Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients

Baxalta

Status and phase: Completed; Phase 3

Conditions: Hemophilia A

Treatments: Biological: Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)

Study type: Interventional

Funder types: Industry

Identifiers

NCT00157157

060103

2004-001623-38 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII (FVIII) before.

Enrollment: 66 patients

Sex: All

Ages: Under 6 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• The subject has severe or moderately severe hemophilia A as defined by a baseline factor VIII level <= 2% of normal, as documented at screening
• The subject is < 6 years of age
• The subject's legally authorized representative has provided written informed consent

Exclusion criteria

• The subject has a history of exposure to factor VIII other than rAHF PFM or more than 3 infusions of commercially available rAHF PFM (i.e., ADVATE) within 28 days prior to screening, as determined by the subject's medical history. Any infusion of factor VIII replacement products prior to the 28-day period excludes the subject from participation

• The subject has received more than 3 infusions of rAHF PFM (commercially available and/or study product) between screening and prior to the initial recovery infusion

• The subject has a detectable inhibitor to factor VIII, as measured in the screening sample by the Nijmegen assay in the central laboratory

• The subject has a history of inhibitor to factor VIII at any time prior to screening

• The subject has a known hypersensitivity to rAHF PFM

• The subject has any 1 of the following laboratory abnormalities at the time of screening:

  1. Platelet count < 100,000/mm^3
  2. Hemoglobin concentration < 10 g/dL (100 g/L)
  3. Serum creatinine > 1.5 times the upper limit of normal (ULN) for age
  4. Total bilirubin > 2 times the ULN for age

• The subject has an inherited or acquired hemostatic defect other than hemophilia A (e.g., qualitative platelet defect or von Willebrand's disease)

• The subject is known to be seropositive for human immunodeficiency virus (HIV), hepatitis C virus (HCV), or hepatitis B virus (HBV), as determined by the subject's medical history

• At the time of enrollment, the subject has a clinically significant chronic disease other than hemophilia A

• The subject is currently participating in another investigational drug study, or has participated in any clinical study involving an investigational drug within 120 days of the screening visit

• The subject (or the subject's legally authorized representative) is identified by the investigator as being unable or unwilling to cooperate with study procedures

• The subject has received any blood product, including packed red blood cells (RBC), platelets, plasma, or cryoprecipitate

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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66 participants in 1 patient group

Single Arm - All Participants

Experimental group

Description:

All subjects enrolled in the study who meet the eligibility criteria.

Treatment:

Biological: Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)