BAX 326 Pediatric Study

NCT ID: NCT01488994

Last Updated: 2021-05-20

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-12-20

Study Completion Date

2013-05-14

Brief Summary

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The purpose of this study is to assess BAX 326 pharmacokinetic parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life in pediatric patients.

Detailed Description

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The secondary outcome measure: Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours (h) Post-infusion analysis was not done due to the different time-points for the last PK blood sample, AUC0-72 h was redundant and only total AUC was included in the PK analysis.

Conditions

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Hemophilia B

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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BAX326 < 6 years of age

Group Type EXPERIMENTAL

BAX326

Intervention Type BIOLOGICAL

All participants underwent a pharmacokinetic evaluation with BAX326 (recombinant Factor IX) followed by twice weekly prophylactic treatment for 6 months or for at least 50 exposure days, whichever occurred last.

BAX326 6 to <12 years of age

Group Type EXPERIMENTAL

BAX326

Intervention Type BIOLOGICAL

All participants underwent a pharmacokinetic evaluation with BAX326 (recombinant Factor IX) followed by twice weekly prophylactic treatment for 6 months or for at least 50 exposure days, whichever occurred last.

Interventions

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BAX326

All participants underwent a pharmacokinetic evaluation with BAX326 (recombinant Factor IX) followed by twice weekly prophylactic treatment for 6 months or for at least 50 exposure days, whichever occurred last.

Intervention Type BIOLOGICAL

Other Intervention Names

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BAX 326 RIXUBIS

Eligibility Criteria

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Inclusion Criteria

* Participant and/or legal representative has/have voluntarily provided signed informed consent
* Participant has severe (FIX level \< 1%) or moderately severe (FIX level ≤ 2%) hemophilia B
* Participant is \< 12 years old at the time of screening
* Participant has no evidence of a history of FIX inhibitors (based on the participant's medical records)
* Participant is immunocompetent as evidenced by a CD4 count ≥ 200 cells/mm\^3

Exclusion Criteria

* Participant has a detectable FIX inhibitor at screening, with a titer ≥ 0.6 Bethesda Unit (BU)
* Participant has a history of allergic reaction, e.g. anaphylaxis, following exposure to FIX concentrate(s)
* Participant has evidence of an ongoing or recent thrombotic disease
* Participant has an inherited or acquired hemostatic defect other than hemophilia B
Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Baxalta now part of Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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LNJP Maulana Azad Medical College & Associated Hospitals

New Delhi, , India

Site Status

University Pediatric Hospital

Krakow, , Poland

Site Status

Stanislaw Popowski Provincial Specialist Pediatric Hospital

Olsztyn, , Poland

Site Status

Professor Tadeusz Sokolowski Independent Public Teaching Hospital of the Pomeranian Medical University in Szczecin

Szczecin, , Poland

Site Status

S.C. Sanador SRL

Bucharest, , Romania

Site Status

Louis Turcanu Emergency Children's Hospital

Timișoara, , Romania

Site Status

Pediatric Regional Clinical Hospital, Hematology Department

Krasnodar, , Russia

Site Status

Republican Center for Hemophilia Treatment

Saint Petersburg, , Russia

Site Status

Regional Clinical Hospital

Yekaterinburg, , Russia

Site Status

State Institution "Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine"

Lviv, , Ukraine

Site Status

Manchester Children´s Hospital

Manchester, , United Kingdom

Site Status

Countries

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India Poland Romania Russia Ukraine United Kingdom

References

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Urasinski T, Stasyshyn O, Andreeva T, Rusen L, Perina FG, Oh MS, Chapman M, Pavlova BG, Valenta-Singer B, Abbuehl BE. Recombinant factor IX (BAX326) in previously treated paediatric patients with haemophilia B: a prospective clinical trial. Haemophilia. 2015 Mar;21(2):196-203. doi: 10.1111/hae.12548. Epub 2014 Dec 11.

Reference Type RESULT
PMID: 25495591 (View on PubMed)

Other Identifiers

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2011-002437-19

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

251101

Identifier Type: -

Identifier Source: org_study_id

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