A clinical trial investigating safety and efficacy of a long-acting factor VIII in children (age <12 years) with severe hemophila A

A multi-center, phase III, non-controlled, open-label trial to evaluate the pharmacokinetics, safety, and efficacy of BAY 94-9027 for prophylaxis and treatment of bleeding in previously treated children (age <12 years) with severe hemophilia A - PROTECT KIDS

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2012-004434-42-NL

Enrollment

Registered

2013-04-22

Start date

2013-08-27

Completion date

Unknown

Last updated

2020-07-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

severe hemophilia A (<1% FVIII:C) MedDRA version: 17.1 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: BAY 94-9027 (500 IU/vial) Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: Not yet available Current Sponsor code: BAY 94-9027 Other descriptive na

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: 1. Male, age 50 ED with any FVIII concentrate(s) (plasma derived or recombinant) 4. Willingness and ability of subjects and/or parents to complete training in the use of the EPD and to document infusions during the study 5. Written informed consent by parent/legal representative. Assent should be sought from subjects, if appropriate Are the trial subjects under 18? yes Number of subjects for this age range: 50 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. Current evidence of inhibitor to FVIII measured using the Nijmegen-modified Bethesda assay (>0.6 BU/mL) at the time of screening (central laboratory). Subjects should not have received FVIII within 72 h prior to the collection of screening samples and should have had FVIII administered within the prior 2-3 weeks. 2. History or presence of Factor VIII inhibitors. Inhibitor to FVIII is defined as a titer >0.6 BU/mL or clinical history suggestive of inhibitor requiring modification of treatment. (Subjects with a maximum historical titer of 2x upper limit of normal 6. Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) > 5x upper limit of normal 7. Known hypersensitivity to the drug substance, or any of its components (eg, mouse or hamster protein) 8. The subject 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. Subjects who are currently participating in an investigational study in which they are treated with a currently marketed FVIII concentrate are not excluded. Subjects currently treated with BAY 81-8973 may continue treatment with the product up to the start of Visit 1. 9. 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. 10. The subject is identified by the investigator as being unable or unwilling to perform study procedures. 11. Previous assignment to treatment during this study.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate pharmacokinetics, safety, and efficacy of BAY 94-9027 for prophylaxis and treatment of bleeding in previously treated patients with hemophilia A;Secondary Objective: Primary endpoints: - Number of bleeding events during prophylactic treatment - Assessment of PK, including Cmax, incremental recovery, MRT, Apparent volume of distribution at steady state (Vss), half-life, area under the curve (AUC), and clearance, in at least 12 subjects in each age subgroup, with at least 4 pre-selected time points between pre-dose and 72 h post-infusion. - Response of acute bleeding events to treatment will be rated using a 4-point scale (poor, moderate, good, or excellent) by the subject/parent or by the treating physician if the subject is hospitalized ;Primary end point(s): Primary endpoints: - Number of bleeding events during prophylactic treatment - Assessment of PK, including Cmax, incremental recovery, MRT, Apparent volume of distribution at steady state (Vss), half-life, area under the curve (AUC), and clearance, in at least 12 subjects in each age subgroup, with at least 4 pre-selected time points between pre-dose and 72 h post-infusion. - Response of acute bleeding events to treatment will be rated using a 4-point scale (poor, moderate, good, or excellent) by the subject/parent or by the treating physician if the subject is hospitalized ;Timepoint(s) of evaluation of this end point: - Number of bleedings: at the end of treatment visit - Assessment of PK values: at the PK visit - Response to treatment of acute bleedings: end of treatment visit	—

Measure

Time frame

Main Objective: To evaluate pharmacokinetics, safety, and efficacy of BAY 94-9027 for prophylaxis and treatment of bleeding in previously treated patients with hemophilia A;Secondary Objective: Primary endpoints: - Number of bleeding events during prophylactic treatment - Assessment of PK, including Cmax, incremental recovery, MRT, Apparent volume of distribution at steady state (Vss), half-life, area under the curve (AUC), and clearance, in at least 12 subjects in each age subgroup, with at least 4 pre-selected time points between pre-dose and 72 h post-infusion. - Response of acute bleeding events to treatment will be rated using a 4-point scale (poor, moderate, good, or excellent) by the subject/parent or by the treating physician if the subject is hospitalized ;Primary end point(s): Primary endpoints: - Number of bleeding events during prophylactic treatment - Assessment of PK, including Cmax, incremental recovery, MRT, Apparent volume of distribution at steady state (Vss), half-life, area under the curve (AUC), and clearance, in at least 12 subjects in each age subgroup, with at least 4 pre-selected time points between pre-dose and 72 h post-infusion. - Response of acute bleeding events to treatment will be rated using a 4-point scale (poor, moderate, good, or excellent) by the subject/parent or by the treating physician if the subject is hospitalized ;Timepoint(s) of evaluation of this end point: - Number of bleedings: at the end of treatment visit - Assessment of PK values: at the PK visit - Response to treatment of acute bleedings: end of treatment visit

—

Secondary

Measure	Time frame
Secondary end point(s): - Inhibitor development after 10-15 and 50 ED - Assess incremental recovery in all subjects - Safety and tolerability assessments;Timepoint(s) of evaluation of this end point: - Inhibitor development: after 10-15 and after 50 ED of each subject - Safety and tolerability assessments: at the end of treatment visit and FU visit	—

Countries

Argentina, Austria, Belgium, Bulgaria, Canada, European Union, Greece, Israel, Italy, Lithuania, Netherlands, Norway, Poland, Spain, United Kingdom, United States

Contacts

Public ContactBayer Clin. Trials Contact CTP Team

Bayer HealthCare AG

clinical-trials-contact@bayerhealthcare.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026