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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
Country [11] 0 0
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
Country [26] 0 0
Poland
State/province [26] 0 0
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
Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. Hemophilia A causes the clotting process to be slowed and the person experiences bleeds causing serious problems that could lead to disability. The current standard treatment for severe hemophilia A is infusion of FVIII to stop bleeding, or regular scheduled treatment to prevent bleeds from occuring. Due to the short half-life of FVIII, prophylaxis may require treatment as often as every other day.

In this trial safety and efficacy of a long-acting recombinant Factor VIII molecule is being evaluated in 50 male subjects, \< 12 years of age, with severe Hemophilia A. These subjects will receive open label treatment with long-acting rFVIII for approximately 6 months (or longer until 50 exposure days) on a regular schedule at least once every 7-days. Doses and dose intervals may be adapted to the subject's clinical need. A second group of patients will receive open label treatment with the same drug for 12 weeks on a regular schedule of 2x/week. Patients will attend the treatment center for routine blood samples and will be required to keep an electronic diary.

Subjects will be offered participation in an optional extension study to collect observations for at least an additional 50 exposure days.
Trial website
https://clinicaltrials.gov/study/NCT01775618
Trial related presentations / publications
Mancuso ME, Biss T, Fischer K, Maas Enriquez M, Steele M, Wang M, Tseneklidou-Stoeter D, Ahuja S, Kenet G. PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A. Haemophilia. 2021 May;27(3):434-444. doi: 10.1111/hae.14294. Epub 2021 Mar 16.
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01775618