Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B.

NCT01467427 | Completed Phase 3 Results FDA Drug

• 4 other identifiers: NN7999-37742011-000826-31U1111-1119-5013JapicCTI- 121877
• Study Type: interventional
• Target: 25
• Locations: 12 countries
• Sites: 51

Brief Summary

This trial is conducted in Asia, Europe and North America. The aim of the trial is to evaluate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC-0156-0000-0009 (nonacog beta pegol, N9-GP) in previously treated children with Haemophilia B.

Conditions

Congenital Bleeding Disorder; Haemophilia B

Outcome Measures

Primary Outcomes (2)

• Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)

  Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of participants who developed inhibitory antibodies against factor IX are reported.

  From week 0 to week 52

• Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)

  Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of participants who developed inhibitory antibodies against factor IX are reported.

  From week 52 to End of trial (EOT) (approximately week 544)

Secondary Outcomes (27)

• Number of Bleeding Episodes During Prophylaxis

  From week 0 to EOT (approximately week 544)

• Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor)

  From week 0 to EOT (approximately week 544)

• Incremental Recovery at 30 Minutes (IR30min)

  Week 0 (30 minutes after first exposure)

• Trough Level (Single-dose )

  Week 0 (one week after first exposure)

• Terminal Half-life (t1/2)

  Week 0 (30 minutes until one week after first exposure)

Study Arms (1)

NNC-0156-000-0009

EXPERIMENTAL

Drug: nonacog beta pegol

Interventions

nonacog beta pegol DRUG

A single dose of 40 U/kg will be administered intravenously, i.v. (into the vein) once weekly.

Also known as: NNC-0156-0000-0009

NNC-0156-000-0009

Eligibility Criteria

• Age: 0 Years - 12 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male patients with moderately severe or severe congenital haemophilia B with a Factor IX activity level below or equal to 2% according to medical records
• Body weight above or equal to 10 kg
• History of at least 50 exposure days (EDs) to other FIX products
• The patient and/or parent(s)/caregiver are capable of assessing a bleeding episode, keeping an electronic diary (eDiary), capable of conducting home treatment and otherwise able to follow trial procedures

You may not qualify if:

• Known history of FIX inhibitors
• Current FIX inhibitors above or equal to 0.6 Bethesda Units (BU)
• Congenital or acquired coagulation disorder other than haemophilia B
• Platelet count below 50,000/mcL at screening
• Alanine aminotransferase (ALT) above 3 times the upper limit of normal reference ranges at screening
• Creatinine level above or equal to 1.5 times above the upper normal limit of normal reference ranges at screening
• Human immunodeficiency virus (HIV) positive, defined by medical records, and with a CD4+ lymphocyte count below or equal to 200/mcL
• Immune modulating or chemotherapeutic medication (except single pulse treatment, inhaled and topical steroids)
• Previous arterial thrombotic events (myocardial infarction and intracranial thrombosis, as defined by medical records)

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (51)

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, 90027, United States

Location

UC Davis Medical Center

Sacramento, California, 95817, United States

Location

Colorado Hemophilia & Thrombosis Center

Aurora, Colorado, 80045, United States

Location

Childrens National Medical Ctr

Washington D.C., District of Columbia, 20010-2978, United States

Location

University of Miami Hospital & Clinics

Miami, Florida, 33136, United States

Location

Emory University_Atlanta_1

Atlanta, Georgia, 30322, United States

Location

Augusta University

Augusta, Georgia, 30912, United States

Location

University Of Iowa

Iowa City, Iowa, 52242, United States

Location

University Of Louisville_Louisville_0

Louisville, Kentucky, 40202, United States

Location

Johns Hopkins University_Baltimore_3

Baltimore, Maryland, 21205, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

Children's Hospitals And Clinics Of Minnesota

Minneapolis, Minnesota, 55404, United States

Location

The Children's Mercy Hospital

Kansas City, Missouri, 64108-4619, United States

Location

Univ of NE Med Center_Omaha

Omaha, Nebraska, 68198, United States

Location

Rutgers-Robert Wood Johnson Medical School

New Brunswick, New Jersey, 08901, United States

Location

Maimonides Medical Center

Brooklyn, New York, 11220, United States

Location

Mount Sinai Medical Center

New York, New York, 10029, United States

Location

Univ Hosp Cleveland Med Ctr

Cleveland, Ohio, 44106, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Dayton Children Hemostati Ctr

Dayton, Ohio, 45404, United States

Location

Univ Oklahoma Sci Ctr OK City

Oklahoma City, Oklahoma, 73104, United States

Location

Children's Hosptl Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Vanderbilt Hemostasis Thrombosis Clinic

Nashville, Tennessee, 37232, United States

Location

Children's Medical Center_Dallas

Dallas, Texas, 75235, United States

Location

Texas Children's Hospital_Houston

Houston, Texas, 77030, United States

Location

Univ TX Hlth Sci Ctr Houston

Houston, Texas, 77030, United States

Location

Nucleo de Pesquisa Instituto Pele Pequeno Principe

Curitiba, Paraná, 80250-060, Brazil

Location

Universidade Estadual de Campinas

Campinas, São Paulo, 13081-970, Brazil

Location

Hospital das Clínicas da Faculdade de Medicina da USP

São Paulo, São Paulo, 05403-000, Brazil

Location

Hemorio-Fundarj

Rio de Janeiro, 20211-030, Brazil

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Clinical Hospital Centre Split, Firule, Paediatric Haem. Dpt

Split, 21 000, Croatia

Location

Ap-Hp-Hopital de Bicetre-1

Le Kremlin-Bicêtre, 94275, France

Location

Hôpital de la Timone

Marseille, 13385, France

Location

Ap-Hp-Hopital Necker-1

Paris, 75015, France

Location

Coagulation Research Center

Duisburg, 47051, Germany

Location

Medizinische Hochschule Hannover - Pädiatrische Hämato-, Onko- und Hämostasiologie

Hanover, 30625, Germany

Location

Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano

Milan, MI, 20124, Italy

Location

St. Marianna University School of Medicine Hospital_Pediatrics

Kanagawa, 216-8511, Japan

Location

Shizuoka Children's Hospital, Hematology-Oncology

Shizuoka, 420-8660, Japan

Location

Ogikubo Hospital_Pediatries & Blood

Tokyo, 167-0035, Japan

Location

National Blood Centre

Kuala Lumpur, 50400, Malaysia

Location

National Taiwan University Children's Hospital

Taipei, 100, Taiwan

Location

Necmettin Erbakan University Pediatric Hematology

Konya, 42090, Turkey (Türkiye)

Location

Basingstoke & North Hampshire Hospital - Centre for Haemophilia, Haemostasis and Thrombosis

Basingstoke, RG24 9NA, United Kingdom

Location

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

Leicester Royal Infirmary - Haemostasis & Thrombosis Unit

Leicester, LE1 5WW, United Kingdom

Location

Leicester Royal Infirmary

Leicester, LE1 5WW, United Kingdom

Location

St Thomas' Hospital - Haemostasis and Thrombosis Centre

London, SE1 7EH, United Kingdom

Location

St Thomas' Hospital

London, SE1 7EH, United Kingdom

Location

John Radcliffe Hospital

Oxford, OX3 9DU, United Kingdom

Location

Related Publications (3)

• Safety, efficacy and pharmacokinetics of nonacog beta pegol (N9-GP) in prophylaxis and treatment of bleeding episodes in previously treated pediatric hemophilia B patients. Carcao M, Zak M, Abdul Karim F, Hanabusa H, Kearney S, Lu M-Y, Persson P, Rangarajan S, Santagostino E. Presented 06-Dec-2014 at the American Society of Hematology - 56th Annual Meeting - held in San Francisco, CA, US (poster #1513)

  BACKGROUND

• Carcao M, Zak M, Abdul Karim F, Hanabusa H, Kearney S, Lu MY, Persson P, Rangarajan S, Santagostino E. Nonacog beta pegol in previously treated children with hemophilia B: results from an international open-label phase 3 trial. J Thromb Haemost. 2016 Aug;14(8):1521-9. doi: 10.1111/jth.13360. Epub 2016 Jun 22.

  PMID: 27174727 RESULT

• Carcao M, Kearney S, Lu MY, Taki M, Rubens D, Shen C, Santagostino E. Long-Term Safety and Efficacy of Nonacog Beta Pegol (N9-GP) Administered for at Least 5 Years in Previously Treated Children with Hemophilia B. Thromb Haemost. 2020 May;120(5):737-746. doi: 10.1055/s-0040-1709521. Epub 2020 May 5.

  PMID: 32369845 RESULT

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

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: October 31, 2011
• First Posted: November 8, 2011
• Study Start: May 16, 2012
• Primary Completion: November 17, 2023
• Study Completion: November 17, 2023
• Last Updated: December 23, 2025
• Results First Posted: November 17, 2017

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

MY (1); TW (1); BR (4); GB (7); IT (1); FR (3); DE (2); US (26); CA (1); TU (1); CR (1); JP (3)