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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
ClinicalTrials.gov Identifier:
NCT01467427
First received: October 31, 2011
Last updated: April 9, 2013
Last verified: April 2013
History of Changes
  Purpose

This trial is conducted in Asia, Europe and North and South America. The aim of this 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:

U.S. FDA Resources

Further study details as provided by Novo Nordisk:

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 ]

Estimated Enrollment: 24
Study Start Date: May 2012
Estimated Study Completion Date: January 2016
Estimated 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01467427

Locations
United States, Georgia
Novo Nordisk Clinical Trial Call Center
Augusta, Georgia, United States, 30912
United States, Minnesota
Novo Nordisk Clinical Trial Call Center
Minneapolis, Minnesota, United States, 55404
United States, New York
Novo Nordisk Clinical Trial Call Center
Brooklyn, New York, United States, 11220
Novo Nordisk Clinical Trial Call Center
New York, New York, United States, 10029
United States, Tennessee
Novo Nordisk Clinical Trial Call Center
Nashville, Tennessee, United States, 37232-9830
United States, Texas
Novo Nordisk Clinical Trial Call Center
Houston, Texas, United States, 77030
Canada, Ontario
Toronto, Ontario, Canada, M5G 1X8
Italy
Milano, Italy, 20124
Japan
Suginami-ku, Tokyo, Japan, 1670035
Malaysia
Kuala Lumpur, Malaysia, 50400
Taiwan
Taipei, Taiwan, 100
Turkey
Konya, Turkey, 42090
United Kingdom
Oxford, United Kingdom, OX3 7LJ
Sponsors and Collaborators
Novo Nordisk
Investigators
Study Director: Lene Hasselriis Novo Nordisk
  More Information

Additional Information:
Clinical Trials at Novo Nordisk  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Novo Nordisk
ClinicalTrials.gov Identifier: NCT01467427     History of Changes
Other Study ID Numbers: NN7999-3774, P/292/2010, 2011-000826-31, U1111-1119-5013, JapicCTI- 121877
Study First Received: October 31, 2011
Last Updated: April 9, 2013
Health Authority: Turkey: Ministry of Health Drug and Pharmaceutical Department
Canada: Health Canada
Japan: Ministry of Health, Labor and Welfare
Malaysia: Ministry of Health
Taiwan: Department of Health
United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on May 23, 2013