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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03472040




Registration number
NCT03472040
Ethics application status
Date submitted
27/02/2018
Date registered
21/03/2018

Titles & IDs
Public title
A Long Term Safety Study of BCX7353 in Hereditary Angioedema
Scientific title
An Open-label Study to Evaluate the Long-term Safety of Daily Oral BCX7353 in Subjects With Type I and II Hereditary Angioedema
Secondary ID [1] 0 0
BCX7353-204
Universal Trial Number (UTN)
Trial acronym
APeX-S
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema 0 0
HAE 0 0
Prophylaxis 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - BCX7353

Experimental: BCX7353 150 mg once daily -


Treatment: Drugs: BCX7353
BCX7353 mg oral capsules administered once daily
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety & Tolerability
Timepoint [1] 0 0
Up to 96 weeks (US) / 216 weeks (Rest of World (ROW)).
Secondary outcome [1] 0 0
Incidence of Acute Attacks of Angioedema in Subjects During Treatment
Timepoint [1] 0 0
Up to 96 weeks (US) / 216 weeks (ROW)
Secondary outcome [2] 0 0
The Durability of Response to Treatment
Timepoint [2] 0 0
Up to 96 weeks (US) / 216 weeks (ROW)
Secondary outcome [3] 0 0
Patient Reported Quality of Life (QoL) During Treatment
Timepoint [3] 0 0
Up to 96 weeks (US) / 216 weeks (ROW)
Secondary outcome [4] 0 0
Patient's Satisfaction With Medication During Long Term Administration of Berotralstat
Timepoint [4] 0 0
Up to 96 weeks (US) / 216 weeks (ROW)

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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)

Study design
Purpose of the study
Prevention
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/02/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
27/04/2022
Sample size
Target
Accrual to date
Final
387
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Study Center - Adelaide
Recruitment hospital [2] 0 0
Study Center - Campbelltown
Recruitment hospital [3] 0 0
Study Center - Camperdown
Recruitment hospital [4] 0 0
Study Center - Melbourne
Recruitment hospital [5] 0 0
Study Center - Murdoch
Recruitment hospital [6] 0 0
Study Center - Nedlands
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Campbelltown
Recruitment postcode(s) [3] 0 0
- Camperdown
Recruitment postcode(s) [4] 0 0
- Melbourne
Recruitment postcode(s) [5] 0 0
- Murdoch
Recruitment postcode(s) [6] 0 0
- Nedlands
Recruitment outside Australia
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United States of America
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Alabama
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Arizona
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Connecticut
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Florida
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Georgia
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Illinois
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Indiana
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Maryland
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Massachusetts
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Michigan
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Mississippi
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Missouri
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Nebraska
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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Rhode Island
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Washington
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Wisconsin
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Austria
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Graz
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Austria
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Vienna
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Odense
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Germany
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Frankfurt
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Germany
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Ulm
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Hong Kong
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Central
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Hungary
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Budapest
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Israel
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Ashkelon
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Israel
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Haifa
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Israel
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Israel
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Tel HaShomer
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Milan
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Italy
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Padova
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Italy
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Salerno
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Korea, Republic of
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Daegu
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Donggu
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Gyeonggi-do
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Seoul
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Auckland
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Wellington
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North Macedonia
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Skopje
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Poland
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Kraków
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Serbia
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Belgrade
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Serbia
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Niš
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Martin
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Birmingham
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Bristol
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Cambridge
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London
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Plymouth
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United Kingdom
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Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
BioCryst Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Henriette Farkas, MD
Address 0 0
Semmelweis University, Budapest, Hungary
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03472040