Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema (APeX-1)

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ClinicalTrials.gov Identifier: NCT02870972

Recruitment Status : Completed

First Posted : August 18, 2016

Last Update Posted : October 26, 2017

Sponsor:

Information provided by (Responsible Party):

BioCryst Pharmaceuticals

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Hereditary Angioedema (HAE)	Drug: BCX7353 Drug: Placebo	Phase 2

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	75 participants
Allocation:	Randomized
Intervention Model:	Parallel Assignment
Masking:	Double (Participant, Investigator)
Primary Purpose:	Prevention
Official Title:	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
Actual Study Start Date :	August 2016
Actual Primary Completion Date :	August 2017
Actual Study Completion Date :	August 2017

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: Hereditary angioedema

Genetic and Rare Diseases Information Center resources: Hereditary Angioedema

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
Experimental: Part 1: BCX7353 350 mg once daily BCX7353 capsules, 350 mg dose administered once per day for 28 days	Drug: BCX7353 Plasma kallikrein inhibitor
Experimental: Parts 2 and 3: BCX7353 250 mg once daily BCX7353 capsules, 250 mg dose administered once per day for 28 days	Drug: BCX7353 Plasma kallikrein inhibitor
Experimental: Parts 2 and 3: BCX7353 125 mg once daily BCX7353 capsules, 125 mg dose administered once per day for 28 days	Drug: BCX7353 Plasma kallikrein inhibitor
Placebo Comparator: Parts 1, 2 and 3: Placebo Placebo capsules, administered once per day for 28 days	Drug: Placebo
Experimental: Part 3: BCX7353 62.5 mg once daily BCX7353 capsules, 62.5 mg dose administered once per day for 28 days	Drug: BCX7353 Plasma kallikrein inhibitor

Outcome Measures

Primary Outcome Measures :

Number of confirmed HAE attacks [ Time Frame: 28 days ]

Secondary Outcome Measures :

Incidence and severity of adverse events and laboratory abnormalities [ Time Frame: 28 days ]
Quality of Life, as measured by the Angioedema Quality of Life Questionnaire [ Time Frame: 28 days ]
Quality of Life, as measured by the Depression, Anxiety, Stress Scales (DASS) questionnaire [ Time Frame: 28 days ]
Plasma concentrations of BCX7353 at steady state [ Time Frame: 28 days ]

Eligibility Criteria

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Ages Eligible for Study:	18 Years to 70 Years (Adult, Older Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

Key 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

Key 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

Contacts and Locations

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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): NCT02870972

Locations

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Australia

Adelaide, Australia

Campbelltown, Australia
Austria

Graz, Austria

Vienna, Austria
Canada

Quebec City, Canada

Toronto, Canada
Denmark

Odense, Denmark
Germany

Berlin, Germany

Frankfurt, Germany

Ulm, Germany
Hungary

Budapest, Hungary
Italy

Milano, Italy

Padova, Italy

Salerno, Italy
Macedonia, The Former Yugoslav Republic of

Skopje, Macedonia, The Former Yugoslav Republic of
Spain

Barcelona, Spain

Madrid, Spain

Sevilla, Spain
Switzerland

Zürich, Switzerland
United Kingdom

Brimingham, United Kingdom

Bristol, United Kingdom

London, United Kingdom

Oxford, United Kingdom

Southampton, United Kingdom

Sponsors and Collaborators

BioCryst Pharmaceuticals

Investigators

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Principal Investigator:	Emel Aygören-Pürsün, MD	University Hospital Frankfurt Goethe University

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Aygören-Pürsün 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.

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Responsible Party:	BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT02870972 History of Changes
Other Study ID Numbers:	BCX7353-203
First Posted:	August 18, 2016 Key Record Dates
Last Update Posted:	October 26, 2017
Last Verified:	October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	Yes

Additional relevant MeSH terms:

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Angioedema Angioedemas, Hereditary Vascular Diseases Cardiovascular Diseases Urticaria Skin Diseases, Vascular	Skin Diseases Hypersensitivity, Immediate Hypersensitivity Immune System Diseases Genetic Diseases, Inborn

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