110mg once daily
    BCX7353 capsules administered orally once daily
    Intervention: Drug: BCX7353 capsules
  • Experimental: BCX7353 150mg once daily
    BCX7353 capsules administered orally once daily
    Intervention: Drug: BCX7353 capsules
  • Placebo Comparator: Placebo
    Matching placebo oral capsules administered orally once daily
    Intervention: Drug: Placebo oral capsule
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 Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: March 11, 2019)
24
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2020
Estimated Primary Completion Date November 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • A clinical diagnosis of hereditary angioedema (HAE) Type 1 or Type 2, defined as having a C1-INH functional level and a C4 level below the lower limit of the normal (LLN) reference range, as assessed during the Screening period.
  • Access to and ability to use one or more acute medications approved by the relevant competent authority for the treatment of acute attacks of HAE
  • Subjects must be medically appropriate for on-demand treatment as the sole medicinal management for their HAE during the study.
  • Subjects must have a specified number of expert-confirmed attacks during the run-in period of 56 days from the Screening visit.
  • 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
  • Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
  • Use of C1-INH within 14 days or use of androgens or tranexamic acid within 28 days prior to the Screening visit for prophylaxis of HAE attacks, or initiation of these drugs during the study
  • Current participation in any other investigational drug study or received another investigational drug within 30 days of the Screening visit
  • Prior enrollment in a BCX7353 study
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Japan
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03873116
Other Study ID Numbers  ICMJE BCX7353-301
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
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: Isao Ohsawa Saiyu Soka Hospital
PRS Account BioCryst Pharmaceuticals
Verification Date August 2019

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