Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT01335061
First received: March 30, 2011
Last updated: May 28, 2014
Last verified: May 2014
  Purpose

The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.


Condition Intervention Phase
Hemophilia B
Biological: Nonacog alfa
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter, Open-Label Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of Nonacog-Alfa (BeneFIX) In Subjects With Moderately Severe To Severe Hemophilia B (FIX:C</=2%)

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • Annualized number of bleeding episodes [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Assessment scores on a 4-point Response Scale for an on-demand bleeding episode, as assessed by subject/caregiver or investigator/qualified staff. The 4 point scale assessments are Excellent, Good, Moderate or No response. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
  • The number of BeneFIX infusions used to treat each bleed. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
  • The number of breakthrough (spontaneous/non-traumatic) bleeds within 48 hours of a prophylaxis dose of BeneFIX . [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
  • The average infusion dose and total factor consumption. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
  • The number of subjects with incidences of less-than-expected therapeutic effect (LETE) in the absence of confounding factors. There are 3 circumstances in which LETE can occur. In the on-demand or prophylaxis settings and for low recovery of Factor IX. [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]

Enrollment: 25
Study Start Date: September 2011
Study Completion Date: April 2014
Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
BeneFIX Biological: Nonacog alfa
Period 1: During on-demand period, dosing at the discretion of investigator.
Other Name: BeneFIX
Biological: Nonacog alfa
Period 2: During the prophylaxis period, 100 IU/kg once weekly
Other Name: BeneFIX

  Eligibility

Ages Eligible for Study:   12 Years to 65 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Documented history of moderately-severe to severe hemophilia B (FIX activity </=2%).
  • Male subjects, aged 12 years to 65 years.
  • Subjects with at least 100 exposure days (EDs) to factor IX products.
  • Subjects with a minimum of 12 bleeding episodes, 6 of which must be joint bleeds, in the 12-month period before the Screening visit.

Exclusion Criteria:

  • Subjects who have received FIX as a primary or secondary prophylaxis regimen within the last 12 months prior to the Screening visit.
  • Subjects who have had major surgery or an orthopedic surgical procedure within the past 3 months prior to Screening visit.
  • Subjects for which major surgery or orthopedic surgery is planned within the duration of study participation.
  • Subjects with a past history of, or current FIX inhibitor, defined as >ULN (upper limit of normal) of the reporting laboratory.
  • Subjects with a known hypersensitivity to any FIX product or 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: NCT01335061

Locations
Bulgaria
UMBAL Sveti Georgi, Klinika po hematologia
Plovdiv, Bulgaria, 4002
Canada, Ontario
The Ottawa Hospital
Ottawa, Ontario, Canada, K1H 8L6
Croatia
University Hospital Center Zagreb
Zagreb, Croatia, 10000
Korea, Republic of
Eulji University Hospital
Daejeon, Korea, Republic of, 302-799
Malaysia
Hospital Tengku Ampuan Afzan
Kuantan, Pahang, Malaysia, 25200
National Blood Centre
Kuala Lumpur, Malaysia, 50400
Mexico
Hospital y Clinica OCA
Monterrey, Nuevo Leon, Mexico, 64000
Instituto Biomedico de Investigacion A.C.
Aguascalientes, Mexico, 20127
Poland
Nzoz Triclinium
Warszawa, Poland, 02-797
Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku
Wroclaw, Poland, 50-367
Singapore
Singapore General Hospital
Singapore, Singapore, 169608
Turkey
Hacettepe Universitesi Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bolumu
Ankara, Turkey, 06100
Ege Universitesi Tip Fakultesi
Bornova/Izmir, Turkey, 35100
Erciyes Universitesi Tip Fakultsi Ic Hastaliklari ABD Hematoloji BD
Erciyes, Turkey
Gaziantep Universitesi Tip Fakultesi Sahinbey Arastirma ve Uygulama Hastanesi Hematoloji Poliklinigi
Gaziantep, Turkey, 27300
Istanbul Universitesi Cerrahpasa Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bilim Dali
Istanbul, Turkey, 34098
Erciyes Universitesi Tip Fakultesi
Kayseri, Turkey, 38039
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT01335061     History of Changes
Other Study ID Numbers: B1821010, 3090A1-3306
Study First Received: March 30, 2011
Last Updated: May 28, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
Hemophilia B
on-demand
prophylaxis
nonacog alfa
BeneFIX
FIX

Additional relevant MeSH terms:
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on September 18, 2014