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

  Show 47 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: 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
Hemostatic Disorders
Hemorrhagic Disorders
Hemophilia A
Hemorrhage
Hematologic Diseases
Vascular Diseases
Cardiovascular Diseases
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Pathologic Processes
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 20, 2014