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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: September 16, 2014
Last verified: September 2014
  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 (N9-GP) in previously treated children with Haemophilia B.


Condition Intervention Phase
Congenital Bleeding Disorder
Haemophilia B
Drug: NNC-0156-0000-0009
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 ] [ Designated as safety issue: No ]

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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Incremental recovery at 30 minutes (IR30min) [ Time Frame: Week 0 ] [ Designated as safety issue: No ]
  • Trough level (single-dose ) [ Time Frame: Week 0 ] [ Designated as safety issue: No ]
  • Trough level (steady state) [ Time Frame: Week 4-44 ] [ Designated as safety issue: No ]
  • Terminal half-life (t1/2) [ Time Frame: Week 0 ] [ Designated as safety issue: No ]

Enrollment: 25
Study Start Date: May 2012
Estimated Study Completion Date: January 2016
Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NNC-0156-000-0009 Drug: NNC-0156-0000-0009
A single dose of 40 U/kg will be administered intravenously, i.v. (into the vein) once weekly.

  Eligibility

Ages Eligible for Study:   up to 13 Years
Genders 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 21 Study Locations
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  More Information

Additional Information:
No publications provided

Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01467427     History of Changes
Other Study ID Numbers: NN7999-3774, 2011-000826-31, U1111-1119-5013, JapicCTI- 121877
Study First Received: October 31, 2011
Last Updated: September 16, 2014
Health Authority: Canada: Health Canada
Germany: Federal Institute for Drugs and Medical Devices
Italy: The Italian Medicines Agency
Japan: Ministry of Health, Labor and Welfare
Malaysia: Ministry of Health
Taiwan: Department of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on November 20, 2014