Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A

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Baxalta

Status and phase

Completed
Phase 3
Phase 2

Conditions

Hemophilia A

Treatments

Drug: Antihemophilic factor, recombinant, manufactured protein-free

Study type

Interventional

Funder types

Industry

Identifiers

NCT00157040
060101

Details and patient eligibility

About

The purpose of this study is to evaluate whether rAHF-PFM is effective and safe in the treatment of children with hemophilia A. The study consists of 2 parts. Part 1 of the study is a pharmacokinetic evaluation, and Part 2 is an evaluation of efficacy and safety.

Sex

All

Ages

Under 6 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Subject is less than 6 years of age
  • Severe or moderately-severe hemophilia A as defined by a baseline factor VIII level <= 2%, documented at screening or on the basis of historical data (e.g., at hemophilia diagnosis)
  • Documented medical history of at least 50 exposure days for treatment with all other factor VIII products
  • Subject's parent or legally authorized representative has provided informed consent

Exclusion criteria

  • Detectable inhibitor to factor VIII measured in the screening sample by the local or central hemostasis laboratory
  • History of inhibitor to factor VIII at any time prior to screening

Subject has any one of the following laboratory abnormalities at the time of screening:

  • platelet count < 100,000/mm3
  • hemoglobin concentration < 10 g/dL (100 g/L)
  • serum creatinine > 1.5 times the ULN for age
  • total bilirubin > 2 times the ULN for age
  • Subject has an inherited or acquired hemostatic defect other than hemophilia A (e.g., platelet dysfunction secondary to uremia, liver failure, von Willebrand's Disease)
  • Subject has known hypersensitivity to RECOMBINATE rAHF
  • Subject is currently participating in another investigational drug study or has participated in any clinical study involving an investigational drug within 30 days of study entry
  • Subject is identified by the investigator as being unable or unwilling to cooperate with study procedures

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Single Group Assignment

Masking

None (Open label)

Trial contacts and locations

12

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Data sourced from clinicaltrials.gov

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