Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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24 January 2022 |
Main ID: |
EUCTR2011-000520-15-GR |
Date of registration:
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27/12/2011 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Study to compare on-demand treatment to a prophylaxis regimen of BeneFIX in Hemophilia B subjects with a Factor IX activity:=2%
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Scientific title:
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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:
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09/01/2012 |
Target sample size:
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24 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-000520-15 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: no Randomised: no Open: no 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
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): no
Therapeutic confirmatory - (Phase III): yes
Therapeutic use (Phase IV): no
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Countries of recruitment
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Argentina
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Bulgaria
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Canada
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Chile
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Colombia
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Croatia
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Greece
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Hungary
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Jordan
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Korea, Democratic People's Republic of
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Malaysia
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Mexico
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Netherlands
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Peru
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Poland
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Portugal
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Romania
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Serbia
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Singapore
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Turkey
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United Kingdom
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United States
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Contacts
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Name:
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Clinical Trials.gov Call Center
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Address:
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235 E 42nd Street
NY 10017
New York
United States |
Telephone:
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0118007181021 |
Email:
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ClinicalTrials.govCallCenter@pfizer.com |
Affiliation:
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Pfizer Inc. |
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Name:
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Clinical Trials.gov Call Center
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Address:
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235 E 42nd Street
NY 10017
New York
United States |
Telephone:
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0118007181021 |
Email:
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ClinicalTrials.govCallCenter@pfizer.com |
Affiliation:
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Pfizer Inc. |
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Key inclusion & exclusion criteria
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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 legally acceptable 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: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 18 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 3
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
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Health Condition(s) or Problem(s) studied
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Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
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Hemophilia B MedDRA version: 14.1
Level: LLT
Classification code 10060614
Term: Hemophilia B (Factor IX)
System Organ Class: 10010331 - Congenital, familial and genetic disorders
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Intervention(s)
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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-
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Primary Outcome(s)
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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.
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Primary end point(s): Annualized number of bleeding episodes
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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.
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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
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Secondary Outcome(s)
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Timepoint(s) of evaluation of this end point: This will be analyzed during the interim analysis and at the end of the study.
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Secondary end point(s): 1. The response to the on demand treatment with BeneFIX for all bleeds (4 point scale of assessment) as assessed by the subject/caregiver or investigator. One assessment per infusion will be required.
2. The number of BeneFIX infusions used to treat each bleed.
3. The number of breakthrough (spontaneous/non traumatic) bleeds within 48 hours of a prophylaxis dose of BeneFIX.
4. The average infusion dose and total factor consumption.
5. The incidence of less than expected therapeutic effect (LETE).
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Secondary ID(s)
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B1821010
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2011-000520-15-PL
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Source(s) of Monetary Support
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Pfizer Inc.
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Ethics review
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Status: Approved
Approval date: 13/12/2011
Contact:
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