Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

NCT ID: NCT00037557

Last Updated: 2007-12-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2002-09-30

Study Completion Date

2007-11-30

Brief Summary

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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.

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.

Detailed Description

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Conditions

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Hemophilia B

Keywords

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Hemophilia B

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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BeneFIX

Intervention Type DRUG

Eligibility Criteria

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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
Maximum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Wyeth is now a wholly owned subsidiary of Pfizer

INDUSTRY

Sponsor Role lead

Principal Investigators

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Medical Monitor, MD

Role: STUDY_DIRECTOR

Wyeth is now a wholly owned subsidiary of Pfizer

Locations

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Aurora, Colorado, United States

Site Status

Detroit, Michigan, United States

Site Status

New Brunswick, New Jersey, United States

Site Status

Chapel Hill, North Carolina, United States

Site Status

Dayton, Ohio, United States

Site Status

Houston, Texas, United States

Site Status

Countries

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United States

References

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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.

Reference Type DERIVED
PMID: 40750723 (View on PubMed)

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.

Reference Type DERIVED
PMID: 26196195 (View on PubMed)

Other Identifiers

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3090A1-301

Identifier Type: -

Identifier Source: org_study_id