Clinical trial NCT02870972: Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hereditary Angioedema (HAE)
Lead sponsor: BioCryst Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2016-08-18
Enrollment: 75
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

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.


Eligibility 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

Primary outcomes:
measure: Number of Confirmed HAE Attacks
timeFrame: 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).
measure: Proportion of Subjects Who Were HAE Attack-free During the Entire Dosing Period
timeFrame: 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:
measure: Number of Confirmed Abdominal HAE Attacks
timeFrame: 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).
measure: Number of Confirmed Peripheral HAE Attacks
timeFrame: 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).
measure: HAE Attacks Requiring Treatment
timeFrame: 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).
measure: HAE Disease Activity - Modified Angioedema Activity Score
timeFrame: 28-day treatment period + 1 day
measure: Angioedema Quality of Life (AE-QoL)
timeFrame: The subject-completed AE-QoL was administered at baseline (Day 1) and at Day 29
measure: DASS (Depression, Anxiety and Stress Scales)
timeFrame: The DASS was administered at baseline (Day 1), Day 14, and Day 29.
Other outcomes:
Locations:
city: Adelaide
country: Australia
city: Campbelltown
country: Australia
city: Graz
country: Austria
city: Vienna
country: Austria
city: Quebec City
country: Canada
city: Toronto
country: Canada
city: Odense
country: Denmark
city: Berlin
country: Germany
city: Frankfurt
country: Germany
city: Ulm
country: Germany
city: Budapest
country: Hungary
city: Milano
country: Italy
city: Padova
country: Italy
city: Salerno
country: Italy
city: Skopje
country: North Macedonia
city: Barcelona
country: Spain
city: Madrid
country: Spain
city: Sevilla
country: Spain
city: Zürich
country: Switzerland
city: Brimingham
country: United Kingdom
city: Bristol
country: United Kingdom
city: London
country: United Kingdom
city: Oxford
country: United Kingdom
city: Southampton
country: United Kingdom
Study officials:
affiliation: University Hospital Frankfurt Goethe University
name: Emel Aygören-Pürsün, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.