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Safety and Efficacy of NNC 0155-0000-0004 in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A (guardian™4)

This study is currently recruiting participants.
Verified February 2013 by Novo Nordisk
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk
ClinicalTrials.gov Identifier:
NCT01493778
First received: December 15, 2011
Last updated: February 18, 2013
Last verified: February 2013
History of Changes
  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 NNC 0155-0000-0004 in prevention and treatment of bleeds in previously untreated children with Haemophilia A.


Condition Intervention Phase
Congenital Bleeding Disorder
Haemophilia A
 Drug: NNC 0155-0000-0004
 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 NNC 0155-0000-0004 in Prevention and Treatment of Bleeds in Paediatric Previously Untreated Patients With Haemophilia A

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Novo Nordisk:

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, end of trial (exposure day 50-55, expected to occur between 9 and 24 months of trial participation) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Haemostatic effect of NNC 0155-0000-0004 on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None [ Time Frame: From Visit 2 (day 14 plus/minus 7 days) to Visit 5, end of trial (exposure day 50-55, expected to occur between 9 and 24 months of trial participation) ] [ Designated as safety issue: No ]
  • Annualized bleeding rate [ Time Frame: From Visit 2 (day 14 plus/minus 7 days) to Visit 5, end of trial (exposure day 50-55, expected to occur between 9 and 24 months of trial participation) ] [ Designated as safety issue: No ]

Estimated Enrollment: 60
Study Start Date: September 2012
Estimated Study Completion Date: September 2016
Estimated Primary Completion Date: September 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NNC 0155-0000-0004 Drug: NNC 0155-0000-0004
Patients will be scheduled to receive treatment with NNC 0155-0000-0004 for at least 50 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:

  • 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 with congenital severe haemophilia A (baseline level Factor VIII below or equal to 1%)
  • No prior use of purified clotting factor products (previous exposure to blood components is acceptable)

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
  • Factor VIII inhibitor (above or equal to 0.6 BU (Bethesda Units)/mL) at screening
  • Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG), routine systemic corticosteroids)
  • Platelet count below 50,000 platelets/mcL
  Contacts and Locations
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
Investigators
Study Director: Pär Ottosson Novo Nordisk
Study Director: Anne Krøger Lemme Novo Nordisk
  More Information

Additional Information:
Clinical Trials at Novo Nordisk  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Novo Nordisk
ClinicalTrials.gov Identifier: NCT01493778     History of Changes
Other Study ID Numbers: NN7008-3809, U1111-1119-6116, 2011-001033-16, P/50/2010
Study First Received: December 15, 2011
Last Updated: February 18, 2013
Health Authority: Austria: Agency for Health and Food Safety
Brazil: National Health Surveillance Agency
China: Ministry of Health
Greece: Ministry of Health
Hong Kong: Department of Health
Japan: Ministry of Health, Labor and Welfare
South Korea: Korea Food and Drug Administration (KFDA)
Malaysia: Ministry of Health
Russia: Federal Service for Control of Health Care and Social Development
Serbia: Agency for Drugs and Medicinal Devices
Spain: Spanish Agency of Medicines and Health Care Products
Thailand: Thai FDA
Turkey: Ministry of Health
United Kingdom: Medicines and Healthcare Regulatory Authority (MHRA)
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 May 19, 2013