Clinical trial NCT03472040: A Long Term Safety Study of BCX7353 in Hereditary Angioedema
Official title: An Open-label Study to Evaluate the Long-term Safety of Daily Oral BCX7353 in Subjects With Type I and II Hereditary Angioedema
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Hereditary Angioedema
HAE
Prophylaxis
Keywords: BCX7353
Hereditary Angioedema
HAE
Lead sponsor: BioCryst Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2018-03-21
Enrollment: 387
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: This is an open-label study to evaluate the long term safety and effectiveness of oral treatment with BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II Hereditary Angioedema (HAE).


Eligibility criteria: Key Inclusion Criteria:

* Subjects with HAE Type I or II who either have participated in a previous BCX7353 study or, in selected countries, in the opinion of the Investigator are expected to derive benefit from an oral treatment for the prevention of angioedema attacks.
* Access to appropriate medication for treatment of acute attacks
* Acceptable effective contraception
* Written informed consent

Key Exclusion Criteria:

* Pregnancy or breast-feeding
* Any clinically significant medical condition or medical history that, in the opinion of the Investigator or Sponsor, would interfere with the subject's safety or ability to participate in the study
* Any laboratory parameter abnormality that, in the opinion of the Investigator, is clinically significant and relevant for this study
* Discontinuation of study drug due to a hypersensitivity reaction BCX7353 in a prior study
* Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
* Unacceptable noncompliance in a previous BCX7353 study (if applicable) as assessed by the Sponsor or Investigator
* Investigational drug exposure, other than BCX7353, within 30 days prior to the screening visit (or baseline if no screening visit)

Primary outcomes:
measure: Safety & Tolerability
timeFrame: Up to 96 weeks (US) / 216 weeks (Rest of World (ROW)).
Secondary outcomes:
measure: Incidence of Acute Attacks of Angioedema in Subjects During Treatment
timeFrame: Up to 96 weeks (US) / 216 weeks (ROW)
measure: The Durability of Response to Treatment
timeFrame: Up to 96 weeks (US) / 216 weeks (ROW)
measure: Patient Reported Quality of Life (QoL) During Treatment
timeFrame: Up to 96 weeks (US) / 216 weeks (ROW)
measure: Patient's Satisfaction With Medication During Long Term Administration of Berotralstat
timeFrame: Up to 96 weeks (US) / 216 weeks (ROW)
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Study Center
state: Alabama
city: Scottsdale
country: United States
facility: Study Center
state: Arizona
city: Bentonville
country: United States
facility: Study Center
state: Arkansas
city: Little Rock
country: United States
facility: Study Center
state: Arkansas
city: San Diego
country: United States
facility: Study Center
state: California
city: Walnut Creek
country: United States
facility: Study Center
state: California
city: Centennial
country: United States
facility: Study Center
state: Colorado
city: Colorado Springs
country: United States
facility: Study Center
state: Colorado
city: Wheat Ridge
country: United States
facility: Study Center
state: Colorado
city: Waterbury
country: United States
facility: Study Center
state: Connecticut
city: Tampa
country: United States
facility: Study Center
state: Florida
city: Marietta
country: United States
facility: Study Center
state: Georgia
city: Normal
country: United States
facility: Study Center
state: Illinois
city: Evansville
country: United States
facility: Study Center
state: Indiana
city: Indianapolis
country: United States
facility: Study Center
state: Indiana
city: Overland Park
country: United States
facility: Study Center
state: Kansas
city: Louisville
country: United States
facility: Study Center
state: Kentucky
city: Chevy Chase
country: United States
facility: Study Center
state: Maryland
city: Boston
country: United States
facility: Study Center
state: Massachusetts
city: Ann Arbor
country: United States
facility: Study Center
state: Michigan
city: Grand Rapids
country: United States
facility: Study Center
state: Michigan
city: Rochester
country: United States
facility: Study Center
state: Minnesota
city: Madison
country: United States
facility: Study Center
state: Mississippi
city: Saint Louis
country: United States
facility: Study Center
state: Missouri
city: Lincoln
country: United States
facility: Study Center
state: Nebraska
city: Charlotte
country: United States
facility: Study Center
state: North Carolina
city: Durham
country: United States
facility: Study Center
state: North Carolina
city: Cincinnati
country: United States
facility: Study Center
state: Ohio
city: Columbus
country: United States
facility: Study Center
state: Ohio
city: Clackamas
country: United States
facility: Study Center
state: Oregon
city: Happy Valley
country: United States
facility: Study Center
state: Oregon
city: Hershey
country: United States
facility: Study Center
state: Pennsylvania
city: East Providence
country: United States
facility: Study Center
state: Rhode Island
city: Greer
country: United States
facility: Study Center
state: South Carolina
city: Austin
country: United States
facility: Study Center
state: Texas
city: Dallas
country: United States
facility: Study Center
state: Texas
city: Irving
country: United States
facility: Study Center
state: Texas
city: San Antonio
country: United States
facility: Study Center
state: Texas
city: Murray
country: United States
facility: Study Center
state: Utah
city: Seattle
country: United States
facility: Study Center
state: Washington
city: Spokane
country: United States
facility: Study Center
state: Washington
city: Milwaukee
country: United States
facility: Study Center
state: Wisconsin
city: Adelaide
country: Australia
facility: Study Center
city: Campbelltown
country: Australia
facility: Study Center
city: Camperdown
country: Australia
facility: Study Center
city: Melbourne
country: Australia
facility: Study Center
city: Murdoch
country: Australia
facility: Study Center
city: Nedlands
country: Australia
facility: Study Center
city: Graz
country: Austria
facility: Study Center
city: Vienna
country: Austria
facility: Study Center
city: Odense
country: Denmark
facility: Study Center
city: Grenoble
country: France
facility: Study Center
city: Lille
country: France
facility: Study Center
city: Paris
country: France
facility: Study Center
city: Berlin
country: Germany
facility: Study Center
city: Frankfurt
country: Germany
facility: Study Center
city: Ulm
country: Germany
facility: Study Center
city: Central
country: Hong Kong
facility: Study Center
city: Budapest
country: Hungary
facility: Study Center
city: Ashkelon
country: Israel
facility: Study Center
city: Haifa
country: Israel
facility: Study Center
city: Tel Aviv
country: Israel
facility: Study Center
city: Tel HaShomer
country: Israel
facility: Study Center
city: Milan
country: Italy
facility: Study Center
city: Padova
country: Italy
facility: Study Center
city: Salerno
country: Italy
facility: Study Center
city: Daegu
country: Korea, Republic of
facility: Study Center
city: Donggu
country: Korea, Republic of
facility: Study Center
city: Gyeonggi-do
country: Korea, Republic of
facility: Study Center
city: Seoul
country: Korea, Republic of
facility: Study Center
city: Auckland
country: New Zealand
facility: Study Center
city: Wellington
country: New Zealand
facility: Study Center
city: Skopje
country: North Macedonia
facility: Study Center
city: Kraków
country: Poland
facility: Study Center
city: Belgrade
country: Serbia
facility: Study Center
city: Niš
country: Serbia
facility: Study Center
city: Martin
country: Slovakia
facility: Study Center
city: Cape Town
country: South Africa
facility: Study Center
city: Barcelona
country: Spain
facility: Study Center
city: Madrid
country: Spain
facility: Study Center
city: Zürich
country: Switzerland
facility: Study Center
city: Birmingham
country: United Kingdom
facility: Study center
city: Bristol
country: United Kingdom
facility: Study Center
city: Cambridge
country: United Kingdom
facility: Study Center
city: London
country: United Kingdom
facility: Study Center
city: Plymouth
country: United Kingdom
facility: Study Center
city: Southampton
country: United Kingdom
facility: Study Center
Study officials:
affiliation: Semmelweis University, Budapest, Hungary
name: Henriette Farkas, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Riedl MA, Neville D, Cloud B, Desai B, Bernstein JA. Shared decision-making in the management of hereditary angioedema: An analysis of patient and physician perspectives. Allergy Asthma Proc. 2022 Sep 1;43(5):397-405. doi: 10.2500/aap.2022.43.220050. Epub 2022 Jul 12.
citation: Farkas H, Stobiecki M, Peter J, Kinaciyan T, Maurer M, Aygoren-Pursun E, Kiani-Alikhan S, Wu A, Reshef A, Bygum A, Fain O, Hagin D, Huissoon A, Jesenak M, Lindsay K, Panovska VG, Steiner UC, Zubrinich C, Best JM, Cornpropst M, Dix D, Dobo SM, Iocca HA, Desai B, Murray SC, Nagy E, Sheridan WP. Long-term safety and effectiveness of berotralstat for hereditary angioedema: The open-label APeX-S study. Clin Transl Allergy. 2021 Jun;11(4):e12035. doi: 10.1002/clt2.12035.