Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema

NCT ID: NCT02870972

Last Updated: 2021-03-23

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

75 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-08-31

Study Completion Date

2017-08-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hereditary Angioedema (HAE)

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Part 1: BCX7353 350 mg once daily

BCX7353 capsules, 350 mg dose administered once per day for 28 days

Group Type EXPERIMENTAL

BCX7353

Intervention Type DRUG

Plasma kallikrein inhibitor

Parts 2 and 3: BCX7353 250 mg once daily

BCX7353 capsules, 250 mg dose administered once per day for 28 days

Group Type EXPERIMENTAL

BCX7353

Intervention Type DRUG

Plasma kallikrein inhibitor

Parts 2 and 3: BCX7353 125 mg once daily

BCX7353 capsules, 125 mg dose administered once per day for 28 days

Group Type EXPERIMENTAL

BCX7353

Intervention Type DRUG

Plasma kallikrein inhibitor

Parts 1, 2 and 3: Placebo

Placebo capsules, administered once per day for 28 days

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Part 3: BCX7353 62.5 mg once daily

BCX7353 capsules, 62.5 mg dose administered once per day for 28 days

Group Type EXPERIMENTAL

BCX7353

Intervention Type DRUG

Plasma kallikrein inhibitor

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

BCX7353

Plasma kallikrein inhibitor

Intervention Type DRUG

Placebo

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* 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

Exclusion Criteria

* 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
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

BioCryst Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Emel Aygören-Pürsün, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital Frankfurt Goethe University

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Adelaide, , Australia

Site Status

Campbelltown, , Australia

Site Status

Graz, , Austria

Site Status

Vienna, , Austria

Site Status

Québec, , Canada

Site Status

Toronto, , Canada

Site Status

Odense, , Denmark

Site Status

Berlin, , Germany

Site Status

Frankfurt, , Germany

Site Status

Ulm, , Germany

Site Status

Budapest, , Hungary

Site Status

Milan, , Italy

Site Status

Padua, , Italy

Site Status

Salerno, , Italy

Site Status

Skopje, , North Macedonia

Site Status

Barcelona, , Spain

Site Status

Madrid, , Spain

Site Status

Seville, , Spain

Site Status

Zurich, , Switzerland

Site Status

Brimingham, , United Kingdom

Site Status

Bristol, , United Kingdom

Site Status

London, , United Kingdom

Site Status

Oxford, , United Kingdom

Site Status

Southampton, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Australia Austria Canada Denmark Germany Hungary Italy North Macedonia Spain Switzerland United Kingdom

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type DERIVED
PMID: 36408587 (View on PubMed)

Beard 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.

Reference Type DERIVED
PMID: 36326435 (View on PubMed)

Aygoren-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.

Reference Type DERIVED
PMID: 30044938 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

BCX7353-203

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

STOP-HAE: A Phase 3 Study of ADX-324 in HAE
NCT06960213 RECRUITING PHASE3
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
NCT05120830 ACTIVE_NOT_RECRUITING PHASE1/PHASE2