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Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE (APeX-2)

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ClinicalTrials.gov Identifier: NCT03485911
Recruitment Status : Active, not recruiting
First Posted : April 3, 2018
Last Update Posted : November 28, 2018
Sponsor:
Information provided by (Responsible Party):
BioCryst Pharmaceuticals

Brief Summary:
This is a phase 3, multicenter, 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.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema HAE Drug: BCX7353 capsules Drug: Placebo oral capsule Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 96 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Prevention
Official Title: 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
Actual Study Start Date : February 6, 2018
Estimated Primary Completion Date : May 2019
Estimated Study Completion Date : June 2019

Resource links provided by the National Library of Medicine


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

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

Placebo Comparator: Placebo
Matching placebo administered as oral capsules once daily
Drug: Placebo oral capsule
Matching oral capsules administered once daily




Primary Outcome Measures :
  1. Part 1: The rate of investigator-confirmed HAE attacks during dosing in the entire 24-week treatment period (Day 1 to Day 168) [ Time Frame: 24 weeks ]

Secondary Outcome Measures :
  1. Part 1: Change from baseline in Angioedema Quality of Life questionnaire at Week 24 (total score) [ Time Frame: 24 weeks ]
    Change in Quality of Life, on a 1-100 scale, where higher scores indicate more impairment

  2. Part 1: Proportion of days with angioedema symptoms through 24 weeks [ Time Frame: 24 weeks ]
  3. Part 1: Rate of investigator-confirmed HAE attacks during dosing in the effective treatment period (beginning on Day 8 through 24 weeks) [ Time Frame: Day 8 to 24 weeks ]
  4. Part 1: The safety of oral BCX7353 capsules [ Time Frame: 24 weeks ]
    The number and percentage of subjects with treatment-emergent adverse events

  5. Part 2: Rate of investigator-confirmed HAE attacks [ Time Frame: Weeks 24-48 ]
  6. Part 2: Durability of response (attack rate trend over time) [ Time Frame: Weeks 24-48 ]
  7. Part 2: Durability in Angioedema Quality of Life questionnaire score [ Time Frame: Weeks 24-48 ]
    Change in Quality of Life, on a 1-100 scale, where higher scores indicate more impairment

  8. Part 2: Proportion of days with angioedema symptoms [ Time Frame: Weeks 24-48 ]


Information from the National Library of Medicine

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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:

  • A clinical diagnosis of hereditary angioedema 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.
  • Subject weight of ≥ 40 kg
  • 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 investigator-confirmed attacks during the run-in period of a maximum 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

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): NCT03485911


  Show 47 Study Locations
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
Principal Investigator: Bruce Zuraw, MD UC San Diego School of Medicine, US HAE Angioedema Center

Responsible Party: BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03485911     History of Changes
Other Study ID Numbers: BCX7353-302
First Posted: April 3, 2018    Key Record Dates
Last Update Posted: November 28, 2018
Last Verified: July 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by BioCryst Pharmaceuticals:
BCX7353

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