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

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: NCT03485911
Recruitment Status : Active, not recruiting
First Posted : April 3, 2018
Last Update Posted : March 8, 2019
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

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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 : October 2020
Estimated Study Completion Date : October 2020

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. The safety of oral BCX7353 capsules: The number and percentage of subjects with treatment-emergent adverse events [ Time Frame: Day 1 - Week 96 ]
  2. 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

  3. Part 1: Proportion of days with angioedema symptoms through 24 weeks [ Time Frame: 24 weeks ]
  4. 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 ]
  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 ]
  9. Part 3: Rate of HAE attacks during dosing [ Time Frame: Weeks 48-96 ]
  10. Part 3: Durability of response (attack rate trend over time) [ Time Frame: Weeks 48-96 ]
  11. Part 3: Durability in Angioedema Quality of Life questionnaire score [ Time Frame: Weeks 48-96 ]
  12. Part 3: Proportion of days with angioedema symptoms [ Time Frame: Weeks 48-96 ]


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


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Locations
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United States, Alabama
Study center
Birmingham, Alabama, United States, 35209
United States, Arizona
Study center
Scottsdale, Arizona, United States, 85251
United States, Arkansas
Study center
Little Rock, Arkansas, United States, 72205
United States, California
Study Center
San Diego, California, United States, 92122
Study center
Santa Monica, California, United States, 90404
Study Center
Walnut Creek, California, United States, 94598
United States, Colorado
Study center
Colorado Springs, Colorado, United States, 80907
United States, Florida
Study Center
Tampa, Florida, United States, 33613
United States, Maryland
Study center
Chevy Chase, Maryland, United States, 20815
United States, Massachusetts
Study Center
Boston, Massachusetts, United States, 02114
United States, Michigan
Study Center
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
Study center
Plymouth, Minnesota, United States, 55446
United States, Missouri
Study Center
Saint Louis, Missouri, United States, 63141
United States, New Jersey
Study center
Belleville, New Jersey, United States, 07109
Study Center
Fair Lawn, New Jersey, United States, 07410
Study center
Piscataway, New Jersey, United States, 08854
United States, New York
Study Center
New York, New York, United States, 10029
United States, North Carolina
Study Site
Charlotte, North Carolina, United States, 28277
Study Center
Durham, North Carolina, United States, 27705
United States, Ohio
Study Center
Cincinnati, Ohio, United States, 45231
Study center
Columbus, Ohio, United States, 43235
United States, Oregon
Study center
Clackamas, Oregon, United States, 97015
United States, Pennsylvania
Study Center
Hershey, Pennsylvania, United States, 17033
United States, Texas
Study center
Austin, Texas, United States, 78731
Study center
Dallas, Texas, United States, 75231
Study Center
San Antonio, Texas, United States, 78229
United States, Washington
Study Center
Spokane, Washington, United States, 99202
Austria
Study Center
Vienna, Austria, 1090
Canada, Ontario
Study Center
Ottawa, Ontario, Canada, K1G 6C6
Study Center
Toronto, Ontario, Canada, M4V 1R2
Canada
Study Center
Québec, Canada, G1V 4W2
Czechia
Study Center
Brno, Czechia, 656 91
Study Center
Plzen, Czechia, 30460
France
Study Center
Grenoble, France, 38043
Study Center
Paris, France, 75012
Germany
Study Center
Berlin, Germany, 10117
Study Center
Frankfurt, Germany, D-60590
Hungary
Study Center
Budapest, Hungary, H-1225
Macedonia, The Former Yugoslav Republic of
Study Center
Skopje, Macedonia, The Former Yugoslav Republic of, 1000
Romania
Study Center
Sângeorgiu de Mureș, Jud. Mureș, Romania, 547530
Spain
Study Center
Barcelona, Spain, 08907
Study Center
Madrid, Spain, 28046
Study Center
Seville, Spain, 41013
United Kingdom
Study Center
Cambridge, United Kingdom, CB2 0QQ
Study Center
Frimley, United Kingdom, GU16 7UJ
Study Center
London, United Kingdom, E1 2ES
Study Center
Plymouth, United Kingdom, PL6 8DH
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
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Principal Investigator: Bruce Zuraw, MD UC San Diego School of Medicine, US HAE Angioedema Center

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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: March 8, 2019
Last Verified: March 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

Keywords provided by BioCryst Pharmaceuticals:
BCX7353

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