Clinical trial NCT00157040: Study of Pharmacokinetics, Efficacy, and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients With Hemophilia A
Official title: Recombinant Antihemophilic Factor (rAHF-PFM) Manufactured and Formulated Without Added Human or Animal Proteins: Evaluation of Pharmacokinetics, Immunogenicity, Efficacy and Safety in Previously Treated Pediatric Patients With Hemophilia A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Hemophilia A
Keywords: Factor VIII Deficiency
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2005-09-12
Enrollment: 50
Sex: ALL
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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:

  1. platelet count \< 100,000/mm3
  2. hemoglobin concentration \< 10 g/dL (100 g/L)
  3. serum creatinine \> 1.5 times the ULN for age
  4. 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

Primary outcomes:
measure: The terminal phase half-life of Recombinant Antihemophilic Factor (rAHF-PFM)
timeFrame: Within 30 minutes prior to the pharmacokinetic infusion and at 1 hour ± 5 minutes, 9 ± 1 hour, 24 ± 2 hours, and 48 ± 2 hours after the infusion
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children´s Hospital Los Angeles
state: California
city: Atlanta
country: United States
facility: Emory University, Department of Pediatrics
state: Georgia
city: Chicago
country: United States
facility: Children´s Memorial Hospital
state: Illinois
city: Peoria
country: United States
facility: Comprehensive Bleeding Disorders Center
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Hemophilia and Thrombosis Center
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics, Iowa Regional Hemophilia Center, Department of Pediatrics
state: Iowa
city: Ann Arbor
country: United States
facility: University of Michigan Hemophilia Treatment Center
state: Michigan
city: Detroit
country: United States
facility: Children´s Hospital of Michigan
state: Michigan
city: Philadelphia
country: United States
facility: Children´s Hospital of Philadelphia
state: Pennsylvania
city: Houston
country: United States
facility: University of Texas Health Science Center
state: Texas
city: Toronto
country: Canada
facility: Hospital for Sick Children, Division of Hematology/Oncology
state: Ontario
city: San Juan
country: Puerto Rico
facility: University Pediatric Hospital
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Shapiro A, Gruppo R, Pabinger I, Collins PW, Hay CR, Schroth P, Casey K, Patrone L, Ehrlich H, Ewenstein BM. Integrated analysis of safety and efficacy of a plasma- and albumin-free recombinant factor VIII (rAHF-PFM) from six clinical studies in patients with hemophilia A. Expert Opin Biol Ther. 2009 Mar;9(3):273-83. doi: 10.1517/14712590902729392.