Prophylaxis versus on demand treatment for children with Hemophilia A

Conditions: Patients with Hemophilia A
MedDRA version: 17.1Level: LLTClassification code 10060612Term: Hemophilia ASystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2014-001362-10-Outside-EU/EEA

Lead Sponsor: Bayer HealthCare AG

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: Male

Target Recruitment: 35

Inclusion Criteria

Inclusion Criteria: - Male, aged 2-16yrs - Severe hemophilia A (<1% FVIII:C [Blood Clotting Factor VIII:C] ) - Minimum of at least 50 documented ED (exposure day) prior to enrolment - No measurable inhibitor activity at baseline and history of FVIII inhibitor antibody formation - Parents or legal guardians document, sign, and date informed consent
Are the trial subjects under 18? yes
Number of subjects for this age range: 35
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: - Another bleeding disease that is different from hemophilia A - Known hypersensitivity to the active substance, mouse or hamster protein - Thrombocytopenia (platelet count <100 000/mm3) based on previous medical records

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of this study is to evaluate the effect of three times a week prophylaxis on all bleeds compared to on-demand treatment.;Secondary Objective: 1. Evaluate the effect of prophylaxis on joint bleeds compared to on-demand treatment.<br>2. Evaluate the effect of prophylaxis on joint function (HJHS or PE) compared to on-demand treatment.<br>3. Evaluate safety and tolerability of prophylaxis versus on-demand treatment.;Primary end point(s): Annualized number of all bleeds recorded during 12 weeks of prophylactic treatment in comparison to the annualized number of all bleeds during the 12 weeks on-demand treatment.;Timepoint(s) of evaluation of this end point: Up to 24 weeks

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Annualized number of joint bleeds; Joint function assessment by the Hemophilia Joint Health Score (HJHS) according to the recommendation of the World Federation of Hemophilia (WFH) for Hemophilia Management.;Timepoint(s) of evaluation of this end point: Up to 24 weeks