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

NCT ID: NCT02053792

Last Updated: 2022-07-14

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 97 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-02-06
• Study Completion Date: 2021-06-02

Brief Summary

This study will examine the long-term safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children and adults with severe hemophilia B. The study will include subjects who have not previously been treated with Factor IX products, subjects who previously completed a CSL-sponsored rIX-FP lead-in study and subjects requiring major non-emergency surgery who have not previously completed a CSL-sponsored rIX-FP lead-in study.

A surgical prophylaxis substudy will examine the efficacy of rIX-FP in subjects with hemophilia B who are undergoing non-emergency major or minor surgery. An additional substudy will examine the safety and PK of subcutaneous (SC) administration of rIX-FP.

Conditions

Hemophilia B

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

rIX-FP

Subjects will administer rIX-FP by intravenous infusion as routine prophylaxis, prevention, and on-demand treatment during a treatment period of approximately 3 years.

The routine prophylaxis treatment interval for previously treated patients may be changed at each scheduled 6-month follow-up assessment. On-demand treatment with rIX-FP will be used for all bleeding episodes requiring treatment. Subjects (other than those in France) may participate in a surgical 'substudy' in which rIX-FP may be administered before, during and after surgery. An additional substudy will examine the safety and PK of subcutaneous administration of rIX-FP.

For previously untreated patients, subjects will administer rIX-FP intravenously as weekly prophylaxis and/or on-demand treatment during the first 12 months, and as weekly routine prophylaxis thereafter.

The dose of rIX-FP administered will be based on the subject's previous rIX-FP use and/or pharmacokinetic data.

Group Type: EXPERIMENTAL

rIX-FP

Intervention Type: BIOLOGICAL

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

Interventions

rIX-FP

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

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

For previously treated subjects, either:

• Completed a CSL-sponsored rIX-FP (CSL654) study, including study CSL654_3001 [NCT01496274] or study CSL654_3002 [NCT01662531].

Or:

• Scheduled to have a major non-emergency surgery within approximately 8 weeks from the anticipated date of receiving the first rIX-FP injection.
• Not previously completed a CSL-sponsored rIX-FP lead-in study.
• Male, 12 to 70 years of age.
• Documented severe hemophilia B (FIX activity of ≤ 2%), or confirmed at screening by the central laboratory.
• Subjects who have received FIX products (plasma-derived and / or recombinant FIX) for > 150 exposure days (EDs), confirmed by their treating physician.
• No confirmed history of FIX inhibitor formation at screening by the central laboratory

For previously untreated subjects:

• Male, up to 18 years of age.
• Documented severe hemophilia B (FIX activity of ≤ 2%), or confirmed at screening by the central laboratory.
• Never previously been treated with FIX clotting factor products (except previous exposure to blood components).
• No confirmed history of FIX inhibitor formation

Surgery substudy inclusion criterion:

• Must require non-emergency surgery

• Male, at least 18 years of age.
• Subjects currently enrolled in Study CSL654_3003
• Subjects who have received rIX-FP for ≥ 100 EDs (single-dose cohorts) or for ≥ 50 EDs (repeated-dose cohort)

Exclusion Criteria

• Currently receiving a therapy not permitted during the study.
• Any issue that, in the opinion of the investigator, would render the subject unsuitable for participation in the study.

For subjects who have previously completed a CSL-sponsored rIX-FP study:

• Unwilling to participate in the study for a total of 100 exposure days.

For subjects requiring major non-emergency surgery who have not previously completed a CSL-sponsored rIX-FP lead-in study:

• Known hypersensitivity (ie, allergic reaction or anaphylaxis) to any FIX product or hamster protein.
• Known congenital or acquired coagulation disorder other than congenital FIX deficiency.
• Currently receiving IV immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment.
• Low platelet count, kidney or liver disease.
• Human immunodeficiency virus positive with a CD4 count < 200/mm3.

For previously untreated subjects:

• Known congenital or acquired coagulation disorder other than congenital FIX deficiency (except for vitamin K deficiency of the newborn).
• Known kidney or liver dysfunction or any condition which, in the investigator's opinion, place the patient at unjustifiable risk.

• Intravenous use of rIX-FP within 14 days of subcutaneous administration of rIX-FP.
• Life-threatening bleeding episode or major surgery during the 3 months prior to substudy entry

• Maximum Eligible Age: 70 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

CSL Behring

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Program Director

Role: STUDY_DIRECTOR

CSL Behring

Locations

University of Colorado

Aurora, Colorado, United States

Indiana Hemophilia & Thrombosis Center Inc.

Indianapolis, Indiana, United States

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

University of Utah

Salt Lake City, Utah, United States

Royal Children's Hospital

Parkville, Victoria, Australia

The Children's Hospital Westmead

Westmead, Victoria, Australia

Department of Pediatrics, Medical University of Vienna

Vienna, , Austria

Medical University of Vienna, Vienna General Hospital

Vienna, , Austria

SHAT "Joan Pavel" ODD [Hemorrhagic Diathesis & Anemia]

Sofia, , Bulgaria

McMaster University

Hamilton, Ontario, Canada

Fakultni nemocnice Brno

Brno, , Czechia

Fakultni Nemocrice Ostrava

Ostrava-Poruba, , Czechia

Fakultni nemocnice v Motole

Prague, , Czechia

CHRU Hopital Morvan

Brest, , France

Hopital Louis Pradel

Bron, , France

Hopital Bicetre - Centre de Traitement del'Hemophilia

Le Kremlin-Bicêtre, , France

Hopital d'Enfants La Timonepital

Marseille, , France

Hopital Necker-Enfants Malades

Paris, , France

Institut fur experimentelle Hamatologie

Bonn, , Germany

Prof. Hess Kinderklinik

Bremen, , Germany

CRC Coagulation Research Centre GmbH

Duisburg, , Germany

Heinrich Heine University Dusseldorf

Düsseldorf, , Germany

Universtatsklinikum Hamburg-Eppendorf

Hamburg, , Germany

Werlhof-Institute for Haemostasis and Thrombosis

Hanover, , Germany

Kurpfalzkrankenhaus Heidlerberg GmbH

Heidelberg, , Germany

Chaim Sheba Medical Center

Tel Aviv, , Israel

IRCCS Ospedale Maggiore[Centro emofilia e Trobosi]

Milan, , Italy

UOS Gestione e Organizzazione Funzlone Hub Emofilia

Parma, , Italy

Centro Malattie Emorragiche e Trombotiche Ospedale

Vicenza, , Italy

Nara Medical University Hospital

Kashihara, , Japan

University of Occupational and Environmental Health

Kitakyushu, , Japan

Nagoya University Hospital

Nagoya, , Japan

The Hospital of Hyogo College of Medicine

Nishinomiya, , Japan

Tokyo Medical University Hospital

Tokyo, , Japan

Ogikubo Hospital

Tokyo, , Japan

St. Marianna University, School of Medicine, Seibu Hospital

Yokohama, , Japan

National Blood Center

Kuala Lumpur, , Malaysia

Perpetual Succour Hospital

Cebu, , Philippines

Haemophilia Comprehensive Care Centre

Parktown, , South Africa

C.H.U. A Coruna

A Coruña, , Spain

Hospital Vall Hebron

Barcelona, , Spain

H.U. La Paz

Madrid, , Spain

Countries

United States; Australia; Austria; Bulgaria; Canada; Czechia; France; Germany; Israel; Italy; Japan; Malaysia; Philippines; South Africa; Spain

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2012-005489-37

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CSL654_3003

Identifier Type: -

Identifier Source: org_study_id