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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05147662




Registration number
NCT05147662
Ethics application status
Date submitted
24/11/2021
Date registered
7/12/2021

Titles & IDs
Public title
A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood
Scientific title
A Phase 3, Single Group Treatment, Open-label, Study to Evaluate the Safety of BAY 94-9027 Infusions for Prophylaxis and Treatment of Bleeding in Previously Treated Children Aged 7 to <12 Years With Severe Hemophilia A
Secondary ID [1] 0 0
2021-004858-30
Secondary ID [2] 0 0
21824
Universal Trial Number (UTN)
Trial acronym
Alfa-PROTECT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Treatment of Bleeding 0 0
Prophylaxis of Bleeding 0 0
Hemophilia A 0 0
Children 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 - Damoctocog alfa pegol (Jivi, BAY94-9027)

Experimental: Main study (Part A) and the extension study (Part B) - Part A will last for 6 months. After completing Part A participants will continue in the extension study for another 18 months.


Treatment: Other: Damoctocog alfa pegol (Jivi, BAY94-9027)
Part A: 40 IU/kg (up to 60 IU/kg at the investigator's discretion), two times per week (2x/week) with the first 4 infusions under medical supervision. Thereafter, participants will continue their treatment as home treatment. Dose may be increased up to 60 IU/kg if needed at any time during the study at the investigator's discretion.

Part B: Each participant may continue on prophylaxis dose regimen as prescribed in part A (40 - 60 IU/kg, 2x per week) or adjustments to prophylaxis dose / dose frequency can be made at the investigator's discretion (based on the bleeding events and individual needs): Dose frequency may be decreased to every 5 days with a prophylaxis dose of 60 IU/kg.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
AESI (hypersensitivity and loss of efficacy) associated with the first 4 exposure days leading to discontinuation
Timepoint [1] 0 0
Up to 6 months
Secondary outcome [1] 0 0
Adverse drug reactions (ADRs)
Timepoint [1] 0 0
Up to 6 months
Secondary outcome [2] 0 0
Anti-drug antibody (ADA) development
Timepoint [2] 0 0
Pre-infusion and up to 6 months
Secondary outcome [3] 0 0
The number of participants with confirmed Factor VIII inhibitors
Timepoint [3] 0 0
Pre-infusion and up to 6 months
Secondary outcome [4] 0 0
Annualized bleeding rate (ABR)
Timepoint [4] 0 0
Up to 24 months
Secondary outcome [5] 0 0
BAY94-9027 consumption
Timepoint [5] 0 0
Up to 24 months
Secondary outcome [6] 0 0
Number of infusions/month and year (Annualized Infusion Rate)
Timepoint [6] 0 0
Up to 24 months

Eligibility
Key inclusion criteria
* Participants with severe hemophilia A (participant's own FVIII activity [FVIII:C] <1%)
* Participants must be previously treated with FVIII concentrate(s) (plasma derived or recombinant) for a minimum of 50 exposure days (EDs) at the time of signing the informed consent
* Participant has understood the study if appropriate for his age, informed consent must be signed by the parent, the participant can only sign the assent
* Willingness and ability of participants and/or parents /caregivers to complete training in the use of the electronic patient diary (EPD) and to document infusions during the study
Minimum age
7 Years
Maximum age
11 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* History of FVIII inhibitors
* Current evidence of inhibitor to FVIII measured using the Nijmegen-modified Bethesda assay (>0.6 BU/mL) at the time of screening (central laboratory)
* Any other inherited or acquired bleeding disorder in addition to hemophilia A (e.g. von Willebrand disease, hemophilia B)
* Known hypersensitivity or allergic reaction to drug substance, excipients or mouse or hamster protein
* Any other significant medical condition that the investigator feels would be a risk to the patient or would impede the study
* Requires any pre-medication to tolerate FVIII treatment (e.g. antihistamines)
* Planned major surgery during the study
* Any individual who is receiving chemotherapy, immune modulatory drugs other than anti-retroviral chemotherapy, or chronic use of oral or intravenous (IV) corticosteroids (> 14 days) within the last 3 months
* Any individual who received commercially available subcutaneous factor substitution therapy (emicizumab) within the last 6 months
* The participant is currently participating in another investigational drug study or has participated in a clinical study involving an investigational drug within 30 days of study entry or previous participation in a clinical study with BAY94-9027

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
23/03/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
10/07/2025
Actual
Sample size
Target
33
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
Argentina
State/province [2] 0 0
Buenos Aires
Country [3] 0 0
Argentina
State/province [3] 0 0
Ciudad Auton. De Buenos Aires
Country [4] 0 0
Argentina
State/province [4] 0 0
Mendoza
Country [5] 0 0
Argentina
State/province [5] 0 0
Santa Fe
Country [6] 0 0
Brazil
State/province [6] 0 0
RJ
Country [7] 0 0
Brazil
State/province [7] 0 0
Sao Paulo
Country [8] 0 0
Brazil
State/province [8] 0 0
Multiple Locations
Country [9] 0 0
Canada
State/province [9] 0 0
Alberta
Country [10] 0 0
Canada
State/province [10] 0 0
Ontario
Country [11] 0 0
Canada
State/province [11] 0 0
Multiple Locations
Country [12] 0 0
Italy
State/province [12] 0 0
Campania
Country [13] 0 0
Italy
State/province [13] 0 0
Lazio
Country [14] 0 0
Norway
State/province [14] 0 0
Oslo
Country [15] 0 0
Turkey
State/province [15] 0 0
Adana
Country [16] 0 0
Turkey
State/province [16] 0 0
Ankara
Country [17] 0 0
Turkey
State/province [17] 0 0
Antalya
Country [18] 0 0
Turkey
State/province [18] 0 0
Gaziantep
Country [19] 0 0
Turkey
State/province [19] 0 0
Izmir
Country [20] 0 0
Turkey
State/province [20] 0 0
Multiple Locations
Country [21] 0 0
Turkey
State/province [21] 0 0
Samsun

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
Address 0 0
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
Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.


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/NCT05147662