Study to compare on-demand treatment to a prophylaxis regimen of BeneFIX in Hemophilia B subjects with a Factor IX activity:=2%

• Register: EUCTR
• Last refreshed on: 10 November 2014
• Main ID: EUCTR2011-000520-15-BG
• Date of registration: 04/10/2011
• Prospective Registration: Yes
• Primary sponsor: Pfizer Inc., 235 East 42nd Street, New York, NY 10017
• Public title: Study to compare on-demand treatment to a prophylaxis regimen of BeneFIX in Hemophilia B subjects with a Factor IX activity:=2%
• Scientific 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%) - Benefix 1010
• Date of first enrolment: 30/11/2011
• Target sample size: 24
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-000520-15
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1

Countries of recruitment

Bulgaria, Canada, Croatia, Greece, Korea, Democratic People's Republic of, Malaysia, Mexico, Poland, Singapore, Turkey

Contacts

• Name: Clinical Trials.gov Call Center
• Affiliation: Pfizer Inc.

• Name: Clinical Trials.gov Call Center
• Affiliation: Pfizer Inc.

Key inclusion & exclusion criteria

Inclusion criteria:
Subject eligibility should be reviewed and documented by an appropriately qualified member of the investigator’s study team before subjects are included in the study.
Subjects must meet all of the following inclusion criteria to be eligible for enrollment into the study:
1. Documented history of moderately severe to severe hemophilia B (FIX activity =2%).
2. Male subjects, aged 12 years to 65 years.
3. Subjects with at least 100 exposure days (EDs) to factor IX products.
4. Subjects with a minimum of 12 bleeding episodes, 6 of which must be joint bleeds, in the 12 month period before the Screening visit.
5. Evidence of a personally signed and dated informed consent/assent document indicating that the subject and if applicable, a legal representative, has been informed of all pertinent aspects of the study.
6. Subjects (and if applicable, their legal guardians), who are willing and able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures.
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 19
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion criteria:
Subjects presenting with any of the following will not be included in the study:
1. Subjects who are investigational site staff members or subjects who are Pfizer employees directly involved in the conduct of the trial.
2. Subjects who have received FIX as a primary or secondary prophylaxis regimen within the last 12 months prior to the Screening visit.
3. For documented HIV+ subjects, CD4 count <200 µL.
4. Subjects with hepatic or renal impairment (ALT and AST =5x Upper limit of normal (ULN), bilirubin >2 mg/dL, albumin 1.25 x ULN).
5. Prothrombin time (PT) >1.5 x ULN or international normalized ratio (INR) >1.5.
6. Platelet count <100,000/µL.
7. Subjects who have had major surgery or an orthopedic surgical procedure within the past 3 months prior to Screening visit.
8. Subjects for which major surgery or orthopedic surgery is planned within the duration of study participation.
9. Subjects with a past history of, or current FIX inhibitor, defined as >ULN of the reporting laboratory.
10. Subjects with a known hypersensitivity to any FIX product or hamster protein.
11. Subjects who received any investigational drug or device within 30 days of Screening visit.
12. Subjects with bleeding disorders other than hemophilia B.
13. Subjects with a documented concurrent inflammatory disease that in the investigator’s judgment could confound the study results (eg, systemic lupus, rheumatoid arthritis or inflammatory bowel disease).
14. Subjects with poor venous access.
15. Participation in other studies (except observational and epidemiological studies) within 30 days before the current study begins.
16. Participation in any other studies during study participation.
17. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Hemophilia B
MedDRA version: 15.1 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850

Intervention(s)

Trade Name: BeneFix
Product Name: Not Applicable
Product Code: Not Applicable
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Nonacog alfa
CAS Number: 181054955
Current Sponsor code: 3090A1
Other descriptive name: recombinant coagulation factor IX
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 250-

Trade Name: BeneFix
Product Name: Not Applicable
Product Code: Not Applicable
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Nonacog alfa
CAS Number: 181054955
Current Sponsor code: 3090A1
Other descriptive name: recombinant coagulation factor IX
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 500-

Trade Name: BeneFix
Product Name: Not Applicable
Product Code: Not Applicable
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Nonacog alfa
CAS Number: 181054955
Current Sponsor code: 3090A1
Other descriptive name: recombinant coagulation factor IX
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 1000-

Trade Name: BeneFix
Product Name: Not Applicable
Product Code: Not Applicable
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Nonacog alfa
CAS Number: 181054955
Current Sponsor code: 3090A1
Other descriptive name: recombinant coagulation factor IX
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 2000-

Primary Outcome(s)

Primary end point(s): Annualized number of bleeding episodes

Timepoint(s) of evaluation of this end point: This will be analyzed during the interim analysis and at the end of the study. There is no set visit that will evaluate annual bleeds

Main Objective: The primary objective is to demonstrate that a prophylaxis regimen of BeneFIX reduces the ABR compared to on demand treatment in subjects with moderately severe to severe hemophilia B.

Secondary Objective: The secondary objective is to describe the safety and efficacy of BeneFIX when administered for prophylaxis at a dose of 100 IU/kg once weekly.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Not Applicable

Secondary end point(s): None

Secondary ID(s)

B1821010

2011-000520-15-PL

Source(s) of Monetary Support

Pfizer Inc.