Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

Hemophilia A Clinical Trial

Official 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01775618
• Other study ID #: 15912
• Secondary ID: 2012-004434-42
• Status: Completed
• Phase: Phase 3
• Start date: May 29, 2013
• Est. completion date: February 19, 2020

Study information

• Verified date: August 2020
• Source: Bayer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 73
• Est. completion date: February 19, 2020
• Est. primary completion date: March 19, 2015
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 12 Years

Eligibility

Inclusion Criteria:

• Males < 12 years of age

• Subjects with severe hemophilia A

• Previously treated with FVIII for > 50 exposure days

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

Related Conditions & MeSH terms

• Hemophilia A

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Bayer

Countries where clinical trial is conducted

United States,  Argentina,  Austria,  Belgium,  Bulgaria,  Canada,  Greece,  Israel,  Italy,  Lithuania,  Netherlands,  New Zealand,  Norway,  Poland,  Romania,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annualized number of all bleeds		At least 50 exposure days (ED) over 6 months, on average 245 days
Primary	Pharmacokinetics profile of BAY94-9027 based on blood concentration over the defined time period	Pharmacokinetics profile includes maximum concentration (Cmax), half-life (t1/2), area under the concentration versus time curve (AUC), mean residence time (MRT), volume of distribution at steady state (Vss), and clearance (CL)	Pre-dose to 72 hours post-dose
Primary	Response of acute bleeding events to treatment based on a 4-point scale (poor, moderate, good, or excellent)		At least 50 exposure days (ED) over 6 months, on average 245 days
Primary	Characterization of a potential immune response		12 weeks
Primary	Inhibitor development in the extension study		At least 50 additional EDs to achieve at least 100 cumulative EDs, on average 5 years
Secondary	Inhibitor development in the main study		After 10 to 15 and 50 exposure days (ED) over 6 months, on average 245 days
Secondary	Assessment of incremental recovery in main study		At least 50 exposure days (ED) over 6 months, on average 245 days
Secondary	Number of participants with adverse events as a measure of safety and tolerability		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)

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