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

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ClinicalTrials.gov Identifier: NCT01493778
Recruitment Status : Active, not recruiting
First Posted : December 16, 2011
Last Update Posted : July 4, 2018
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Brief Summary:
This trial is conducted in Asia, Europe and North 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 or disease Intervention/treatment Phase
Congenital Bleeding Disorder Haemophilia A Drug: turoctocog alfa Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Intervention Model: Single Group Assignment
Masking: None (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
Actual Study Start Date : September 17, 2012
Actual Primary Completion Date : August 16, 2017
Estimated Study Completion Date : December 10, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bleeding Hemophilia

Arm Intervention/treatment
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.




Primary Outcome Measures :
  1. 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) ]

Secondary Outcome Measures :
  1. 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) ]
  2. 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) ]
  3. 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) ]
  4. 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) ]
  5. 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) ]
  6. 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 ]


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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:

  • 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) including commercially available NovoEight® /Novoeight®

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)

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): NCT01493778


  Show 69 Study Locations
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S

Additional Information:
Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01493778     History of Changes
Other Study ID Numbers: NN7008-3809
U1111-1119-6116 ( Other Identifier: WHO )
2011-001033-16 ( EudraCT Number )
P/50/2010 ( Other Identifier: EMA (PDCO) )
JapicCTI-142544 ( Other Identifier: JAPIC )
CTR20150455 ( Registry Identifier: ChinaDrugTrials )
First Posted: December 16, 2011    Key Record Dates
Last Update Posted: July 4, 2018
Last Verified: July 2018

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