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

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ClinicalTrials.gov Identifier: NCT01467427
Recruitment Status : Active, not recruiting
First Posted : November 8, 2011
Results First Posted : November 17, 2017
Last Update Posted : December 21, 2017
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

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.

Condition or disease Intervention/treatment Phase
Congenital Bleeding Disorder Haemophilia B Drug: nonacog beta pegol Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
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
Actual Study Start Date : May 16, 2012
Primary Completion Date : March 30, 2014
Estimated Study Completion Date : November 30, 2023

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
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



Primary Outcome Measures :
  1. 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 ]
    Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of subjects who developed inhibitory antibodies against factor IX are reported.

  2. Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU) [ Time Frame: From week 52 until the last patient has completed the trial (no later than 31-Oct-2018) ]

Secondary Outcome Measures :
  1. Number of Bleeding Episodes During Prophylaxis [ Time Frame: From 0 to 52 weeks ]
    The number of bleeding episodes per subject during routine prophylaxis was assessed using the individual annualised bleeding rates (bleeding episodes per subject per year).

  2. 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 ]

    Description of the haemostatic effect of nonacog beta pegol when used for treatment of bleeding episodes was measured and listed according to the four point scale for haemostatic response as below:

    1. Excellent - abrupt pain relief and/or clear improvement in objective signs of bleeding within 8 hours after a single infusion.
    2. Good - noticeable pain relief and/or improvement in signs of bleeding within 8 hours after a single injection.
    3. Moderate - probable or slight beneficial effect within the first 8 hours after the first injection but requiring more than one infusion within 8 hours.
    4. Poor - no improvement, or worsening of symptoms within 8 hours after two injections.

    A success rate was calculated based on counting good or excellent as successes and poor and moderate as failures.


  3. Incremental Recovery at 30 Minutes (IR30min) [ Time Frame: Week 0 (30 minutes after first exposure) ]
    The incremental recovery was calculated by dividing the baseline-subtracted factor IX activity (U/mL) measured in plasma 30 min after dosing by the dose injected at time 0 expressed as U/kg body weight.

  4. Trough Level (Single-dose ) [ Time Frame: Week 0 (one week after first exposure) ]
    The mean pre-dose factor IX levels was measured with the one-stage clotting assay during the trial. Geometric mean of the lowest activity of factor IX recorded at week 0 (immediately before next dose was given).

  5. Trough Level (Steady State) [ Time Frame: Week 4 to 52 weeks ]
    The mean pre-dose factor IX levels was measured with the one-stage clotting assay during the trial. The estimated mean of the lowest activity recorded immediately before next dose was given from week 4 to week 52. The analysis is based on a mixed model on the log-transformed plasma concentrations with subject as a random effect and the mean trough level is presented back-transformed to the natural scale.

  6. Terminal Half-life (t1/2) [ Time Frame: Week 0 (30 minutes until one week after first exposure) ]
  7. Number of Bleeding Episodes During Prophylaxis [ Time Frame: From week 52 until the last patient has completed the trial (no later than 31-Oct-2018) ]
  8. Trough Level (Steady State) [ Time Frame: From week 52 until the patient has completed the trial (no later than 31-Oct-2018) ]
  9. 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 week 52 until the last patient has completed the trial (no later than 31-Oct-2018) ]


Information from the National Library of Medicine

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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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01467427


  Hide Study Locations
Locations
United States, District of Columbia
Novo Nordisk Investigational Site
Washington, District of Columbia, United States, 20010-2978
United States, Florida
Novo Nordisk Investigational Site
Miami, Florida, United States, 33136
United States, Georgia
Novo Nordisk Investigational Site
Augusta, Georgia, United States, 30912
United States, Iowa
Novo Nordisk Investigational Site
Iowa City, Iowa, United States, 52242
United States, Minnesota
Novo Nordisk Investigational Site
Minneapolis, Minnesota, United States, 55404
United States, Missouri
Novo Nordisk Investigational Site
Kansas City, Missouri, United States, 64108-4619
United States, Nebraska
Novo Nordisk Investigational Site
Omaha, Nebraska, United States, 68198-2168
United States, New Jersey
Novo Nordisk Investigational Site
New Brunswick, New Jersey, United States, 08901
United States, New York
Novo Nordisk Investigational Site
Brooklyn, New York, United States, 11220
Novo Nordisk Investigational Site
New York, New York, United States, 10029
United States, Ohio
Novo Nordisk Investigational Site
Cleveland, Ohio, United States, 44106
United States, Oklahoma
Novo Nordisk Investigational Site
Oklahoma City, Oklahoma, United States, 73104
United States, Pennsylvania
Novo Nordisk Investigational Site
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Novo Nordisk Investigational Site
Nashville, Tennessee, United States, 37232-9830
United States, Texas
Novo Nordisk Investigational Site
Dallas, Texas, United States, 75235
Novo Nordisk Investigational Site
Houston, Texas, United States, 77030
Brazil
Novo Nordisk Investigational Site
Curitiba, Parana, Brazil, 80250-060
Novo Nordisk Investigational Site
Campinas, Sao Paulo, Brazil, 13081970
Novo Nordisk Investigational Site
Rio de Janeiro, Brazil, 20211-030
Canada, Ontario
Novo Nordisk Investigational Site
Toronto, Ontario, Canada, M5G 1X8
Croatia
Novo Nordisk Investigational Site
Split, Croatia, 21 000
France
Novo Nordisk Investigational Site
Kremlin-Bicêtre, France, 94270
Novo Nordisk Investigational Site
Marseille, France, 13385
Novo Nordisk Investigational Site
Paris, France, 75015
Germany
Novo Nordisk Investigational Site
Duisburg, Germany, 47051
Novo Nordisk Investigational Site
Hannover, Germany, 30625
Italy
Novo Nordisk Investigational Site
Milano, Italy, 20124
Japan
Novo Nordisk Investigational Site
Kawasaki-shi, Kanagawa, Japan, 216-8511
Novo Nordisk Investigational Site
Shizuoka-shi, Shizuoka, Japan, 4208660
Novo Nordisk Investigational Site
Suginami-ku, Tokyo, Japan, 167 0035
Malaysia
Novo Nordisk Investigational Site
Kuala Lumpur, Malaysia, 50400
Taiwan
Novo Nordisk Investigational Site
Taipei, Taiwan, 100
United Kingdom
Novo Nordisk Investigational Site
Basingstoke, United Kingdom, RG24 9NA
Novo Nordisk Investigational Site
Birmingham, United Kingdom, B4 6NH
Novo Nordisk Investigational Site
Leicester, United Kingdom, LE1 5WW
Novo Nordisk Investigational Site
London, United Kingdom, SE1 7EH
Novo Nordisk Investigational Site
Oxford, United Kingdom, OX3 9DU
Sponsors and Collaborators
Novo Nordisk A/S

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 )
First Posted: November 8, 2011    Key Record Dates
Results First Posted: November 17, 2017
Last Update Posted: December 21, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: According to the Novo Nordiskdisclosure commitment on novonordisk-trials.com
URL: http://

Studies a U.S. FDA-regulated Drug Product: Yes
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