Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A (guardian™ 3)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01138501
First received: May 28, 2010
Last updated: September 3, 2014
Last verified: September 2014
  Purpose

This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated paediatric subjects 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: A Multi-Centre, Open-Label, Non-Controlled Trial on Safety and Efficacy of N8 in Previously Treated Paediatric 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 Bethesda Units (BU)) [ Time Frame: The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject. ] [ Designated as safety issue: No ]
    The incidence rate of FVIII inhibitors was calculated by including all patients with inhibitors in the nominator and including all patients with a minimum 50 exposure plus any patients with less than 50 exposures but with inhibitors in denominator.


Secondary Outcome Measures:
  • Frequency of Adverse Events (AEs) [ Time Frame: The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject ] [ Designated as safety issue: No ]
    Adverse event was defined as events occurring after administration of trial product. Severe AEs: considerable interference with subject's daily activities, unacceptable. Moderate AEs: Marked symptoms, moderate interference with the patient's daily activities. Mild AEs: No or transient symptoms, no interference with the patient's daily activities. Serious AE: AE that at any dose results in any of the following: death, a life-threatening experience, in-subject hospitalization/prolongation of existing hospitalisation, persistent/significant disability/incapacity/congenital anomaly/birth defect.


Enrollment: 65
Study Start Date: June 2010
Study Completion Date: November 2011
Primary Completion Date: November 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: rFVIII Drug: turoctocog alfa
Subjects will be treated 3 times per week or every second day with intravenous injections of turoctocog alfa. The dose range for preventive treatment is 25-60 IU/kg body weight depending on treatment regimen.
Drug: turoctocog alfa
Subjects will undergo half-life evaluation of their current factor VIII product and pharmacokinetic session with turoctocog alfa before entering preventive treatment (subjects with available evaluation of terminal half-life within the last year are to be excluded from receiving factor VIII). Intravenous injections. The dose range for preventive treatment is 25-60 IU/kg body weight depending on treatment regimen.

  Eligibility

Ages Eligible for Study:   up to 12 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male patients with severe (baseline FVIII less than or equal to 1%) haemophilia A
  • Age below 12 years and weight at least 11 kg

Exclusion Criteria:

  • Surgery planned to occur during trial participation (exceptions are port placement, dental extractions, and minor, uncomplicated emergent procedures)
  • Congenital or acquired coagulation disorders other than haemophilia A
  • Any history of FVIII inhibitors (greater than or equal to 0.6 BU/mL)
  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: NCT01138501

  Show 20 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: NCT01138501     History of Changes
Obsolete Identifiers: NCT01250028
Other Study ID Numbers: NN7008-3545, U1111-1113-7182, 2009-016383-36
Study First Received: May 28, 2010
Results First Received: November 14, 2013
Last Updated: September 3, 2014
Health Authority: Brazil: National Health Surveillance Agency
Italy: Ministry of Health
Lithuania: Lithuanian Bioethics Committee
Macedonia, The Former Yugoslav Republic of: Ministry of Health of Republic of Macedonia
Malaysia: Ministry of Health
Poland: Ministry of Health
Russia: Federal Service for Control of Health Care and Social Development
Serbia: Agency for Drugs and Medicinal Devices
Taiwan: Department of Health
Turkey: Ministry of Health
United States: Food and Drug Administration

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
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 18, 2014