Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A (guardian™4)

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2014 by Novo Nordisk A/S
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01493778
First received: December 15, 2011
Last updated: June 23, 2014
Last verified: June 2014
  Purpose

This trial is conducted in Asia, Europe and North and South America. The purpose of the trial is to evaluate the safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with haemophilia A.


Condition Intervention Phase
Congenital Bleeding Disorder
Haemophilia A
Drug: turoctocog alfa
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Paediatric Previously Untreated Patients With Haemophilia A

Resource links provided by NLM:


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

Primary Outcome Measures:
  • Incidence rate of Factor VIII inhibitors (above or equal to 0.6 BU (Bethesda Units)/mL) [ Time Frame: From Visit 2 to Visit 5, main phase of the trial (exposure day 50-55, expected to occur between 3 and 24 months of trial participation) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Haemostatic effect of turoctocog alfa on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None [ Time Frame: From Visit 2 to Visit 5, main phase of the trial (exposure day 50-55, expected to occur between 3 and 24 months of trial participation) ] [ Designated as safety issue: No ]
  • Haemostatic effect of turoctocog alfa on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None [ Time Frame: From Visit 6 to end of trial, extension phase of the trial (exposure day 100, expected to occur between 6 and 48 months of trial participation) ] [ Designated as safety issue: No ]
  • Haemostatic effect of turoctocog alfa on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None [ Time Frame: From Visit 2 to end of trial, the combined main and extension phases of the trial (exposure day 100, expected to occur between 6 and 48 months) ] [ Designated as safety issue: No ]
  • Annualized bleeding rate [ Time Frame: From Visit 2 to Visit 5, main phase of the trial (exposure day 50-55, expected to occur between 3 and 24 months of trial participation) ] [ Designated as safety issue: No ]
  • Annualized bleeding rate [ Time Frame: From Visit 6 to end of trial, the extension phase of the trial (exposure day 100, expected to occur between 6 and 48 months of trial participation) ] [ Designated as safety issue: No ]
  • Annualized bleeding rate [ Time Frame: From Visit 2 to end of trial, the combined main and extension phases of the trial (exposure day 100, expected to occur between 6 and 48 months of trial participation ] [ Designated as safety issue: No ]

Estimated Enrollment: 60
Study Start Date: September 2012
Estimated Study Completion Date: June 2018
Estimated Primary Completion Date: June 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: turoctocog alfa Drug: turoctocog alfa
Patients will be scheduled to receive treatment with turoctocog alfa for at least 100 exposure days. In most cases, treatment will be given at home with intravenous (i.v., into the vein) self-injection by the parent/caregiver/support person.

  Eligibility

Ages Eligible for Study:   up to 6 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age below 6 years
  • Informed consent obtained before any trial-related activities (trial-related activities are any procedure that would not have been performed during normal management of the patient)
  • Male patients diagnosed with congenital severe haemophilia A (FVIII level equal to or below 1%)
  • No prior use of purified clotting factor products (previous exposure, equal to or less than 5 ED to blood components, e.g. cryoprecipitate, fresh frozen plasma, is accepted)

Exclusion Criteria:

  • Known or suspected allergy to hamster protein or intolerance to trial product(s) or related products
  • Previous participation in this trial defined as withdrawal after administration of trial product
  • Congenital or acquired coagulation disorders other than haemophilia A
  • Any history of Factor VIII inhibitor
  • Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG), routine systemic corticosteroids)
  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: NCT01493778

Contacts
Contact: Novo Nordisk clinicaltrials@novonordisk.com

  Show 29 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: NCT01493778     History of Changes
Other Study ID Numbers: NN7008-3809, U1111-1119-6116, 2011-001033-16, P/50/2010, JapicCTI-142544
Study First Received: December 15, 2011
Last Updated: June 23, 2014
Health Authority: Austria: Agency for Health and Food Safety
China: Ministry of Health
Greece: Ministry of Health
Hong Kong: Department of Health
Japan: Ministry of Health, Labor and Welfare
Russia: Federal Service for Control of Health Care and Social Development
Spain: Spanish Agency of Medicines and Health Care Products
Turkey: Ministry of Health
United States: Food and Drug Administration

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 July 31, 2014