Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B

NCT00364182 | Completed Phase 3 Results

• 2 other identifiers: 3090A1-400B1821002
• Study Type: interventional
• Target: 50
• Locations: 9 countries
• Sites: 19

Brief Summary

This study is designed to evaluate BeneFIX infused as prophylaxis regimens, compared with BeneFIX administered as an on-demand regimen only. In the trial, subjects will participate in 4 study periods. The first period is a 16-week on-demand treatment, during which subjects will utilize BeneFIX to treat bleeding events episodically only as they occur (i.e., on-demand treatment). At the end of this period, subjects will be randomly assigned to 1 of 2 BeneFIX prophylaxis regimens: either 100 IU/kg once weekly or 50 IU/kg twice weekly, and followed for 16 weeks. At the end of this period, subjects will use on-demand treatment for 8 weeks. The purpose of this 8-week period is to mitigate the potential carry-over effect of the prior prophylaxis regimen on bleeding frequency. Following this 8-week on-demand treatment, subjects will cross-over and receive the alternate study prophylactic regimen for 16 weeks. The primary endpoint is annualized number of bleeding episodes, compared between the first on-demand period and each of the prophylaxis regimens. A comparison of annualized bleeding episodes between the two prophylaxis regimens will also be performed. Subject-reported outcomes will also be collected using a patient diary. At 24 and 48 hours following the onset of each joint bleeding episode, information on pain, sleep, and work will be collected. Additional data on physical functioning will be recorded on the diary at the end of the 16-week on-demand period, and at the end of each prophylaxis period. Information on safety will also be collected. Each subject will participate in this study for approximately 59 weeks (15 months), including a screening period of up to 3 weeks, an initial 16-week on-demand period, two prophylaxis treatment periods of 16 weeks each, separated by an 8-week on-demand treatment period. A modified FIX recovery study will be performed once during each prophylaxis period. The study diary will also be used by subjects to collect secondary endpoints. These endpoints, which are subject-reported outcomes, will be recorded in the diary at 24 and 48 hours following the onset of each joint bleeding episode. Additional data on physical functioning will be recorded on the diary at the end of the 16-week on-demand period, and at the end of each prophylaxis period. Patients will be recruited in the United States, Canada, Europe and Russia.

Conditions

Hemophilia B

Keywords

Hemophilia B; BeneFIX

Outcome Measures

Primary Outcomes (1)

• Annualized Number of Bleeding Episodes

  Annualized bleed rate (ABR) or number of bleeds per year derived for each participant for each treatment regimen by using the following formula: ABR = number of bleeds / (days on treatment regimen / 365.25)

  Baseline up to Week 56

Secondary Outcomes (6)

• Amount of Sleep Measured by Sleep Diary After Hemarthrosis

  24 and 48 hours post-bleed

• Quality of Sleep Measured by Sleep Diary After Hemarthrosis

  24 and 48 hours post-bleed

• Acute Pain After Hemarthrosis

  24 and 48 hours post-bleed

• Health-Related Productivity Questionnaire (HRPQ) Score: Hours Lost From Work or School at 24 Hours Post-bleed

  24 hours post-bleed

• HRPQ Score: Hours Lost From Work or School at 48 Hours Post-bleed

  48 hours post-bleed

Study Arms (2)

A

EXPERIMENTAL

Drug: Recombinant Coagulation Factor IX (BeneFIX)

B

EXPERIMENTAL

Drug: Recombinant Coagulation Factor IX (BeneFIX)

Interventions

Recombinant Coagulation Factor IX (BeneFIX) DRUG

100 IU/kg once weekly then crossover to 50 IU/kg twice weekly

A

Eligibility Criteria

• Age: 6 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Documented history of moderately severe or severe hemophilia B (FIX:C less than or equal to 2%)
• Male subjects, aged 6 years to 65 years.
• Subjects with a minimum of 12 bleeding episodes, 6 of which must be joint bleeds, in the 12 months before screening.

You may not qualify if:

• Subjects currently utilizing FIX primary prophylaxis.
• Subjects with HIV+ who have a CD4 count less than 200
• Subjects with hepatic or renal impairment.
• Prothrombin time or International Normalized Ration more than 1.5 times the upper limit of normal.
• Major surgery or an orthopedic surgical procedure within the past 3 months or is planned within the duration of participation in this study.
• Past history of, or current FIX inhibitor.
• Hypersensitivity to any FIX product or hamster protein.
• Exposure to any investigational drug, except for rFIX-R, or device within 30 days of providing consent/assent (as appropriate) for this study.
• Bleeding disorders other than hemophilia B.
• Concurrent inflammatory disease that in the investigator's judgment could confound the study results.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (19)

Pfizer Investigational Site

Phoenix, Arizona, 85016, United States

Location

Pfizer Investigational Site

Chicago, Illinois, 60612-3833, United States

Location

Pfizer Investigational Site

Chicago, Illinois, 60614, United States

Location

Pfizer Investigational Site

New Brunswick, New Jersey, 08903-0019, United States

Location

Pfizer Investigational Site

Houston, Texas, 77030-4009, United States

Location

Pfizer Investigational Site

Edmonton, Alberta, T6G 2B7, Canada

Location

Pfizer Investigational Site

Edmonton, Alberta, T6G 2C8, Canada

Location

Pfizer Investigational Site

Ottawa, Ontario, K1H 8L6, Canada

Location

Pfizer Investigational Site

Zagreb, 10000, Croatia

Location

Pfizer Investigational Site

Budapest, 1134, Hungary

Location

Pfizer Investigational Site

Castelfranco Veneto (TV), 31033, Italy

Location

Pfizer Investigational Site

Coppito (AQ), 67100, Italy

Location

Pfizer Investigational Site

Bucharest, 011155, Romania

Location

Pfizer Investigational Site

Moscow, 125167, Russia

Location

Pfizer Investigational Site

Saint Petersburg, 191186, Russia

Location

Pfizer Investigational Site

Belgrade, 11000, Serbia

Location

Pfizer Investigational Site

Niš, 18000, Serbia

Location

Pfizer Investigational Site

Madrid, Madrid, 28046, Spain

Location

Pfizer Investigational Site

Seville, Sevilla, 41013, Spain

Location

Related Publications (4)

• 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: 40750723 DERIVED

• Rendo P, Smith L, Lee HY, Shafer F. Nonacog alfa: an analysis of safety data from six prospective clinical studies in different patient populations with haemophilia B treated with different therapeutic modalities. Blood Coagul Fibrinolysis. 2015 Dec;26(8):912-8. doi: 10.1097/MBC.0000000000000359.

  PMID: 26196195 DERIVED

• Valentino LA, Rusen L, Elezovic I, Smith LM, Korth-Bradley JM, Rendo P. Multicentre, randomized, open-label study of on-demand treatment with two prophylaxis regimens of recombinant coagulation factor IX in haemophilia B subjects. Haemophilia. 2014 May;20(3):398-406. doi: 10.1111/hae.12344. Epub 2014 Jan 13.

  PMID: 24418368 DERIVED

• Shafer F, Smith L, Vendetti N, Rendo P, Carr M. Lack of seasonal variation in bleeding and patient-assessed pain patterns in patients with haemophilia B receiving on-demand therapy. Haemophilia. 2014 May;20(3):349-53. doi: 10.1111/hae.12305. Epub 2013 Nov 29.

  PMID: 24286226 DERIVED

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Hemophilia B

Interventions

Factor IX

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Limitations and Caveats

Although the protocol-defined endpoint was days lost from work or school after hemarthrosis, data was collected and reported in hours.

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: RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 14, 2006
• First Posted: August 15, 2006
• Study Start: May 1, 2007
• Primary Completion: October 1, 2010
• Study Completion: October 1, 2010
• Last Updated: October 4, 2011
• Results First Posted: August 23, 2011

Record last verified: 2011-08

Locations

HU (1); IT (2); RO (1); SE (2); RU (2); ES (2); CA (3); CR (1); US (5)