Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01775618




Registration number
NCT01775618
Ethics application status
Date submitted
23/01/2013
Date registered
25/01/2013
Date last updated
21/08/2020

Titles & IDs
Public title
Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A
Scientific title
A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Pharmacokinetics, Safety, and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children (Age <12 Years) With Severe Hemophilia A
Secondary ID [1] 0 0
2012-004434-42
Secondary ID [2] 0 0
15912
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - BAY94-9027
Treatment: Other - BAY94-9027
Treatment: Other - BAY94-9027

Experimental: Main study - Participants were treated and prophylaxis administered with BAY94-9027 at a dose of 25-60 international units/kilogram (IU/kg) twice per week or 45-60 IU/kg every 5 days or 60 IU/kg every 7 days as an intravenous (IV) infusion as per clinical needs of each subject up to at least 50 exposure days (EDs) and a minimum of at least 6 months.

Experimental: Part 2 (Expansion group) - Participants were administered with BAY94-9027 at a dose of 25-60 IU/kg twice per week for prophylaxis for 12 weeks.

Experimental: Extension study - Participants were treated and prophylaxis administered with BAY94-9027 at a dose of 25- 60 IU/kg twice per week or 45-60 IU/kg every 5 days or 60 IU/kg every 7 days as an IV infusion as per clinical needs of each subject for at least 50 EDs or until marketing authorization of the drug.


Treatment: Other: BAY94-9027
Study drug dosing was adjusted to the clinical needs of each subject in the range of 25-60 IU/kg/administration, intravenous infusion, at least 50 EDs and a minimum of at least 6 months

Treatment: Other: BAY94-9027
Twice per week prophylaxis: 25-60 IU/kg, intravenous infusion, for 12 weeks

Treatment: Other: BAY94-9027
Study drug dosing was adjusted to the clinical needs of each subject in the range of 25-60 IU/kg/administration, intravenous infusion, at least 50 additional EDs to achieve at least 100 cumulative EDs, or until marketing authorization of the drug
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized number of all bleeds
Timepoint [1] 0 0
At least 50 exposure days (ED) over 6 months, on average 245 days
Primary outcome [2] 0 0
Pharmacokinetics profile of BAY94-9027 based on blood concentration over the defined time period
Timepoint [2] 0 0
Pre-dose to 72 hours post-dose
Primary outcome [3] 0 0
Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent)
Timepoint [3] 0 0
At least 50 exposure days (ED) over 6 months, on average 245 days
Primary outcome [4] 0 0
Characterization of a potential immune response
Timepoint [4] 0 0
12 weeks
Primary outcome [5] 0 0
Inhibitor development in the extension study
Timepoint [5] 0 0
At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years
Secondary outcome [1] 0 0
Inhibitor development in the main study
Timepoint [1] 0 0
After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days
Secondary outcome [2] 0 0
Assessment of incremental recovery in main study
Timepoint [2] 0 0
At least 50 exposure days (ED) over 6 months, on average 245 days
Secondary outcome [3] 0 0
Number of participants with adverse events as a measure of safety and tolerability
Timepoint [3] 0 0
From the start of study treatment up to 7 days after the last dose (Main study: on average 245+7 days; Part 2: 12 weeks+7 days; Extension study: on average 5 years+7 days)

Eligibility
Key inclusion criteria
* Males < 12 years of age
* Subjects with severe hemophilia A
* Previously treated with FVIII for > 50 exposure days
Minimum age
No limit
Maximum age
12 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Subjects with current evidence of or history of inhibitors to FVIII
* Any other inherited or acquired bleeding disorder
* Platelet counts < 100,000/mm^3
* Creatinine > 2x the upper limit of normal
* Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) > 5x the upper limit of normal

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
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
29/05/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
19/02/2020
Sample size
Target
Accrual to date
Final
73
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Ohio
Country [4] 0 0
United States of America
State/province [4] 0 0
Pennsylvania
Country [5] 0 0
United States of America
State/province [5] 0 0
Utah
Country [6] 0 0
Argentina
State/province [6] 0 0
Buenos Aires
Country [7] 0 0
Austria
State/province [7] 0 0
Wien
Country [8] 0 0
Belgium
State/province [8] 0 0
Gent
Country [9] 0 0
Belgium
State/province [9] 0 0
Leuven
Country [10] 0 0
Bulgaria
State/province [10] 0 0
Plovdiv
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Bulgaria
State/province [11] 0 0
Sofia
Country [12] 0 0
Bulgaria
State/province [12] 0 0
Varna
Country [13] 0 0
Canada
State/province [13] 0 0
Alberta
Country [14] 0 0
Canada
State/province [14] 0 0
Ontario
Country [15] 0 0
Greece
State/province [15] 0 0
Thessaloniki
Country [16] 0 0
Israel
State/province [16] 0 0
Ramat Gan
Country [17] 0 0
Italy
State/province [17] 0 0
Lombardia
Country [18] 0 0
Italy
State/province [18] 0 0
Sicilia
Country [19] 0 0
Italy
State/province [19] 0 0
Veneto
Country [20] 0 0
Lithuania
State/province [20] 0 0
Vilnius
Country [21] 0 0
Netherlands
State/province [21] 0 0
Amsterdam
Country [22] 0 0
Netherlands
State/province [22] 0 0
Utrecht
Country [23] 0 0
New Zealand
State/province [23] 0 0
Christchurch
Country [24] 0 0
New Zealand
State/province [24] 0 0
Hamilton
Country [25] 0 0
Norway
State/province [25] 0 0
Oslo
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Poland
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Lodz
Country [27] 0 0
Poland
State/province [27] 0 0
Olsztyn
Country [28] 0 0
Romania
State/province [28] 0 0
Bucharest
Country [29] 0 0
Romania
State/province [29] 0 0
Timisoara
Country [30] 0 0
Spain
State/province [30] 0 0
Barcelona
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Tyne And Wear
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Bristol
Country [33] 0 0
United Kingdom
State/province [33] 0 0
Manchester
Country [34] 0 0
United Kingdom
State/province [34] 0 0
Sheffield

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

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Bayer Study Director
Address 0 0
Bayer
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT01775618