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Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

NCT ID: NCT03240133

Last Updated: 2021-04-01

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

58 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-07-31

Study Completion Date

2019-01-29

Brief Summary

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This 3-part study will evaluate the efficacy and safety of an oral kallikrein inhibitor, BCX7353, in the treatment angioedema attacks in subjects with Type I or II hereditary angioedema (HAE). In each study part, subjects will treat 3 attacks with BCX7353 (2 attacks) or placebo (1 attack), in a randomly allocated order. In Part 1, the dose of 750mg will be assessed relative to placebo in up to 36 patients. If this is shown to be effective, then a further 12 patients will be enrolled at a 500mg dose (Part 1), followed by a further 12 (if efficacy still shown) at a dose of 250mg (Part 3) to determine the minimum effective dose of BCX7353 compared to placebo for treating HAE attacks. Efficacy will be determined by subject diary entries completed at pre-defined times post-dose.

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Detailed Description

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Conditions

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Hereditary Angioedema (HAE)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Part1: BCX7353 750 mg

Group Type EXPERIMENTAL

BCX7353

Intervention Type DRUG

oral liquid formulation

Part 2: BCX7353 500 mg

Group Type EXPERIMENTAL

BCX7353

Intervention Type DRUG

oral liquid formulation

Part 3: BCX7353 250 mg

Group Type EXPERIMENTAL

BCX7353

Intervention Type DRUG

oral liquid formulation

Parts 1, 2 and 3: placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

oral liquid formulation to match BCX7353

Interventions

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BCX7353

oral liquid formulation

Intervention Type DRUG

Placebo

oral liquid formulation to match BCX7353

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Able to provide written, informed consent.
2. A clinical diagnosis of hereditary angioedema Type 1 or Type 2 as documented at any time in the medical records or at the screening visit.
3. Access to and ability to use standard of care acute attack treatment for attacks of HAE.
4. Sexually active women of child-bearing potential and sexually active men must utilize effective contraception.

Exclusion Criteria

1. Women who are pregnant or breast-feeding.
2. Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study.
3. Use of C1INH, androgens or tranexamic acid for prophylaxis of HAE attacks.
4. History of or current alcohol or drug abuse.
5. Infection with hepatitis B, hepatitis C or HIV.
6. Participation in any other investigational drug study currently or within the last 30 days.
7. Positive drugs of abuse screen (unless as used as medical treatment, e.g., with a prescription).
8. An immediate family relationship to either Sponsor employees, the Investigator or employees of the study site.
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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BioCryst Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Hilary Longhurst, MBBS, PhD

Role: PRINCIPAL_INVESTIGATOR

Barts & The London NHS Trust

Locations

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Study Center

Graz, , Austria

Site Status

Study Center

Odense, , Denmark

Site Status

Study Center

Grenoble, , France

Site Status

Study Center

Lille, , France

Site Status

Study Center

Berlin, , Germany

Site Status

Study Center

Frankfurt, , Germany

Site Status

Study Center

Budapest, , Hungary

Site Status

Study Center

Ashkelon, , Israel

Site Status

Study Center

Tel Aviv, , Israel

Site Status

Study Center

Tel Litwinsky, , Israel

Site Status

Study Center

Milan, , Italy

Site Status

Study center

Padua, , Italy

Site Status

Study Center

Salerno, , Italy

Site Status

Study Center

Skopje, , North Macedonia

Site Status

Study Center

Krakow, , Poland

Site Status

Study Center

Târgu Mureş, , Romania

Site Status

Study Center

Zurich, , Switzerland

Site Status

Study Center

Birmingham, , United Kingdom

Site Status

Study Center

Bristol, , United Kingdom

Site Status

Study Center

Cambridge, , United Kingdom

Site Status

Study Center

London, , United Kingdom

Site Status

Study Center

Manchester, , United Kingdom

Site Status

Study Center

Plymouth, , United Kingdom

Site Status

Study Center

Southampton, , United Kingdom

Site Status

Countries

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Austria Denmark France Germany Hungary Israel Italy North Macedonia Poland Romania Switzerland United Kingdom

References

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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)

Provided Documents

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Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Other Identifiers

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BCX7353-202

Identifier Type: -

Identifier Source: org_study_id

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