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.

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

NL-OMON

Registry ID

NL-OMON45210

Enrollment

Registered

2013-04-22

Start date

2013-09-27

Completion date

Unknown

Last updated

2024-04-29

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

Conditions

Hemophilia A

Interventions

Subjects will receive BAY 94-9027 once or a number of times per week (see section *study design*).

Eligibility

Age

2 Years to 11 Years

Inclusion criteria

Inclusion criteria: - Males 50 exposure days - Subjects in the expansion group

Exclusion criteria

Exclusion criteria: - Subjects with current evidence of or history of inhibitors to FVIII - Any other inherited or acquired bleeding disorder - Platelet counts 2x the upper limit of normal - Aspartate aminotransferase (AST) / Alanine aminotransferase (ALT) > 5x the upper limit of normal

Design outcomes

Primary

Measure	Time frame
Number of bleeding events during prophylactic treatment Assessment of PK, including Cmax, incremental recovery, Mean Residence Time (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.	—

Measure

Time frame

Number of bleeding events during prophylactic treatment Assessment of PK, including Cmax, incremental recovery, Mean Residence Time (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.

—

Secondary

Measure	Time frame
Inhibitor development after 10-15 and 50 ED Assess incremental recovery in all subjects Safety and tolerability assessments	—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)