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A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A (pathfinder™5)

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01731600
First Posted: November 22, 2012
Last Update Posted: October 31, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Novo Nordisk A/S
  Purpose
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products.

Condition Intervention Phase
Congenital Bleeding Disorder Haemophilia A Drug: turoctocog alfa pegol Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A

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 VIII (FVIII) equal to or above 0.6 Bethesda units [ Time Frame: During the main phase of the trial (from 0-26 weeks of treatment) ]

Secondary Outcome Measures:
  • Frequency of adverse events including serious adverse events reported during the trial period [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ]
  • Haemostatic effect of N8-GP when used for treatment of bleeding episodes and assessed as: Excellent, Good, Moderate, or None [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ]
  • Number of bleeding episodes during prophylactic treatment with N8-GP (annualised bleeding rate) [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ]
  • Consumption of N8-GP per bleeding episode (number of injections) [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ]
  • Consumption of N8-GP per bleeding episode (U/kg) [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ]
  • Consumption of N8-GP during prophylaxis (number of injections) [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ]
  • Consumption of N8-GP during prophylaxis ( U/kg per month and year) [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ]
  • Incremental recovery (defined as the peak level recorded 60 min after end of injection) evaluated for FVIII product [ Time Frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product. ]
  • Incremental recovery (defined as the peak level recorded 60 min after end of injection) evaluated for N8-GP [ Time Frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP ]
  • Area under the curve evaluated for FVIII product [ Time Frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product. ]
  • Area under the curve evaluated for N8-GP [ Time Frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP ]
  • Terminal half-life evaluated for FVIII product [ Time Frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product. ]
  • Terminal half-life evaluated for N8-GP [ Time Frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP ]
  • Clearance evaluated for FVIII product [ Time Frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product. ]
  • Clearance evaluated for N8-GP [ Time Frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP ]

Enrollment: 68
Actual Study Start Date: February 20, 2013
Estimated Study Completion Date: May 11, 2018
Estimated Primary Completion Date: May 11, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: N8-GP Drug: turoctocog alfa pegol
Fixed dose of turoctocog alfa pegol for intravenous injections (i.v.) twice weekly for prophylaxis. In addition, turoctocog alfa pegol will be administered to treat bleeding episodes during the trial period. Bleeding episodes will be treated with doses of 20-75 U/kg body weight.
Other Names:
  • NNC 0129-0000-1003
  • N8-GP

  Eligibility

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   up to 11 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male patients with severe congenital haemophilia A (FVIII activity level below 1%)
  • Weight above or equal to 10 kg
  • Documented history of 150 exposure days (ED) to FVIII products for patients aged 6-11 years and above 50 ED to FVIII products for patients aged 0-5 years

Exclusion Criteria:

  • Any history of FVIII inhibitors
  Contacts and Locations
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): NCT01731600


  Hide Study Locations
Locations
United States, Arizona
Novo Nordisk Investigational Site
Phoenix, Arizona, United States, 85016-7710
United States, California
Novo Nordisk Investigational Site
Long Beach, California, United States, 90806
Novo Nordisk Investigational Site
Orange, California, United States, 92868
United States, District of Columbia
Novo Nordisk Investigational Site
Washington, D.C., District of Columbia, United States, 20010-2978
United States, Florida
Novo Nordisk Investigational Site
Orlando, Florida, United States, 32827
Novo Nordisk Investigational Site
Tampa, Florida, United States, 33607
United States, Idaho
Novo Nordisk Investigational Site
Boise, Idaho, United States, 83712
United States, Iowa
Novo Nordisk Investigational Site
Iowa City, Iowa, United States, 52242
United States, Louisiana
Novo Nordisk Investigational Site
New Orleans, Louisiana, United States, 70118-5720
United States, Massachusetts
Novo Nordisk Investigational Site
Boston, Massachusetts, United States, 02115
United States, Minnesota
Novo Nordisk Investigational Site
Minneapolis, Minnesota, United States, 55404
United States, Nebraska
Novo Nordisk Investigational Site
Omaha, Nebraska, United States, 68198-2168
United States, New York
Novo Nordisk Investigational Site
New Hyde Park, New York, United States, 11040
United States, North Carolina
Novo Nordisk Investigational Site
Charlotte, North Carolina, United States, 28204
United States, Ohio
Novo Nordisk Investigational Site
Cincinnati, Ohio, United States, 45229
Novo Nordisk Investigational Site
Dayton, Ohio, United States, 45404
United States, Pennsylvania
Novo Nordisk Investigational Site
Philadelphia, Pennsylvania, United States, 19104
Novo Nordisk Investigational Site
Philadelphia, Pennsylvania, United States, 19134
United States, South Carolina
Novo Nordisk Investigational Site
Charleston, South Carolina, United States, 29425
United States, Tennessee
Novo Nordisk Investigational Site
Nashville, Tennessee, United States, 37232
United States, Texas
Novo Nordisk Investigational Site
Dallas, Texas, United States, 75235
Novo Nordisk Investigational Site
Houston, Texas, United States, 77030
United States, Virginia
Novo Nordisk Investigational Site
Charlottesville, Virginia, United States, 22903
United States, Washington
Novo Nordisk Investigational Site
Spokane, Washington, United States, 99204
Brazil
Novo Nordisk Investigational Site
Rio de Janeiro, Brazil, 20211-030
Canada, Ontario
Novo Nordisk Investigational Site
Toronto, Ontario, Canada, M5G 1X8
France
Novo Nordisk Investigational Site
Bron Cedex, France, 69677
Novo Nordisk Investigational Site
Lille, France, 59097
Novo Nordisk Investigational Site
Paris, France, 75015
Greece
Novo Nordisk Investigational Site
Athens, Greece, GR-11527
Novo Nordisk Investigational Site
Thessaloniki, Greece, GR 54642
Israel
Novo Nordisk Investigational Site
Tel-Hashomer, Israel, 52621
Italy
Novo Nordisk Investigational Site
Vicenza, Italy, 36100
Japan
Novo Nordisk Investigational Site
Kitakyusyu, Fukuoka, Japan, 8078555
Novo Nordisk Investigational Site
Suginami-ku, Tokyo, Japan, 167 0035
Lithuania
Novo Nordisk Investigational Site
Vilnius, Lithuania, LT-08406
Malaysia
Novo Nordisk Investigational Site
Kuala Lumpur, Malaysia, 50400
Portugal
Novo Nordisk Investigational Site
Porto, Portugal, 4200-319
Puerto Rico
Novo Nordisk Investigational Site
San Juan, Puerto Rico, 00935
Switzerland
Novo Nordisk Investigational Site
Bellinzona, Switzerland, 6500
Novo Nordisk Investigational Site
Luzern 16, Switzerland, 6000
Novo Nordisk Investigational Site
Zürich, Switzerland, 8032
Turkey
Novo Nordisk Investigational Site
Antalya, Turkey, 01010
Novo Nordisk Investigational Site
Bornova-IZMIR, Turkey, 35100
Novo Nordisk Investigational Site
Izmit, Turkey, 41380
Novo Nordisk Investigational Site
Samsun, Turkey
Ukraine
Novo Nordisk Investigational Site
Donetsk, Ukraine, 83045
Novo Nordisk Investigational Site
Lviv, Ukraine, 79044
United Kingdom
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
Investigators
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  More Information

Additional Information:
Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01731600     History of Changes
Other Study ID Numbers: NN7088-3885
U1111-1129-6009 ( Other Identifier: WHO )
2012-001711-23 ( EudraCT Number )
JapicCTI-132214 ( Registry Identifier: JAPIC )
First Submitted: November 9, 2012
First Posted: November 22, 2012
Last Update Posted: October 31, 2017
Last Verified: October 2017

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders
Hemostatic Disorders
Blood Coagulation Disorders, Inherited
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Vascular Diseases
Cardiovascular Diseases
Factor VIII
Coagulants