Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema
APeX-1
A Randomized, Double-blind, Placebo-controlled, Dose-ranging, Parallel-group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BCX7353 as a Preventative Treatment to Reduce the Frequency of Attacks in Subjects With Hereditary Angioedema
1 other identifier
interventional
75
10 countries
22
Brief Summary
This 3-part study will evaluate the safety and efficacy of an oral treatment, BCX7353, in preventing angioedema attacks in subjects with hereditary angioedema (HAE). In Part 1 of the study, eligible subjects will be randomized to receive oral BCX7353 or placebo for 4 weeks. Assuming successful completion of Part 1, additional subjects will be randomized in Part 2 to one of 2 lower doses of BCX7353 or placebo. Part 3 will enroll additional subjects into one of three doses of BCX7353 or placebo. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical and pharmacologic outcomes, and the safety and tolerability of each dose of BCX7353 compared to placebo.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Aug 2016
Shorter than P25 for phase_2
22 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
August 1, 2016
CompletedFirst Submitted
Initial submission to the registry
August 10, 2016
CompletedFirst Posted
Study publicly available on registry
August 18, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2017
CompletedResults Posted
Study results publicly available
March 4, 2021
CompletedMarch 23, 2021
March 1, 2021
1 year
August 10, 2016
November 17, 2020
March 3, 2021
Conditions
Outcome Measures
Primary Outcomes (2)
Number of Confirmed HAE Attacks
Efficacy was evaluated by the number of acute angioedema attacks. To ensure that consistent, objective assessments were used in accepting subject-reported attack data, a panel of expert physicians in the treatment of HAE patients adjudicated all subject-reported attacks prior to their inclusion in primary efficacy analyses.
Investigators collected data from patient diaries from the first day of dosing through to Day 29 or last day of dosing +24 hrs (the effective dosing period).
Proportion of Subjects Who Were HAE Attack-free During the Entire Dosing Period
Assessment of the proportion of subjects who had no HAE attacks during the entire dosing period
Investigators collected data from patient diaries from the first day of dosing through to Day 29 or last day of dosing +24 hrs (the effective dosing period).
Secondary Outcomes (6)
Number of Confirmed Abdominal HAE Attacks
Investigators collected data from patient diaries from the first day of dosing through to Day 29 or last day of dosing +24 hrs (the effective dosing period).
Number of Confirmed Peripheral HAE Attacks
Investigators collected data from patient diaries from the first day of dosing through to Day 29 or last day of dosing +24 hrs (the effective dosing period).
HAE Attacks Requiring Treatment
Investigators collected data from patient diaries from the first day of dosing through to Day 29 or last day of dosing +24 hrs (the effective dosing period).
HAE Disease Activity - Modified Angioedema Activity Score
28-day treatment period + 1 day
Angioedema Quality of Life (AE-QoL)
The subject-completed AE-QoL was administered at baseline (Day 1) and at Day 29
- +1 more secondary outcomes
Study Arms (5)
Part 1: BCX7353 350 mg once daily
EXPERIMENTALBCX7353 capsules, 350 mg dose administered once per day for 28 days
Parts 2 and 3: BCX7353 250 mg once daily
EXPERIMENTALBCX7353 capsules, 250 mg dose administered once per day for 28 days
Parts 2 and 3: BCX7353 125 mg once daily
EXPERIMENTALBCX7353 capsules, 125 mg dose administered once per day for 28 days
Parts 1, 2 and 3: Placebo
PLACEBO COMPARATORPlacebo capsules, administered once per day for 28 days
Part 3: BCX7353 62.5 mg once daily
EXPERIMENTALBCX7353 capsules, 62.5 mg dose administered once per day for 28 days
Interventions
Eligibility Criteria
You may qualify if:
- A clinical diagnosis of HAE type I or II
- Documented HAE attacks within a defined calendar period
- Access to acute attack medications
- Sexually active women of child-bearing potential and sexually active men must utilize effective contraception
You may not qualify if:
- Women who are pregnant or breast-feeding
- Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study
- Use of C1INH, androgens or tranexamic acid for prophylaxis of HAE attacks
- History of or current alcohol or drug abuse
- Infection with hepatitis B, hepatitis C or HIV
- Participation in any other investigational drug study currently or within the last 30 days
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (24)
Unknown Facility
Adelaide, Australia
Unknown Facility
Campbelltown, Australia
Unknown Facility
Graz, Austria
Unknown Facility
Vienna, Austria
Unknown Facility
Québec, Canada
Unknown Facility
Toronto, Canada
Unknown Facility
Odense, Denmark
Unknown Facility
Berlin, Germany
Unknown Facility
Frankfurt, Germany
Unknown Facility
Ulm, Germany
Unknown Facility
Budapest, Hungary
Unknown Facility
Milan, Italy
Unknown Facility
Padua, Italy
Unknown Facility
Salerno, Italy
Unknown Facility
Skopje, North Macedonia
Unknown Facility
Barcelona, Spain
Unknown Facility
Madrid, Spain
Unknown Facility
Seville, Spain
Unknown Facility
Zurich, Switzerland
Unknown Facility
Brimingham, United Kingdom
Unknown Facility
Bristol, United Kingdom
Unknown Facility
London, United Kingdom
Unknown Facility
Oxford, United Kingdom
Unknown Facility
Southampton, United Kingdom
Related Publications (3)
Farkas H, Balla Z. A review of berotralstat for the treatment of hereditary angioedema. Expert Rev Clin Immunol. 2023 Feb;19(2):145-153. doi: 10.1080/1744666X.2023.2150611. Epub 2022 Nov 29.
PMID: 36408587DERIVEDBeard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
PMID: 36326435DERIVEDAygoren-Pursun E, Bygum A, Grivcheva-Panovska V, Magerl M, Graff J, Steiner UC, Fain O, Huissoon A, Kinaciyan T, Farkas H, Lleonart R, Longhurst HJ, Rae W, Triggiani M, Aberer W, Cancian M, Zanichelli A, Smith WB, Baeza ML, Du-Thanh A, Gompels M, Gonzalez-Quevedo T, Greve J, Guilarte M, Katelaris C, Dobo S, Cornpropst M, Clemons D, Fang L, Collis P, Sheridan W, Maurer M, Cicardi M. Oral Plasma Kallikrein Inhibitor for Prophylaxis in Hereditary Angioedema. N Engl J Med. 2018 Jul 26;379(4):352-362. doi: 10.1056/NEJMoa1716995.
PMID: 30044938DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- BioCryst Pharmaceuticals Inc
Study Officials
- PRINCIPAL INVESTIGATOR
Emel Aygören-Pürsün, MD
University Hospital Frankfurt Goethe University
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- PREVENTION
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 10, 2016
First Posted
August 18, 2016
Study Start
August 1, 2016
Primary Completion
August 1, 2017
Study Completion
August 1, 2017
Last Updated
March 23, 2021
Results First Posted
March 4, 2021
Record last verified: 2021-03
Data Sharing
- IPD Sharing
- Will share