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Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A (pathfinder™6)

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.
 
ClinicalTrials.gov Identifier: NCT02137850
Recruitment Status : Active, not recruiting
First Posted : May 14, 2014
Last Update Posted : October 19, 2020
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Brief Summary:
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.

Condition or disease Intervention/treatment Phase
Congenital Bleeding Disorder Haemophilia A Drug: turoctocog alfa pegol Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 125 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Single-arm Multicentre Non-controlled Phase 3a Trial Investigating Safety and Efficacy of N8-GP in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Paediatric Patients With Severe Haemophilia A
Actual Study Start Date : June 26, 2014
Estimated Primary Completion Date : November 13, 2021
Estimated Study Completion Date : November 13, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arm Intervention/treatment
Experimental: 50 EDs (exposure days) Drug: turoctocog alfa pegol
For intravenous (i.v.) injection. Frequency and dosage (20-75 U/kg) dependent on whether given as treatment for bleeding episode or as prophylaxis
Other Names:
  • NNC 0129-0000-1003
  • N8-GP




Primary Outcome Measures :
  1. Incidence of inhibitory antibodies against coagulation factor VIII (FVIII) [ Time Frame: When the first 50 PUP have reached at least 50 exposure dates. (Expected to reach between 6 - 18 months) ]
  2. Incidence of inhibitory antibodies against coagulation factor VIII (FVIII) [ Time Frame: At the end of the trial. End of trial will be up to 4 years after the patient has reached 100 exposure dates. (Expected to reach between 12 - 60 months) ]

Secondary Outcome Measures :
  1. Frequency of adverse events including serious adverse events and medical events of special interest [ Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days ]
  2. Incidence of confirmed high titre inhibitors (defined as inhibitor titre above 5 Bethesda Units (BU) [ Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days ]
  3. Number of breakthrough bleeding episodes during prophylaxis with turoctocog alfa pegol (N8-GP) (annualised bleeding rate) [ Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days ]
  4. Haemostatic effect of N8-GP in treatment of bleeding episodes, assessed by a predefined 4-point haemostatic response scale ("excellent", "good", "moderate" and "none") [ Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days ]
  5. Consumption of N8-GP for prophylaxis (number of injections and U/Kg per month and per year) [ Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days ]
  6. Consumption of N8-GP for treatment of bleeding episodes (number of injections and U/Kg required per bleeding episode) [ Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days ]
  7. Total consumption of N8-GP per patient (prevention and treatment of bleeding episodes) per month and annualised value [ Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days ]
  8. Outcome of immune tolerance induction (ITI), assessed by a predefined 4-point ITI outcome scale ("success", "partial success", "failure", "other") [ Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days ]


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Ages Eligible for Study:   up to 6 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
  • Male, age below 6 years of age at the time of signing informed consent
  • Diagnosis of severe haemophilia A (FVIII activity level 1%) based on medical records or central laboratory results
  • No prior use of purified clotting factor products (5 previous exposures to blood components is acceptable)

Exclusion Criteria:

  • Any history of FVIII inhibitor (defined by medical records) - Known or suspected hypersensitivity to trial product or related products
  • Previous participation in this trial. Participation is defined as first dose administered of trial product
  • Receipt of any investigational medicinal product within 30 days before screening
  • Congenital or acquired coagulation disorder other than haemophilia A
  • Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise the patient's safety or compliance with the protocol
  • Patient's parent(s')/legally acceptable representative (LAR(s')) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation

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): NCT02137850


Locations
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Sponsors and Collaborators
Novo Nordisk A/S
Investigators
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Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
Additional Information:
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Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT02137850    
Other Study ID Numbers: NN7088-3908
2013-004025-88 ( EudraCT Number )
U1111-1148-1897 ( Other Identifier: WHO )
REec-2014-0898 ( Registry Identifier: Spanish Register of Clinical Studies (REec) )
JapicCTI-142577 ( Other Identifier: JAPIC )
First Posted: May 14, 2014    Key Record Dates
Last Update Posted: October 19, 2020
Last Verified: October 2020
Additional relevant MeSH terms:
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Hemostatic Disorders
Hemophilia A
Blood Coagulation Disorders
Hemorrhage
Pathologic Processes
Blood Coagulation Disorders, Inherited
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Vascular Diseases
Cardiovascular Diseases