Study Evaluating Safety And Efficacy Of Moroctocog Alfa (AF-CC) In Previously Treated Hemophilia A Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00914459
First received: June 4, 2009
Last updated: May 10, 2016
Last verified: May 2016
  Purpose
The study will be investigating pharmacokinetics, safety and efficacy in patients less than 12 years of age with severe hemophilia A that have been previously treated with Factor VIII products ( including blood products).

Condition Intervention Phase
Hemophilia A
Biological: Moroctocog alfa ( AF-CC)
Procedure: Laboratory tests
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Non-randomized, Open-label Study To Evaluate The Pharmacokinetics, Safety And Efficacy Of Refacto Af In Previously Treated Pediatric Subjects Less Than Twelve Years Of Age With Severe Hemophilia A (Fviii:c <1%).

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • Incidence of clinically significant FVIII inhibitor development. [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Annualized bleeding rates (ABRs) in subjects receiving treatment with ReFacto AF. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Responses to the first on-demand treatment with ReFacto AF for all new bleeds (4 point scale of assessment) as assessed by the parent/legal representative. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Number of ReFacto AF infusions to treat each new bleed. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Number of bleeds within 48 hours of a preventive/prophylaxis dose of ReFacto AF. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Average infusion dose and total factor consumption, and the incidence of less-than-expected therapeutic effect (LETE). [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Incidence of SAEs and nonserious AEs. [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]

Enrollment: 38
Study Start Date: December 2009
Study Completion Date: April 2016
Primary Completion Date: March 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Moroctocog alfa (AF-CC)
Open Label
Biological: Moroctocog alfa ( AF-CC)
Dosing is at the discretion of the Investigator
Other Name: ReFacto AF
Procedure: Laboratory tests
Factor VIII PK samples, Hematology, Chemistry and Coagulation testing, FactorVIII Inhibitor and Anti Factor VIII antibody

  Eligibility

Ages Eligible for Study:   up to 11 Years   (Child)
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male subjects less than 12 years of age with a documented history of severe hemophilia A (FVIII:C less than 1%).
  • Subjects who are less than 6 years of age must have had at least 50 Exposure Days (EDs) to prior FVIII products (including blood products).
  • Subjects who are equal to or greater than 6 years of age must have had greater than 150 EDs to prior FVIII products (including blood products).

Exclusion Criteria:

  • For laboratory assessments, any measured Bethesda inhibitor titer equal to or greater than 0.6 BU, regardless of the laboratory normal range, or any Bethesda inhibitor titer greater than ULN for the testing laboratory at the time of screening.
  • Any other bleeding disorder in addition to hemophilia A.
  • Treatment with any investigational drug or device within 30 days before the time of signing the parental informed consent/assent form.
  • Major surgery planned to occur during the course of the study.
  • Regular (e.g., daily; every other day) use of agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDS).
  • Regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic corticosteroids), or currently receiving immune tolerance induction (ITI) for inhibitor treatment.
  • The subject is receiving treatment for HIV or hepatitis infection (unless the subject is on a stable antiviral regimen [i.e., consistent treatment regimen for at least 3 months before the parental informed consent/assent form is signed]).
  • Platelet count less than 100,000/µL.
  • Prothrombin time (PT) equal to or greater than 1.25 x ULN, or international normalized ratio (INR) equal to or greater than 1.5.
  • Known hypersensitivity to hamster protein.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00914459

Locations
Finland
Kuopio University Hospital
Kuopio, Finland, 70211
Georgia
LTD Medinvesti- Institute of hematology and transfusiology
Tbilisi, Georgia, 0186
Italy
Centro di Riferimento Regionale per la cura dell'Emofilia e delle Malattie Emorragiche Congenite
Parma, Italy, I-43100
Romania
Spitalul Clinic Judetean de Urgenta Craiova
Craiova, Dolj, Romania, 200642
Sanador
Bucharest, Romania, 011026
Serbia
University Children's Hospital
Belgrade, Serbia, 11000
Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic"
Belgrade, Serbia, 11070
Spain
Hospital Jerez de la Frontera
Jerez de la Frontera, Cádiz, Spain, 11407
Hospital Universitario Puerta del Mar
Cadiz, Spain, 11009
Hospital La Paz
Madrid, Spain, 28046
Hospital Universitario Miguel Servet
Zaragoza, Spain, 50009
Sweden
Karolinska Universitetssjukhuset-Solna
Stockholm, Sweden, 171 76
Turkey
Cukurova University Department of Pediatrics, Pediatric Hematology Division
Adana, Balcali/adana, Turkey, 01330
Ege University Department of Pediatrics, Pediatric Hematology Division
Izmir, Bornova /izmir, Turkey, 35100
Akdeniz Universitesi Tip Fakultesi
Antalya, Kampus, Turkey, 07059
Ukraine
Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii medych
Lviv, Ukraine, 79044
Komunalna ustanova "Zaporizka oblasna klinichna dytiacha likarnia"
Zaporizhzhia, Ukraine, 69063
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00914459     History of Changes
Other Study ID Numbers: 3082B2-4433  B1831005  2008-008435-29 
Study First Received: June 4, 2009
Last Updated: May 10, 2016
Health Authority: European Union: European Medicines Agency

Keywords provided by Pfizer:
hemophilia A

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants

ClinicalTrials.gov processed this record on August 23, 2016