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Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B (paradigm™5)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01467427
First received: October 31, 2011
Last updated: May 31, 2017
Last verified: May 2017
  Purpose
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.

Condition Intervention Phase
Congenital Bleeding Disorder Haemophilia B Drug: nonacog beta pegol Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B

Resource links provided by NLM:


Further study details as provided by Novo Nordisk A/S:

Primary Outcome Measures:
  • Incidence of inhibitory antibodies against coagulation factor IX (FIX) defined as titre above or equal to 0.6 Bethesda Units (BU) [ Time Frame: From 0 to 52 weeks and from week 52 until the last patient has completed the trial ]

Secondary Outcome Measures:
  • Number of bleeding episodes during prophylaxis [ Time Frame: From 0 to 52 weeks and from week 52 until the last patient has completed the trial ]
  • Haemostatic effect of N9-GP in treatment of bleeding episodes by 4-point categorical scale for haemostatic response (excellent, good, moderate and poor) [ Time Frame: From 0 to 52 weeks and from week 52 until the last patient has completed the trial ]
  • Incremental recovery at 30 minutes (IR30min) [ Time Frame: Week 0 ]
  • Trough level (single-dose ) [ Time Frame: Week 0 ]
  • Trough level (steady state) [ Time Frame: Week 4 to 52 weeks and from week 52 until the patient has completed the trial ]
  • Terminal half-life (t1/2) [ Time Frame: Week 0 ]

Enrollment: 25
Actual Study Start Date: May 16, 2012
Estimated Study Completion Date: October 31, 2018
Primary Completion Date: April 1, 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NNC-0156-000-0009 Drug: nonacog beta pegol
A single dose of 40 U/kg will be administered intravenously, i.v. (into the vein) once weekly.
Other Name: NNC-0156-0000-0009

  Eligibility

Ages Eligible for Study:   up to 12 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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
  • Age below or equal to 12 years (until patient turns 13 years, at time of inclusion)
  • 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

Exclusion Criteria:

  • 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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01467427

  Show 37 Study Locations
Sponsors and Collaborators
Novo Nordisk A/S
  More Information

Additional Information:
Publications:
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)

Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01467427     History of Changes
Other Study ID Numbers: NN7999-3774
2011-000826-31 ( EudraCT Number )
U1111-1119-5013 ( Other Identifier: WHO )
JapicCTI- 121877 ( Registry Identifier: JAPIC )
Study First Received: October 31, 2011
Last Updated: May 31, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: According to the Novo Nordiskdisclosure commitment on novonordisk-trials.com

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders
Hemostatic Disorders
Blood Coagulation Disorders, Inherited
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Vascular Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on August 22, 2017