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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03472040
Registration number
NCT03472040
Ethics application status
Date submitted
27/02/2018
Date registered
21/03/2018
Date last updated
18/06/2023
Titles & IDs
Public title
A Long Term Safety Study of BCX7353 in Hereditary Angioedema
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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
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Secondary ID [1]
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BCX7353-204
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Universal Trial Number (UTN)
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Trial acronym
APeX-S
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema
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HAE
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Prophylaxis
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Condition category
Condition code
Cardiovascular
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Diseases of the vasculature and circulation including the lymphatic system
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Blood
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Other blood disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - BCX7353
Experimental: BCX7353 150 mg once daily -
Treatment: Drugs: BCX7353
BCX7353 mg oral capsules administered once daily
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Safety & Tolerability
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Assessment method [1]
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The number and percentage of subjects with treatment-emergent adverse events.
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Timepoint [1]
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Up to 96 weeks (US) / 216 weeks (Rest of World (ROW)).
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Secondary outcome [1]
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Incidence of Acute Attacks of Angioedema in Subjects During Treatment
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Assessment method [1]
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Number of 'adjusted' attacks were assessed. Adjusted attacks included at least 1 symptom of swelling, had a response of 'no' to the diary question, 'In retrospect, could there be an alternative explanation for your symptoms other than an HAE attack (i.e., allergic reaction, viral cold etc.)?', and were considered unique (attack began > 24 hours from the end of the prior attack). Any attack that began within 24 hours from the end of a prior attack was combined with the prior attack.
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Timepoint [1]
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Up to 96 weeks (US) / 216 weeks (ROW)
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Secondary outcome [2]
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The Durability of Response to Treatment
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Assessment method [2]
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To evaluate if the rate of attacks remains consistent (durable) over time, the monthly attack rate was assessed at 0 to 24 weeks, 24 to 48 weeks, 48 to 96 weeks and 96 weeks until the end of the study. Monthly attack rate was defined as the total number of adjusted HAE attacks experienced during the treatment period adjusted for the length of a month (defined as 28 days) and the number of days the subject was on treatment during that month.
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Timepoint [2]
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Up to 96 weeks (US) / 216 weeks (ROW)
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Secondary outcome [3]
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Patient Reported Quality of Life (QoL) During Treatment
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Assessment method [3]
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Quality of Life (QoL) specific to hereditary angioedema (HAE) was assessed at baseline and at each study visit until the end of the study. The questionnaire (i.e. AE-QoL) consisted of 17 questions spanning 4 domains (functioning, fatigue/mood, fear/shame, and nutrition). Each AE-QoL question had 5 answer options (scored 1-5), with lower and higher scores indicting less and more adverse impact, respectively. Per-subject scores for each domain were computed using the appropriate scoring algorithm applied to the question response scores for each domain. Per-subject total scores (including all 4 domains) were similarly computed using the question response scores for all 17 questions. The outputs from the scoring algorithm were normalized on a scale ranging from 0 (less adverse impact) to 100 (most adverse impact). The Mean change from baseline (CFB) in AE-QoL total score over time is presented below.
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Timepoint [3]
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Up to 96 weeks (US) / 216 weeks (ROW)
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Secondary outcome [4]
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Patient's Satisfaction With Medication During Long Term Administration of Berotralstat
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Assessment method [4]
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The Treatment Satisfaction Questionnaire for Medication (TSQM) was completed by subjects at baseline and at each study visit until the end of the study. TSQM scores consisted of 14 items of which 13 items were made up of 3 specific scales (Effectiveness, Side Effects, and Convenience) and 1 global satisfaction scale (Global Satisfaction). At baseline, TSQM questionnaires were completed based on subject's satisfaction with usual medications. At all other time points for collection of TSQM, subjects were asked about their level of satisfaction or dissatisfaction with the study drug. Scale scores were calculated for each scale and were transformed into scores ranging from 0 to 100, with higher scores indicating higher satisfaction. TSQM score and corresponding change from baseline values were calculated at each visit. Note: Subjects in Hong Kong did not complete the TSQM.
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Timepoint [4]
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Up to 96 weeks (US) / 216 weeks (ROW)
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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
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Minimum age
12
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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)
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Study design
Purpose of the study
Prevention
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2/Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
16/02/2018
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
27/04/2022
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Sample size
Target
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Accrual to date
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Final
387
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Study Center - Adelaide
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Study Center - Campbelltown
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Study Center - Camperdown
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Study Center - Murdoch
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Study Center - Nedlands
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- Adelaide
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- Campbelltown
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- Camperdown
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- Melbourne
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Recruitment postcode(s) [5]
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- Murdoch
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Recruitment postcode(s) [6]
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- Nedlands
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Recruitment outside Australia
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London
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Plymouth
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Southampton
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
BioCryst Pharmaceuticals
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Address
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Ethics approval
Ethics application status
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Summary
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).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03472040
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Henriette Farkas, MD
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Address
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Semmelweis University, Budapest, Hungary
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03472040
Download to PDF