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

NCT02141074 | Completed Phase 3 Results

• 5 other identifiers: NN7999-38952012-004867-38U1111-1135-9557JapicCTI-142611NL53683.091.15
• Study Type: interventional
• Target: 54
• Locations: 23 countries
• Sites: 91

Brief Summary

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.

Conditions

Haemophilia B; Congenital Bleeding Disorder

Outcome Measures

Primary Outcomes (3)

• Number of Participants With Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) (50 Exposure Days)

  Number of participants with incidence of inihibitory antibodies against FIX after 50 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.

  When minimum 20 previously untreated patients (PUPs) have reached at least 50 exposure days (ED) (up to 156 weeks)

• Number of Participants With Incidence of Inhibitory Antibodies Against FIX (100 ED)

  Number of participants with incidence of inihibitory antibodies against FIX after 100 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.

  When minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)

• Number of Participants With Incidence of Inhibitory Antibodies Against FIX (At End of Trial)

  Number of participants with incidence of inihibitory antibodies against FIX at end of trial is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.

  At end of trial (up to 434 weeks)

Secondary Outcomes (13)

• Number of Adverse Events

  When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)

• Frequency of Adverse Events

  When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)

• Number of Serious Adverse Events

  When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)

• Frequency of Serious Adverse Events

  When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)

• Number of Medical Events of Special Interest

  When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)

Study Arms (1)

50 EDs (exposure days)

EXPERIMENTAL

Drug: nonacog beta pegol

Interventions

nonacog beta pegol 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.

50 EDs (exposure days)

Eligibility Criteria

• Age: 0 Years - 6 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (93)

Miller Children's Hospital Long Beach

Long Beach, California, 90806, United States

Location

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, 90027, United States

Location

Shands Hospital at the University of Florida

Gainesville, Florida, 32610, United States

Location

Nemours Chld Clnc Jacksonville

Jacksonville, Florida, 32207, United States

Location

Augusta University

Augusta, Georgia, 30912, United States

Location

Hope for Kids

Macon, Georgia, 31201, United States

Location

St. Luke's Mountain States Tumor Institute

Boise, Idaho, 83712, United States

Location

University Of Iowa

Iowa City, Iowa, 52242, United States

Location

Children's Hosp-New Orleans

New Orleans, Louisiana, 70118-5720, United States

Location

Johns Hopkins University_Baltimore_3

Baltimore, Maryland, 21205, United States

Location

Univ of NE Med Center_Omaha

Omaha, Nebraska, 68198-6828, United States

Location

North Shore Long Island Jewish Medical Center

New Hyde Park, New York, 11040, United States

Location

University of North Carlolina-Chapel Hill

Chapel Hill, North Carolina, 27514, United States

Location

Novant Hlth Vasc Ins Charlotte

Charlotte, North Carolina, 28204, United States

Location

Univ Hosp Cleveland Med Ctr

Cleveland, Ohio, 44106, United States

Location

Dayton Children Hemostati Ctr

Dayton, Ohio, 45404, United States

Location

Penn State MS Hershey Med Ctr

Hershey, Pennsylvania, 17033, United States

Location

St Christopher Hosp for Child

Philadelphia, Pennsylvania, 19134, United States

Location

Medical University Of SC

Charleston, South Carolina, 29425, United States

Location

Vanderbilt Hemostasis Thrombosis Clinic

Nashville, Tennessee, 37232, United States

Location

Univ of Utah Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

Location

University of Virginia Children's Hospital

Charlottesville, Virginia, 22903, United States

Location

Children's Hsptl Of The Kings

Norfolk, Virginia, 23507, United States

Location

Beni Messous Hospital Issaad Hassani

Algiers, 16000, Algeria

Location

University Hospital Saadna Abdenour of Setif

Sétif, 19000, Algeria

Location

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

CABA, C1245AAM, Argentina

Location

Sanatorio Mayo Privado S.A

Córdoba, X5000FAL, Argentina

Location

Lady Cilento Children's Hospital

South Brisbane, Queensland, 4101, Australia

Location

Royal Children's Hospital

Parkville, Victoria, 3052, Australia

Location

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

Graz, 8036, Austria

Location

Universitätsklinik Kinder-Jugendheilkunde Innsbruck

Innsbruck, 6020, Austria

Location

Klinikum Klagenfurt am Wörthersee (LKH Klagenfurt)

Klagenfurt, 9020, Austria

Location

Landes-Frauen und Kinderklinik Linz

Linz, 4020, Austria

Location

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

Salzburg, 5020, Austria

Location

Ordination Prof. Zwiauer

Sankt Pölten, A 3100, Austria

Location

Universitätsklinik für Kinder- und Jugendheilkunde

Vienna, A 1090, Austria

Location

UMHAT Sveti Georgi EAD, Plovdiv, Clinic of Pediatrics

Plovdiv, 4002, Bulgaria

Location

Hamltn Hth Sci/McMstr Child Hosp

Hamilton, Ontario, L8N 3Z5, Canada

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

CHU Estaing

Clermont-Ferrand, 63003, France

Location

Ap-Hp-Hopital de Bicetre-1

Le Kremlin-Bicêtre, 94275, France

Location

Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu

Nantes, 44093, France

Location

Centre Hospitalier Universitaire de Rennes - Hopital Pontchaillou

Rennes, 35033, France

Location

CHU Haute Pierre

Strasbourg, 67098, France

Location

Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie

Bonn, 53127, Germany

Location

Coagulation Research Center

Duisburg, 47051, Germany

Location

HZRM Haemophilie-Zentrum Rhein Main GmbH

Frankfurt am Main, 60596, Germany

Location

Werlhof-Institut

Hanover, 30159, Germany

Location

Aghia Sophia Childrens' Hospital

Athens, GR-11527, Greece

Location

'Ippokrateio' General Hospital of Thessaloniki

Thessaloniki, 54642, Greece

Location

Sheba MC - The Israeli National Hemophilia Center

Tel Litwinsky, 52621, Israel

Location

Dipartimento di Ematologia Univ. Firenze

Florence, 50134, Italy

Location

IRCCS Meyer Firenze

Florence, 50139, Italy

Location

Nagoya University Hospital_Blood Transfusion

Aichi, 466-8560, Japan

Location

Kanagawa Children's Medical Center _ Hematology / Oncology

Kanagawa, 232-8555, Japan

Location

Saitama Children's Med Centre_Hematology-Oncology

Saitama, 330-8777, Japan

Location

Shizuoka Children's Hospital, Hematology-Oncology

Shizuoka, 420-8660, Japan

Location

National Center for Child Health and Development_Hematology

Tokyo, 157-8535, Japan

Location

Ogikubo Hospital_Pediatries & Blood

Tokyo, 167-0035, Japan

Location

Hospital Tengku Ampuan Afzan

Kuantan, Pahang, 25100, Malaysia

Location

Hospital Pulau Pinang_Georgetown, Penang

George Town, Pulau Pinang, 10450, Malaysia

Location

Hospital Tengku Ampuan Rahimah

Klang, Selangor, 41200, Malaysia

Location

National Blood Centre

Kuala Lumpur, 50400, Malaysia

Location

Centro Medico Nacional SXXI-Hospital de Pediatria, IMSS

Mexico City, México, D.F., 06720, Mexico

Location

Hospital Universitario Dr. José Eleuterio González_Monterrey

Monterrey, Nuevo León, 64460, Mexico

Location

Radboudumc

Nijmegen, 6525 GA, Netherlands

Location

Centro Hospitalar Lisboa Norte-HSM

Lisbon, 1649-035, Portugal

Location

ULS São João, E.P.E.

Porto, 4200-319, Portugal

Location

,,Louis Ţurcanu'' Emergency Hospital for Children

Timișoara, Timiș County, 300011, Romania

Location

1st Paediatric Department, Fundeni Clinical Institute

Bucharest, 022328, Romania

Location

University Children's Hospital Tirsova

Belgrade, 11000, Serbia

Location

Institute for Mother and Child Health Care of Serbia

Belgrade, 11070, Serbia

Location

Hospital Sant Joan de Déu

Esplugues Llobregat, 08950, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital La Fe - Endocrinología y Nutrición

Valencia, 46026, Spain

Location

Changhua Christian Hospital_Hematology Dept.

Changhua, 500, Taiwan

Location

Changhua Christian Hospital

Changhua, 500, Taiwan

Location

Kaohsiung Medical University Chung-Ho Memorial Hospital_Dept of Pediatrics

Kaohsiung City, 807, Taiwan

Location

China Medical University Children's Hospital

Taichung, 40447, Taiwan

Location

China Medical University Hospital - Children Building

Taichung, 40447, Taiwan

Location

National Taiwan University Children's Hospital

Taipei, 100, Taiwan

Location

King Chulalongkorn Memorial Hospital_Pediatric Hematology-Oncology

Bangkok, 10330, Thailand

Location

Ramathibodi Hospital_Paediatrics

Bangkok, 10400, Thailand

Location

Maharaj Nakorn Chiang Mai Hospital _Pediatric Hematology and Oncology

Chiang Mai, 50200, Thailand

Location

SI Institute of Urgent and Recovery Surgery - Haematology

Donetsk, 83045, Ukraine

Location

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

Lviv, 79044, Ukraine

Location

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

Kent and Canterbury Hospital - Kent Haemophilia and Thrombosis Centre

Canterbury, CT1 3NG, United Kingdom

Location

Royal Hospital for Children

Glasgow, G51 4TF, United Kingdom

Location

Leicester Royal Infirmary - Haemostasis & Thrombosis Unit

Leicester, LE1 5WW, United Kingdom

Location

St Thomas' Hospital - Haemostasis and Thrombosis Centre

London, SE1 7EH, United Kingdom

Location

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

Location

John Radcliffe Hospital

Oxford, OX3 9DU, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 33134776 BACKGROUND

Related Links

• Clinical Trials at Novo Nordisk

MeSH Terms

Conditions

Hemophilia B

Interventions

nonacog beta pegol

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Results Point of Contact

• Title: Clinical Reporting Office (2834)
• Organization: Novo Nordisk A/S

clinicaltrials@novonordisk.com

(+1) 866-867-7178

Study Officials

• Global Clinical Registry (GCR, 1452)

  Novo Nordisk A/S

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 28, 2014
• First Posted: May 19, 2014
• Study Start: July 2, 2014
• Primary Completion: October 27, 2022
• Study Completion: October 27, 2022
• Last Updated: December 23, 2025
• Results First Posted: December 29, 2023

Record last verified: 2025-12

Locations

TH (3); AU (7); CA (2); AL (2); FR (5); GR (2); JP (6); MY (4); ME (2); BU (1); IT (2); PT (2); RO (2); SE (2); AR (2); IL (1); DE (4); US (23); TW (6); GB (7); UA (2); NL (1); ES (3)