A study of the pharmacokinetics, efficacy, safety, and immunogenicity of BAX-855 Administered for Prevention of Bleeding in PreviouslyTreated Pediatric Patients with Severe Hemophilia A (a blood clotting disorder).

Phase 1

Conditions: Severe hemophilia A (FVIII<1%)
MedDRA version: 17.0Level: LLTClassification code 10060612Term: Hemophilia ASystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2014-000742-30-RO

Lead Sponsor: Baxter Innovations GmbH

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 66

Inclusion Criteria

1.The subject has severe hemophilia A (FVIII <1%) as determined by the central laboratory.
2.Subject is <12 years old at the time of screening.
3.Subjects aged =6 to <12 years of age have been previously treated with plasma-derived and/or rFVIII concentrate(s) for a minimum of 150 EDs (based on the subjects’ medical records).
4.Subjects <6 years of age have been previously treated with plasma-derived and/or rFVIII concentrate(s) for at least 50 EDs (based on the subjects’ medical records).
5.Subject is human immunodeficiency virus (HIV) negative; or HIV positive with stable disease and CD4+ count of =200 cells/mm3, as confirmed by central laboratory.
6.The subject and/or legal representative accepts prophylactic treatment over a period of 6 months.
7.The subject and/or the legal representative is willing and able to comply with the requirements of the protocol.
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

1.Subject has detectable FVIII inhibitory antibodies (=0.4 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
2.Subject has a history of FVIII inhibitory antibodies (=0.4 BU using the Nijmegen modification of the Bethesda assay or =0.6 BU using the Bethesda assay) at any time prior to screening.
3.Subject has known hypersensitivity towards mouse or hamster proteins, PEG, or Tween 80.
4.Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand’s disease).
5.Subject’s platelet count is <100,000/µL.
6.Subject has severe chronic hepatic dysfunction (eg, =5 times upper limit of normal [ULN] alanine aminotransferase as confirmed by central laboratory at screening, or a documented international normalized ratio >1.5).
7.Subject has severe renal impairment (serum creatinine >1.5 times ULN).
8.Subject is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone >10 mg/day, or a-interferon) other than anti-retroviral chemotherapy.
9.Subject has current or recent (<30 days) use of other PEGylated drugs prior to study participation or is scheduled to use such drugs during study participation.
10.Subject has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
11.Subject has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the Investigator, would affect subject safety or compliance.
12.Subject’s legal representative is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Secondary Objective: 1.To evaluate the PK parameters of BAX 855 in pediatric PTPs <12 years of age.<br>2.To monitor incremental recovery (IR) of BAX 855 over time.<br>3.To evaluate hemostatic efficacy of BAX 855 in the management of acute bleeding episodes and for prophylaxis over a period of 6 months.<br>4.To assess all AEs possibly or probably related to BAX 855.<br>5.To evaluate immunogenicity (binding antibodies to FVIII, BAX 855, PEG, and Chinese hamster ovary [CHO]) and clinically significant changes in routine laboratory parameters (hematology, clinical chemistry, and lipids) and vital signs.<br>6.To evaluate changes in HRQoL and health resource use.;Timepoint(s) of evaluation of this end point: 6 months (±2 weeks) or at least 50 EDs;Main Objective: To evaluate FVIII inhibitory antibodies (=0.6 BU using the Nijmegen modification of the Bethesda assay);Primary end point(s): The primary outcome measure is the incidence of FVIII inhibitory antibodies

Secondary Outcome Measures

Name	Time	Method

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