NCT01335061

Brief Summary

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Sep 2011

Typical duration for phase_3

Geographic Reach
9 countries

17 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 30, 2011

Completed
14 days until next milestone

First Posted

Study publicly available on registry

April 13, 2011

Completed
5 months until next milestone

Study Start

First participant enrolled

September 1, 2011

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2014

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

May 4, 2015

Completed
Last Updated

June 8, 2015

Status Verified

May 1, 2015

Enrollment Period

2.6 years

First QC Date

March 30, 2011

Results QC Date

March 18, 2015

Last Update Submit

May 12, 2015

Conditions

Hemophilia B

Keywords

Hemophilia Bon-demandprophylaxisnonacog alfaBeneFIXFIX

Outcome Measures

Primary Outcomes (1)

  • Annualized Number of Bleeding Episodes.

    The annualized bleed rate (ABR) or the annualized number of bleeding episodes per year, will be derived for each participant for each treatment period by using the following formula: ABR = number of bleeds / (Days on treatment period / 365.25) The number of bleeds for the ABR calculation includes all bleeds requiring treatment with factor IX product during the time on treatment.

    2 years

Secondary Outcomes (6)

  • Response to On-Demand Treatment for All Bleeding Episodes.

    2 years

  • Number of Nonacog Alfa, Recombinant Factor IX (BeneFIX) Infusions Used to Treat Each Bleeding Episode.

    2 years

  • Number of Breakthrough (Spontaneous/Non-Traumatic) Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX.

    2 years

  • Average Infusion Dose.

    2 years

  • Total Factor Consumption.

    2 years

  • +1 more secondary outcomes

Study Arms (1)

BeneFIX

OTHER
Biological: Nonacog alfa

Interventions

Nonacog alfaBIOLOGICAL

Period 1: During on-demand period, dosing at the discretion of investigator.

Also known as: BeneFIX
BeneFIX

Eligibility Criteria

Age12 Years - 65 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

UMBAL Sveti Georgi, Klinika po hematologia

Plovdiv, 4002, Bulgaria

Location

The Ottawa Hospital

Ottawa, Ontario, K1H 8L6, Canada

Location

University Hospital Center Zagreb

Zagreb, 10000, Croatia

Location

National Blood Centre

Kuala Lumpur, Kuala Lumpur, 50400, Malaysia

Location

Hospital Tengku Ampuan Afzan

Kuantan, Pahang, 25200, Malaysia

Location

Instituto Biomedico de Investigacion A.C.

Aguascalientes, Aguascalientes, 20127, Mexico

Location

Hospital y Clinica OCA

Monterrey, Nuevo León, 64000, Mexico

Location

Nzoz Triclinium

Warsaw, 02-797, Poland

Location

Klinika Hematologii, Nowotworow Krwi i Transplantacji Szpiku

Wroclaw, 50-367, Poland

Location

Singapore General Hospital

Singapore, 169608, Singapore

Location

Eulji University Hospital

Daejeon, 302-799, South Korea

Location

Istanbul Universitesi Cerrahpasa Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bilim Dali

Fatih, Istanbul, 34098, Turkey (Türkiye)

Location

Hacettepe Universitesi Tip Fakultesi Ic Hastaliklari Anabilim Dali Hematoloji Bolumu

Ankara, 06100, Turkey (Türkiye)

Location

Ege Universitesi Tip Fakultesi

Bornova/Izmir, 35100, Turkey (Türkiye)

Location

Gaziantep Universitesi Tip Fakultesi Sahinbey Arastirma ve Uygulama Hastanesi Hematoloji Poliklinigi

Gaziantep, 27300, Turkey (Türkiye)

Location

Erciyes Universitesi Tip Fakultesi

Kayseri, 38039, Turkey (Türkiye)

Location

Erciyes Universitesi Tip Fakultesi Hastaneleri M. Kemal Dedeman Onkoloji Hastanesi

Kayseri̇, Erciyes, 38039, Turkey (Türkiye)

Location

Related Publications (2)

  • Wojciechowski J, Gaitonde P, Hughes JH, Ravva P. Population Modeling of Factor IX Activity Following Administration of Fidanacogene Elaparvovec Gene Therapy in Participants with Hemophilia B. Clin Pharmacokinet. 2025 Oct;64(10):1531-1548. doi: 10.1007/s40262-025-01535-y. Epub 2025 Aug 1.

    PMID: 40750723DERIVED

  • Tortella BJ, Carr ME, Rendo P, Korth-Bradley J, Smith LM, Kavakli K. Once-weekly prophylaxis regimen of nonacog alfa in patients with hemophilia B: an analysis of timing of bleeding event onset. Blood Coagul Fibrinolysis. 2021 Apr 1;32(3):180-185. doi: 10.1097/MBC.0000000000001012.

    PMID: 33631774DERIVED

Related Links

MeSH Terms

Conditions

Hemophilia B

Interventions

Factor IX

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Enzyme PrecursorsEnzymes and CoenzymesBlood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Results Point of Contact

Title
Pfizer ClinicalTrials.gov Call Center
Organization
Pfizer, Inc.
ClinicalTrials.gov_Inquiries@pfizer.com
1-800-718-1021

Study Officials

  • Pfizer CT.gov Call Center

    Pfizer

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 30, 2011

First Posted

April 13, 2011

Study Start

September 1, 2011

Primary Completion

April 1, 2014

Study Completion

April 1, 2014

Last Updated

June 8, 2015

Results First Posted

May 4, 2015

Record last verified: 2015-05

Locations

MY(2)CA(1)KR(1)PL(2)SG(1)ME(2)TU(6)CR(1)BU(1)