Pharmacokinetic Study of ADVATE Reconstituted in 2 mL Sterile Water for Injection

Baxalta

Status and phase: Completed; Phase 1

Conditions: Hemophilia A

Treatments: Biological: Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM). (Antihemophilic factor is also known as Factor VIII)

Study type: Interventional

Funder types: Industry

Identifiers

NCT00952822

060702

Details and patient eligibility

About

The purpose of this study is to determine the pharmacokinetics and safety of Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) reconstituted in 2 mL sterile water for injection (SWFI) and compare with those of rAHF-PFM reconstituted in 5 mL of SWFI.

Enrollment: 52 patients

Sex: All

Ages: 2 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• The subject or subject's legally authorized representative has provided written informed consent
• The subject has severe hemophilia A as defined by a baseline FVIII activity <= 1% of normal; tested at screening
• The adolescent/adult subject has a documented history of at least 150 exposure days to FVIII concentrates (either plasma-derived or recombinant), and the pediatric subject has at least 50 exposure days
• The subject is >= 12 to <= 65 years of age for the complete pharmacokinetic assessment and >= 2 to < 12 years for the incremental recovery assessment The subject has a Karnofsky performance score > 60
• The subject is human immunodeficiency virus negative (HIV-) or HIV+ with stable CD4 count >= 200 cells/mm³ (CD4 count determined at screening, if necessary)

Exclusion criteria

• The subject has a known hypersensitivity to mouse or hamster proteins or to FVIII concentrates
• The subject has a history of FVIII inhibitors with titer >= 0. 5 BU (Bethesda Assay) or >= 0.4 BU (Nijmegen modification of the Bethesda Assay) any time prior to screening
• The subject has a detectable FVIII inhibitor at screening, >= 0.4 BU (Nijmegen modification of the Bethesda Assay), in the central laboratory
• The subject has severe chronic liver disease as evidenced by, but not limited to, any of the following: International Normalized Ratio (INR) > 1.4, hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices
• The subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (e.g. qualitative platelet defect or Von Willebrand Disease)
• The subject has received another investigational product within 30 days of enrollment
• The subject's clinical condition may require major or moderate surgery (estimated blood loss > 500 mL) during the period of participation in the study
• Subjects with clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance
• The subject is a female of childbearing potential with a positive pregnancy test at screening

Trial design

Primary purpose: Other

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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52 participants in 2 patient groups

1

Experimental group

Description:

ADVATE reconstituted in 2 mL sterile water for infusion

Treatment:

Biological: Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM). (Antihemophilic factor is also known as Factor VIII)

2

Active Comparator group

Description:

ADVATE reconstituted in 5 mL sterile water for infusion

Treatment:

Biological: Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM). (Antihemophilic factor is also known as Factor VIII)