- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00037557
Study Evaluating rFIX; BeneFIX in Severe Hemophilia B
An Open-Label, Single-Arm, Safety and Efficacy Study of Recombinant Human Factor IX (rFIX; BeneFIX®) in Children Less Than 6 Years of Age With Severe Hemophilia B
To characterize the safety and efficacy of rFIX in children less than 6 years of age with severe hemophilia B in the setting of acute bleeding episodes, prophylaxis, and/or surgery.
This study will provide an opportunity for systematic observation of treatment with rFIX in children less than 6 years of age regardless of prior FIX treatment. Younger patients exhibit a different pharmacokinetic profile and therefore may respond differently to rFIX infusions when compared with older children and adults. This evaluation will provide data from which recommendations can be made regarding rFIX dosing and treatment of these patients. Surveillance for certain observations that have been made in patients treated with rFIX in the clinical and postmarketing setting will be performed, including inhibitor development, thrombogenicity, FIX recovery/lack of effect, allergic-type manifestations, and RBC agglutination. Comparisons will be derived from published reports and communications describing experience with other FIX products and protein therapeutics in general.
Study Overview
Study Type
Enrollment
Phase
- Phase 3
Contacts and Locations
Study Locations
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Colorado
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Aurora, Colorado, United States, 80262
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Michigan
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Detroit, Michigan, United States, 48201
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New Jersey
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New Brunswick, New Jersey, United States, 08903
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North Carolina
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Chapel Hill, North Carolina, United States, 27599-7220
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Ohio
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Dayton, Ohio, United States, 45404
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Texas
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Houston, Texas, United States, 77030
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Severe hemophilia B
- Less than 5 years of age
- In the investigator's judgment, the patient and/or caregiver will be compliant to study procedures
Exclusion Criteria:
- A currently detectable FIX inhibitor. A family history of inhibitors will not exclude the patient.
- Impaired liver function
- Impaired renal function
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
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To characterize the safety and efficacy of rFIX in children less than 6 years of age with severe hemophilia B in the setting of acute bleeding episodes, prophylaxis, and/or surgery.
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Secondary Outcome Measures
Outcome Measure |
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To measure the incremental recovery of rFIX in children following a 75-IU/kg bolus infusion.
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Collaborators and Investigators
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 3090A1-301
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Hemophilia B
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Catalyst BiosciencesCompletedHemophilia A | Hemophilia B | Hemophilia A With Inhibitor | Hemophilia B With Inhibitor | Hemophilia A Without Inhibitor | Hemophilia B Without InhibitorBulgaria, Russian Federation
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BayerCompletedHemophilia A; Hemophilia BIsrael
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American Thrombosis and Hemostasis NetworkTakeda; CSL Behring; OctapharmaCompletedHemophilia A | Hemophilia B | Hemophilia | Hemophilia A With Inhibitor | Haemophilia | Hemophilia B With Inhibitor | Haemophilia A Without Inhibitor | Haemophilia B Without InhibitorUnited States
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American Thrombosis and Hemostasis NetworkGenentech, Inc.Active, not recruitingHemophilia A With Inhibitor | Hemophilia B With Inhibitor | Haemophilia A Without Inhibitor | Haemophilia B Without InhibitorUnited States
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Laboratoire français de Fractionnement et de BiotechnologiesLFB USA, Inc.CompletedA Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (PERSEPT2)Hemophilia A With Inhibitors | Hemophilia B With InhibitorsBulgaria, Ukraine, Czechia, United States, Georgia, South Africa
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University College, LondonRecruiting
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University of British ColumbiaBiogenCompletedHemophilia A, Congenital | Hemophilia B, CongenitalCanada
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Suzhou Alphamab Co., Ltd.RecruitingHemophilia A With Inhibitor | Hemophilia B With InhibitorChina
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AryoGen Pharmed Co.CompletedHemophilia A With Inhibitor | Hemophilia B With InhibitorIran, Islamic Republic of
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Chia Tai Tianqing Pharmaceutical Group Co., Ltd.UnknownHemophilia A With Inhibitor | Hemophilia B With InhibitorChina
Clinical Trials on BeneFIX
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PfizerCompleted
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PfizerCompleted
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PfizerCompletedHemophilia BGermany, Austria
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PfizerCompletedHemophilia BCanada, Singapore, Turkey, Croatia, Korea, Republic of, Mexico, Poland, Bulgaria, Malaysia
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PfizerCompleted
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Grifols Biologicals, LLCCompleted
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PfizerCompletedHemophilia BKorea, Republic of
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Wyeth is now a wholly owned subsidiary of PfizerCompleted
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PfizerCompletedHemophilia BUnited States, Spain, Croatia, Serbia, Russian Federation, Canada, Hungary, Italy, Romania