A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B

NCT ID: NCT01662531

Last Updated: 2016-05-09

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

PHASE3

Total Enrollment

27 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-01-31

Brief Summary

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This study will examine the pharmacokinetics, safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children who have previously received factor replacement therapy for hemophilia B.

Detailed Description

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Conditions

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Hemophilia B

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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rIX-FP

Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) will be administered by IV infusion as routine weekly prophylaxis and episodic treatment for bleeding episodes.

Group Type EXPERIMENTAL

rIX-FP

Intervention Type BIOLOGICAL

Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP)

Interventions

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rIX-FP

Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP)

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Male subjects, younger than 12 years old.
* Severe hemophilia B (Factor IX \[FIX\] activity of ≤ 2%).
* Body weight ≥ 10 kg.
* Subjects who have received FIX products (plasma-derived and/or recombinant FIX) for \> 150 exposure days (EDs) (6 to \< 12 years), and \> 50 EDs (\< 6 years).
* No history of FIX inhibitor formation, no detectable inhibitors at Screening and no family history of inhibitors against FIX.
* Written informed consent for study participation.

Exclusion Criteria

* Known hypersensitivity to any FIX product or hamster protein.
* Known congenital or acquired coagulation disorder other than congenital FIX deficiency.
* Kidney or liver disease.
* Recent life-threatening bleeding episode.
Maximum Eligible Age

11 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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CSL Behring

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Program Director

Role: STUDY_DIRECTOR

CSL Behring

Locations

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The Royal Children's Hospital, Melbourne

Parkville, Victoria, Australia

Site Status

The Children's Hospital at Westmead

Westmead, , Australia

Site Status

AKH Wien (Paediatrics)

Vienna, , Austria

Site Status

McMaster Children's Hospital

Hamilton, Ontario, Canada

Site Status

Fakultni nemocnice Brno

Brno, , Czechia

Site Status

Fakultni nemocnice Ostrava

Ostrava, , Czechia

Site Status

Fakultni nemocnice Motole

Prague, , Czechia

Site Status

C.R.T.H. Hopital de Bicentre (Hemophilie)

Le Kremlin-Bicentre, , France

Site Status

Hospital Edouard Herriot

Lyon, , France

Site Status

Hôpital d'enfants La Timone

Marseille, , France

Site Status

CRC Coagulation Research Center GmbH

Duisburg/Altstadt, , Germany

Site Status

Universitätsklinikum Düsseldorf

Düsseldorf, , Germany

Site Status

Sheba Medical Center

Tel Litwinsky, , Israel

Site Status

AOU Careggi

Florence, , Italy

Site Status

IRCCS Ospendale Maggiore (Centro emofilia e Trombosi)

Milan, , Italy

Site Status

FGU "Kirov Research Institute of Haemotology and Blood Trans)

Kirov, , Russia

Site Status

H.U. La Paz

Madrid, , Spain

Site Status

Countries

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Australia Austria Canada Czechia France Germany Israel Italy Russia Spain

References

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Alvarez-Roman MT, Merchan RD, Mellado RCR, Jimenez-Yuste V. Switching and increasing prophylaxis regimen with a genetically recombinant fusion of coagulation factor IX and albumin in haemophilia B: a case report. Curr Opin Hematol. 2023 Sep 1;30(5):175-179. doi: 10.1097/MOH.0000000000000775.

Reference Type DERIVED
PMID: 37522479 (View on PubMed)

Kenet G, Chambost H, Male C, Lambert T, Halimeh S, Chernova T, Mancuso ME, Curtin J, Voigt C, Li Y, Jacobs I, Santagostino E; PROLONG-9FP Investigator Study Group. Long-acting recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in children. Results of a phase 3 trial. Thromb Haemost. 2016 Sep 27;116(4):659-68. doi: 10.1160/TH16-03-0179. Epub 2016 Sep 1.

Reference Type DERIVED
PMID: 27583313 (View on PubMed)

Other Identifiers

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2011-006032-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CSL654_3002

Identifier Type: -

Identifier Source: org_study_id

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