Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A (pathfinder™2)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01480180
First received: November 23, 2011
Last updated: July 22, 2014
Last verified: July 2014
  Purpose

This trial is conducted globally. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in subjects with Haemophilia A.


Condition Intervention Phase
Congenital Bleeding Disorder
Haemophilia A
Drug: NNC 0129-0000-1003
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multi-national Trial Evaluating Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Patients With Haemophilia A

Resource links provided by NLM:


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

Primary Outcome Measures:
  • The Incidence rate of FVIII-inhibitors greater than or equal to 0.6 BU (Bethesda Unit) [ Time Frame: The endpoints will be analysed based on all available information after approximately 24 and 36 months and until the end of trial (EOT) visit ] [ Designated as safety issue: No ]
  • Annualised bleeding rate in the prophylaxis arm [ Time Frame: The endpoints will be analysed based on all available information after approximately 24 and 36 months and until the end of trial (EOT) visit ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Haemostatic effect of N8-GP when used for treatment of bleeds, assessed on a four-point scale (excellent, good, moderate and none) [ Time Frame: The endpoints will be analysed based on all available information until the end of trial (EOT) visit and up to approximately 24 and 36 months ] [ Designated as safety issue: No ]

Enrollment: 186
Study Start Date: January 2012
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Prophylaxis Drug: NNC 0129-0000-1003
Administered i.v.
Experimental: On-demand Drug: NNC 0129-0000-1003
Administered i.v.

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male patients with severe congenital haemophilia A (FVIII activity below 1%, according to medical records)
  • Documented history of at least 150 EDs (exposure days) to other FVIII products
  • At least 12 years and body weight at least 35 kg (except for Croatia, France, Russia, Israel and the Netherlands where the lower age limit will be 18 years)

Exclusion Criteria:

  • Previous participation in this trial defined as withdrawal after administration N8-GP
  • Any history of FVIII inhibitors
  • FVIII inhibitors above or equal to 0.6 BU/mL at screening
  • HIV (human immunodeficiency virus) positive, defined by medical records with CD4+ (T-lymphocyte subtype) count below or equal to 200/mcL or a viral load of more than 400000 copies/mL. If the data is not available in medical records within last 6 months, CD4+ will be measured at the screening visit
  • Congenital or acquired coagulation disorders other than haemophilia A
  • Previous significant thromboembolic events (e.g. myocardial infarction, cerebrovascular disease or deep venous thrombosis) as defined by available medical records
  • Platelet count below 50,000 platelets/mcL (laboratory value at the screening visit)
  • ALAT (alanine aminotransferase) above 3 times the upper limit of normal reference ranges at central laboratory
  • Creatinine level equal to or greater than 1.5 times above upper normal limit (according to central laboratory reference ranges)
  • Ongoing immune modulating or chemotherapeutic medication
  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: NCT01480180

  Hide Study Locations
Locations
United States, Arizona
Novo Nordisk Clinical Trial Call Center
Phoenix, Arizona, United States, 85016-7710
United States, California
Novo Nordisk Clinical Trial Call Center
Long Beach, California, United States, 90806
Novo Nordisk Clinical Trial Call Center
Sacramento, California, United States, 95817
Novo Nordisk Clinical Trial Call Center
Torrance, California, United States, 90502-2004
United States, District of Columbia
Novo Nordisk Clinical Trial Call Center
Washington, District of Columbia, United States, 20010-2978
United States, Florida
Novo Nordisk Clinical Trial Call Center
Orlando, Florida, United States, 32827
Novo Nordisk Clinical Trial Call Center
Tampa, Florida, United States, 33607
United States, Georgia
Novo Nordisk Clinical Trial Call Center
Augusta, Georgia, United States, 30912
United States, Iowa
Novo Nordisk Clinical Trial Call Center
Iowa City, Iowa, United States, 52242
United States, Louisiana
Novo Nordisk Clinical Trial Call Center
New Orleans, Louisiana, United States, 70118-5720
United States, Maryland
Novo Nordisk Clinical Trial Call Center
Baltimore, Maryland, United States, 21287
United States, Michigan
Novo Nordisk Clinical Trial Call Center
Detroit, Michigan, United States, 48201
Novo Nordisk Clinical Trial Call Center
East Lansing, Michigan, United States, 48823
United States, Minnesota
Novo Nordisk Clinical Trial Call Center
Minneapolis, Minnesota, United States, 55404
United States, Nebraska
Novo Nordisk Clinical Trial Call Center
Omaha, Nebraska, United States, 68198-5456
United States, New Jersey
Novo Nordisk Clinical Trial Call Center
Newark, New Jersey, United States, 07102
United States, Ohio
Novo Nordisk Clinical Trial Call Center
Cincinnati, Ohio, United States, 45229
Novo Nordisk Clinical Trial Call Center
Dayton, Ohio, United States, 45404
United States, Oregon
Novo Nordisk Clinical Trial Call Center
Portland, Oregon, United States, 97239
United States, Pennsylvania
Novo Nordisk Clinical Trial Call Center
Philadelphia, Pennsylvania, United States, 19134
United States, South Carolina
Novo Nordisk Clinical Trial Call Center
Charleston, South Carolina, United States, 29425
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
United States, Virginia
Novo Nordisk Clinical Trial Call Center
Charlottesville, Virginia, United States, 22908
Novo Nordisk Clinical Trial Call Center
Norfolk, Virginia, United States, 23507
United States, Washington
Novo Nordisk Clinical Trial Call Center
Spokane, Washington, United States, 99204
Australia, Victoria
Parkville, Victoria, Australia, 3052
Brazil
Campinas, Sao Paulo, Brazil, 13081970
Croatia
Zagreb, Croatia, 10 000
Denmark
Århus N, Denmark, 8200
France
Kremlin-Bicêtre, France, 94270
Germany
Hannover, Germany, 30625
Hungary
Debrecen, Hungary, 4012
Israel
Tel-Hashomer, Israel, 52621
Italy
Milano, Italy, 20124
Japan
Hiroshima-shi, Hiroshima, Japan, 734 8551
Korea, Republic of
Daejeon, Korea, Republic of, 302-799
Malaysia
Kuala Lumpur, Malaysia, 50400
Netherlands
Groningen, Netherlands, 9713 GZ
Norway
Oslo, Norway, 0027
Russian Federation
Saint-Petersburg, Russian Federation, 191186
Spain
Madrid, Spain, 28046
Sweden
Malmö, Sweden, 214 28
Switzerland
Zürich, Switzerland, 8091
Taiwan
Taipei, Taiwan, 100
Turkey
Adana, Turkey, 01130
United Kingdom
London, United Kingdom, SE1 7EH
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: NCT01480180     History of Changes
Other Study ID Numbers: NN7088-3859, U1111-1119-7416, 2011-001142-15, JapicCTI-121749
Study First Received: November 23, 2011
Last Updated: July 22, 2014
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
Brazil: National Health Surveillance Agency
Croatia: Ministry of Health and Social Care
Denmark: Danish Medicines Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medical Devices
Hungary: National Institute of Pharmacy
Italy: AIFA, National Medicines Agency
Japan: Ministry of Health, Labor and Welfare
Malaysia: Drug Control Authority (DCA)
Netherlands: Medicines Evaluation Board
Norway: Norwegian Medicines Control Authority
Spain: Spanish Agency of Medicines and Health Care Products
South Korea: Korea Food and Drug Administration (KFDA)
Sweden: Medical Products Agency
Switzerland: Federal Office of Public Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
United States: Food and Drug Administration
Taiwan: Department of Health
Turkey: Ministry of Health Drug and Pharmaceutical Department
Russia: Federal Service for Control of Health Care and Social Development
Israel: Ministry of Health

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

ClinicalTrials.gov processed this record on October 22, 2014