NCT00037557

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Sep 2002

Longer than P75 for phase_3

Geographic Reach
1 country

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 17, 2002

Completed
3 days until next milestone

First Posted

Study publicly available on registry

May 20, 2002

Completed
3 months until next milestone

Study Start

First participant enrolled

September 1, 2002

Completed
5.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2007

Completed
Last Updated

December 27, 2007

Status Verified

December 1, 2007

First QC Date

May 17, 2002

Last Update Submit

December 18, 2007

Conditions

Hemophilia B

Keywords

Hemophilia B

Outcome Measures

Primary Outcomes (1)

  • 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.

Secondary Outcomes (1)

  • To measure the incremental recovery of rFIX in children following a 75-IU/kg bolus infusion.

Interventions

BeneFIXDRUG

Eligibility Criteria

AgeUp to 5 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • A currently detectable FIX inhibitor. A family history of inhibitors will not exclude the patient.
  • Impaired liver function
  • Impaired renal function

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Unknown Facility

Aurora, Colorado, 80262, United States

Location

Unknown Facility

Detroit, Michigan, 48201, United States

Location

Unknown Facility

New Brunswick, New Jersey, 08903, United States

Location

Unknown Facility

Chapel Hill, North Carolina, 27599-7220, United States

Location

Unknown Facility

Dayton, Ohio, 45404, United States

Location

Unknown Facility

Houston, Texas, 77030, United States

Location

Related Publications (2)

  • 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: 40750723DERIVED

  • 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: 26196195DERIVED

MeSH Terms

Conditions

Hemophilia B

Interventions

Factor IX

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Enzyme PrecursorsEnzymes and CoenzymesBlood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Study Officials

  • Medical Monitor, MD

    Wyeth is now a wholly owned subsidiary of Pfizer

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

May 17, 2002

First Posted

May 20, 2002

Study Start

September 1, 2002

Study Completion

November 1, 2007

Last Updated

December 27, 2007

Record last verified: 2007-12

Locations

US(6)