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A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A (pathfinder8)

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ClinicalTrials.gov Identifier: NCT03528551
Recruitment Status : Enrolling by invitation
First Posted : May 18, 2018
Last Update Posted : December 13, 2018
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Brief Summary:
This study will look at how a known study medicine N8-GP works in previously N8-GP treated people with haemophilia A. The aim is to look at how N8-GP works during regular use. Participants will get N8-GP. N8-GP has been tested in more than 200 people with haemophilia A for several years. Participants will get an injection of N8-GP into a blood vessel, one, two or three times weekly. Participants will get more doses if they bleed or if they will need a surgery. The study will last for about 2 years. Participants will have at least 9 visits with the study doctor. If participants agree to be in this study, they will get their first injection (in this study) at the first visit. Participants will also get an injection at visit 3, 5 and 7. Participants will be trained to give all other injections themselves. Participants must not use any clotting factors other than N8-GP or any anticoagulants (blood thinners) during the study.

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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 173 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Prophylaxis and Treatment of Bleeds in Previously N8-GP Treated Patients With Severe Haemophilia A
Actual Study Start Date : April 30, 2018
Estimated Primary Completion Date : December 3, 2020
Estimated Study Completion Date : December 3, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arm Intervention/treatment
Experimental: N8-GP, once weekly
All participants will receive turoctocog alfa pegol (N8-GP) once weekly.
Drug: Turoctocog alfa pegol
Turoctocog alfa pegol 75 IU/kg body weight will be administered once weekly as intravenous injections for a duration of 2 years.

Experimental: N8-GP, twice weekly
All participants will receive N8-GP twice weekly.
Drug: Turoctocog alfa pegol
Turoctocog alfa pegol 60 IU/kg body weight (for patients younger than 12 years) and 50 IU/kg body weight (for patients, 12 years or older) will be administered twice weekly as intravenous injections for a duration of 2 years.

Experimental: N8-GP, three times weekly
All participants will receive N8-GP three times weekly.
Drug: Turoctocog alfa pegol
Turoctocog alfa pegol 50 IU/kg body weight will be administered three times weekly as intravenous injections for a duration of 2 years.




Primary Outcome Measures :
  1. Number of adverse events reported [ Time Frame: Weeks 0-104 ]
    Count of adverse events


Secondary Outcome Measures :
  1. Incidence of FVIII inhibitors ≥0.6 BU [ Time Frame: Weeks 0-104 ]
    Count of presence of inhibitors

  2. Number of bleeding episodes on prophylaxis [ Time Frame: Weeks 0-104 ]
    Count of episodes

  3. Number of spontaneous bleeding episodes on prophylaxis [ Time Frame: Weeks 0-104 ]
    Count of episodes

  4. Haemostatic effect of N8-GP when used for treatment of bleeding episodes assessed as: Excellent, Good, Moderate, or None [ Time Frame: Weeks 0-104 ]
    Assessed as: Excellent, good, moderate, or none

  5. Number of N8-GP injections required per bleeding episode [ Time Frame: Weeks 0-104 ]
    Number of injections

  6. Pre-dose FVIII activity levels on N8-GP prophylaxis (IU/dL) [ Time Frame: Weeks 0-104 ]
    Calculated based on plasma FVIII activity measured in blood

  7. Change in joint health status from start to end of trial (based on Haemophilia Joint Health Score) [ Time Frame: Week 0, Week 104 ]
    Based on Haemophilia Joint Health Score

  8. Haemostatic response during major surgical interventions assessed as: Excellent, Good, Moderate, or None [ Time Frame: Weeks 0-104 ]
    Assessed as: Excellent, good, moderate, or none

  9. Consumption of N8-GP per bleed [ Time Frame: Weeks 0-104 ]
    Number of infusions and IU/kg per bleed

  10. Consumption of N8-GP during prophylaxis treatment [ Time Frame: Weeks 0-104 ]
    Number of infusions and IU/kg per month and per year

  11. Change from start till end of trial in treatment satisfaction (based on Hemo-SAT score) [ Time Frame: Week 0, Week 104 ]
    Based on Haemophilia Satisfaction Survey (Hemo-SAT) score



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male patients of all ages with the diagnosis of severe congenital haemophilia A (coagulation Factor VIII [FVIII] activity less than 1%) based on medical records
  • On-going participation in NN7088-3859 (pathfinder2), or NN7088-3885 (pathfinder5) at the time of transfer

Exclusion Criteria:

  • Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products
  • Any disorder, except for conditions associated with haemophilia, which in the investigator's opinion might jeopardise patient's safety or compliance with the protocol - Current participation in any clinical trial (except NN7088-3859 (pathfinder2) or NN7088-3885 (pathfinder5)) of an approved or non-approved investigational medicinal product

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): NCT03528551


  Show 66 Study Locations
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Clinical Reporting Anchor and Disclosure 1452 Novo Nordisk A/S

Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT03528551     History of Changes
Other Study ID Numbers: NN7088-4410
U1111-1202-2780 ( Other Identifier: World Health Organization (WHO) )
2017-003788-36 ( Registry Identifier: European Medicines Agency (EudraCT) )
JapicCTI-183952 ( Registry Identifier: JAPIC )
First Posted: May 18, 2018    Key Record Dates
Last Update Posted: December 13, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
URL: http://www.novonordisk-trials.com/website/content/how-to-access-clinical-trial-datasets.aspx

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

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