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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: March 17, 2017
Last verified: March 2017
  Purpose
This trial is conducted globally. The aim of the 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: turoctocog alfa pegol
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: No masking
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 ]
  • 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 ]

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 ]

Enrollment: 186
Actual Study Start Date: January 30, 2012
Estimated Study Completion Date: December 3, 2018
Estimated Primary Completion Date: December 3, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Prophylaxis Drug: turoctocog alfa pegol
Administered i.v.
Other Name: NNC 0129-0000-1003
Experimental: On-demand Drug: turoctocog alfa pegol
Administered i.v.
Other Name: NNC 0129-0000-1003

  Eligibility

Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes 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 93 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:
Publications:
Giangrande P, Chowdary P, Enhrenforth S, Hanabusa H, Leebeek FW, Lentz SR, Nemes L, Poulsen LH, Santagostino E, You CW, Clausen WHO, Oldenburg J and on behalf of for the pathfinder™2 Investigators. Clinical evaluation of novel recombinant glycopegylated FVIII (turoctocog alfa pegol, N8-GP): efficacy and safety in previously treated patients with severe hemophilia A - results of pathfinder™2 international trial. Journal of Thrombosis and Haemostasis (Abstracts) 2015; 13 (Supplement S2): 1-997 [OR212]

Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01480180     History of Changes
Other Study ID Numbers: NN7088-3859
U1111-1119-7416 ( Other Identifier: WHO )
2011-001142-15 ( EudraCT Number )
JapicCTI-121749 ( Other Identifier: Japic )
Study First Received: November 23, 2011
Last Updated: March 17, 2017

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

ClinicalTrials.gov processed this record on March 23, 2017