Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B

NCT ID: NCT02141074

Last Updated: 2025-12-23

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

54 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-07-02

Study Completion Date

2022-10-27

Brief Summary

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This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.

Detailed Description

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Conditions

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Congenital Bleeding Disorder Haemophilia 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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50 EDs (exposure days)

Group Type EXPERIMENTAL

nonacog beta pegol

Intervention Type DRUG

For intravenous (i.v.) injection. A single dose of 40 U/kg, unless the bleeding episode is severe in which case it should be treated with 80 U/kg.

Interventions

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nonacog beta pegol

For intravenous (i.v.) injection. A single dose of 40 U/kg, unless the bleeding episode is severe in which case it should be treated with 80 U/kg.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
* Male, age below 6 years at the time of signing informed consent
* Patients with the diagnosis of haemophilia B (FIX (coagulation factor IX) activity level below or equal to 2%) based on medical records or central laboratory results
* Previously untreated or exposed to FIX containing products less than or equal to 3 exposure days (5 previous exposures to blood components is acceptable)

Exclusion Criteria

* Any history of FIX inhibitors (defined by medical records)
* Known or suspected hypersensitivity to trial product or related products
* Previous participation in this trial. Participation is defined as first dose administered of trial product
* Receipt of any investigational medicinal product within 30 days before screening
* Congenital or acquired coagulation disorder other than haemophilia B
* Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise the patient's safety or compliance with the protocol
* Patient's parent(s)/LAR(s) (legally acceptable representative) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation
Minimum Eligible Age

0 Years

Maximum Eligible Age

6 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Novo Nordisk A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Global Clinical Registry (GCR, 1452)

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

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Miller Children's Hospital Long Beach

Long Beach, California, United States

Site Status

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, United States

Site Status

Shands Hospital at the University of Florida

Gainesville, Florida, United States

Site Status

Nemours Chld Clnc Jacksonville

Jacksonville, Florida, United States

Site Status

Augusta University

Augusta, Georgia, United States

Site Status

Hope for Kids

Macon, Georgia, United States

Site Status

St. Luke's Mountain States Tumor Institute

Boise, Idaho, United States

Site Status

University Of Iowa

Iowa City, Iowa, United States

Site Status

Children's Hosp-New Orleans

New Orleans, Louisiana, United States

Site Status

Johns Hopkins University_Baltimore_3

Baltimore, Maryland, United States

Site Status

Univ of NE Med Center_Omaha

Omaha, Nebraska, United States

Site Status

North Shore Long Island Jewish Medical Center

New Hyde Park, New York, United States

Site Status

University of North Carlolina-Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Novant Hlth Vasc Ins Charlotte

Charlotte, North Carolina, United States

Site Status

Univ Hosp Cleveland Med Ctr

Cleveland, Ohio, United States

Site Status

Dayton Children Hemostati Ctr

Dayton, Ohio, United States

Site Status

Penn State MS Hershey Med Ctr

Hershey, Pennsylvania, United States

Site Status

St Christopher Hosp for Child

Philadelphia, Pennsylvania, United States

Site Status

Medical University Of SC

Charleston, South Carolina, United States

Site Status

Vanderbilt Hemostasis Thrombosis Clinic

Nashville, Tennessee, United States

Site Status

Univ of Utah Primary Children's Hospital

Salt Lake City, Utah, United States

Site Status

University of Virginia Children's Hospital

Charlottesville, Virginia, United States

Site Status

Children's Hsptl Of The Kings

Norfolk, Virginia, United States

Site Status

Beni Messous Hospital Issaad Hassani

Algiers, , Algeria

Site Status

University Hospital Saadna Abdenour of Setif

Sétif, , Algeria

Site Status

Hospital de Pediatría S.A.M.I.C. "Prof. Dr. Juan P. Garrahan

CABA, , Argentina

Site Status

Sanatorio Mayo Privado S.A

Córdoba, , Argentina

Site Status

Lady Cilento Children's Hospital

South Brisbane, Queensland, Australia

Site Status

Royal Children's Hospital

Parkville, Victoria, Australia

Site Status

Med. Univ. Graz -Klinische Abteilung f. Allgemeine Pädiatrie

Graz, , Austria

Site Status

Universitätsklinik Kinder-Jugendheilkunde Innsbruck

Innsbruck, , Austria

Site Status

Klinikum Klagenfurt am Wörthersee (LKH Klagenfurt)

Klagenfurt, , Austria

Site Status

Landes-Frauen und Kinderklinik Linz

Linz, , Austria

Site Status

LKH Salzburg- Univ. Klinik f. Kinder- und Jugendheilkunde

Salzburg, , Austria

Site Status

Ordination Prof. Zwiauer

Sankt Pölten, , Austria

Site Status

Universitätsklinik für Kinder- und Jugendheilkunde

Vienna, , Austria

Site Status

UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics

Plovdiv, , Bulgaria

Site Status

Hamltn Hth Sci/McMstr Child Hosp

Hamilton, Ontario, Canada

Site Status

The Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

CHU Estaing

Clermont-Ferrand, , France

Site Status

Ap-Hp-Hopital de Bicetre-1

Le Kremlin-Bicêtre, , France

Site Status

Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu

Nantes, , France

Site Status

Centre Hospitalier Universitaire de Rennes - Hopital Pontchaillou

Rennes, , France

Site Status

CHU Haute Pierre

Strasbourg, , France

Site Status

Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie

Bonn, , Germany

Site Status

Coagulation Research Center

Duisburg, , Germany

Site Status

HZRM Haemophilie-Zentrum Rhein Main GmbH

Frankfurt am Main, , Germany

Site Status

Werlhof-Institut

Hanover, , Germany

Site Status

Aghia Sophia Childrens' Hospital

Athens, , Greece

Site Status

'Ippokrateio' General Hospital of Thessaloniki

Thessaloniki, , Greece

Site Status

Sheba MC - The Israeli National Hemophilia Center

Tel Litwinsky, , Israel

Site Status

Dipartimento di Ematologia Univ. Firenze

Florence, , Italy

Site Status

IRCCS Meyer Firenze

Florence, , Italy

Site Status

Nagoya University Hospital_Blood Transfusion

Aichi, , Japan

Site Status

Kanagawa Children's Medical Center _ Hematology / Oncology

Kanagawa, , Japan

Site Status

Saitama Children's Med Centre_Hematology-Oncology

Saitama, , Japan

Site Status

Shizuoka Children's Hospital, Hematology-Oncology

Shizuoka, , Japan

Site Status

National Center for Child Health and Development_Hematology

Tokyo, , Japan

Site Status

Ogikubo Hospital_Pediatries & Blood

Tokyo, , Japan

Site Status

Hospital Tengku Ampuan Afzan

Kuantan, Pahang, Malaysia

Site Status

Hospital Pulau Pinang_Georgetown, Penang

George Town, Pulau Pinang, Malaysia

Site Status

Hospital Tengku Ampuan Rahimah

Klang, Selangor, , Malaysia

Site Status

National Blood Centre

Kuala Lumpur, , Malaysia

Site Status

Centro Medico Nacional SXXI-Hospital de Pediatria, IMSS

Mexico City, México, D.F., Mexico

Site Status

Hospital Universitario Dr. José Eleuterio González_Monterrey

Monterrey, Nuevo León, Mexico

Site Status

Radboudumc

Nijmegen, , Netherlands

Site Status

Centro Hospitalar Lisboa Norte-HSM

Lisbon, , Portugal

Site Status

ULS São João, E.P.E.

Porto, , Portugal

Site Status

,,Louis Ţurcanu'' Emergency Hospital for Children

Timișoara, Timiș County, Romania

Site Status

1st Paediatric Department, Fundeni Clinical Institute

Bucharest, , Romania

Site Status

University Children's Hospital Tirsova

Belgrade, , Serbia

Site Status

Institute for Mother and Child Health Care of Serbia

Belgrade, , Serbia

Site Status

Hospital Sant Joan de Déu

Esplugues Llobregat, , Spain

Site Status

Hospital Universitario La Paz

Madrid, , Spain

Site Status

Hospital La Fe - Endocrinología y Nutrición

Valencia, , Spain

Site Status

Changhua Christian Hospital_Hematology Dept.

Changhua, , Taiwan

Site Status

Changhua Christian Hospital

Changhua, , Taiwan

Site Status

Kaohsiung Medical University Chung-Ho Memorial Hospital_Dept of Pediatrics

Kaohsiung City, , Taiwan

Site Status

China Medical University Children's Hospital

Taichung, , Taiwan

Site Status

China Medical University Hospital - Children Building

Taichung, , Taiwan

Site Status

National Taiwan University Children's Hospital

Taipei, , Taiwan

Site Status

King Chulalongkorn Memorial Hospital_Pediatric Hematology-Oncology

Bangkok, , Thailand

Site Status

Ramathibodi Hospital_Paediatrics

Bangkok, , Thailand

Site Status

Maharaj Nakorn Chiang Mai Hospital _Pediatric Hematology and Oncology

Chiang Mai, , Thailand

Site Status

SI Institute of Urgent and Recovery Surgery - Haematology

Donetsk, , Ukraine

Site Status

Institute of blood pathology and transfusion medicine of NAMSU - General and haematol. surgery

Lviv, , Ukraine

Site Status

Birmingham Children's Hospital

Birmingham, , United Kingdom

Site Status

Kent and Canterbury Hospital - Kent Haemophilia and Thrombosis Centre

Canterbury, , United Kingdom

Site Status

Royal Hospital for Children

Glasgow, , United Kingdom

Site Status

Leicester Royal Infirmary - Haemostasis & Thrombosis Unit

Leicester, , United Kingdom

Site Status

St Thomas' Hospital - Haemostasis and Thrombosis Centre

London, , United Kingdom

Site Status

Royal Manchester Children's Hospital

Manchester, , United Kingdom

Site Status

John Radcliffe Hospital

Oxford, , United Kingdom

Site Status

Countries

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United States Algeria Argentina Australia Austria Bulgaria Canada France Germany Greece Israel Italy Japan Malaysia Mexico Netherlands Portugal Romania Serbia Spain Taiwan Thailand Ukraine United Kingdom

References

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Chan AK, Alamelu J, Barnes C, Chuansumrit A, Garly ML, Meldgaard RM, Young G. Nonacog beta pegol (N9-GP) in hemophilia B: First report on safety and efficacy in previously untreated and minimally treated patients. Res Pract Thromb Haemost. 2020 Jul 29;4(7):1101-1113. doi: 10.1002/rth2.12412. eCollection 2020 Oct.

Reference Type BACKGROUND
PMID: 33134776 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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http://novonordisk-trials.com

Clinical Trials at Novo Nordisk

Other Identifiers

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2012-004867-38

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1135-9557

Identifier Type: OTHER

Identifier Source: secondary_id

JapicCTI-142611

Identifier Type: REGISTRY

Identifier Source: secondary_id

NL53683.091.15

Identifier Type: OTHER

Identifier Source: secondary_id

NN7999-3895

Identifier Type: -

Identifier Source: org_study_id