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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02870972




Registration number
NCT02870972
Ethics application status
Date submitted
10/08/2016
Date registered
18/08/2016

Titles & IDs
Public title
Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema
Scientific 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
Secondary ID [1] 0 0
BCX7353-203
Universal Trial Number (UTN)
Trial acronym
APeX-1
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema (HAE) 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: Part 1: BCX7353 350 mg once daily - BCX7353 capsules, 350 mg dose administered once per day for 28 days

Experimental: Parts 2 and 3: BCX7353 250 mg once daily - BCX7353 capsules, 250 mg dose administered once per day for 28 days

Experimental: Parts 2 and 3: BCX7353 125 mg once daily - BCX7353 capsules, 125 mg dose administered once per day for 28 days

Placebo comparator: Parts 1, 2 and 3: Placebo - Placebo capsules, administered once per day for 28 days

Experimental: Part 3: BCX7353 62.5 mg once daily - BCX7353 capsules, 62.5 mg dose administered once per day for 28 days


Treatment: Drugs: BCX7353
Plasma kallikrein inhibitor
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Confirmed HAE Attacks
Timepoint [1] 0 0
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).
Primary outcome [2] 0 0
Proportion of Subjects Who Were HAE Attack-free During the Entire Dosing Period
Timepoint [2] 0 0
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 outcome [1] 0 0
Number of Confirmed Abdominal HAE Attacks
Timepoint [1] 0 0
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 outcome [2] 0 0
Number of Confirmed Peripheral HAE Attacks
Timepoint [2] 0 0
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 outcome [3] 0 0
HAE Attacks Requiring Treatment
Timepoint [3] 0 0
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 outcome [4] 0 0
HAE Disease Activity - Modified Angioedema Activity Score
Timepoint [4] 0 0
28-day treatment period + 1 day
Secondary outcome [5] 0 0
Angioedema Quality of Life (AE-QoL)
Timepoint [5] 0 0
The subject-completed AE-QoL was administered at baseline (Day 1) and at Day 29
Secondary outcome [6] 0 0
DASS (Depression, Anxiety and Stress Scales)
Timepoint [6] 0 0
The DASS was administered at baseline (Day 1), Day 14, and Day 29.

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Prevention
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
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
1/08/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/08/2017
Sample size
Target
Accrual to date
Final
75
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Adelaide
Recruitment hospital [2] 0 0
- Campbelltown
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Campbelltown
Recruitment outside Australia
Country [1] 0 0
Austria
State/province [1] 0 0
Graz
Country [2] 0 0
Austria
State/province [2] 0 0
Vienna
Country [3] 0 0
Canada
State/province [3] 0 0
Quebec City
Country [4] 0 0
Canada
State/province [4] 0 0
Toronto
Country [5] 0 0
Denmark
State/province [5] 0 0
Odense
Country [6] 0 0
Germany
State/province [6] 0 0
Berlin
Country [7] 0 0
Germany
State/province [7] 0 0
Frankfurt
Country [8] 0 0
Germany
State/province [8] 0 0
Ulm
Country [9] 0 0
Hungary
State/province [9] 0 0
Budapest
Country [10] 0 0
Italy
State/province [10] 0 0
Milano
Country [11] 0 0
Italy
State/province [11] 0 0
Padova
Country [12] 0 0
Italy
State/province [12] 0 0
Salerno
Country [13] 0 0
North Macedonia
State/province [13] 0 0
Skopje
Country [14] 0 0
Spain
State/province [14] 0 0
Barcelona
Country [15] 0 0
Spain
State/province [15] 0 0
Madrid
Country [16] 0 0
Spain
State/province [16] 0 0
Sevilla
Country [17] 0 0
Switzerland
State/province [17] 0 0
Zürich
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Brimingham
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Bristol
Country [20] 0 0
United Kingdom
State/province [20] 0 0
London
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Oxford
Country [22] 0 0
United Kingdom
State/province [22] 0 0
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
Emel Aygören-Pürsün, MD
Address 0 0
University Hospital Frankfurt Goethe University
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)?
Yes
What data in particular will be shared?
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?


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/NCT02870972