A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A - Full Text View

Purpose

This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products.

Condition	Intervention	Phase
Congenital Bleeding Disorder Haemophilia A	Drug: turoctocog alfa pegol	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 Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A

Resource links provided by NLM:

Genetics Home Reference related topics: hemophilia

MedlinePlus related topics: Hemophilia

Genetic and Rare Diseases Information Center resources: Hemophilia Hemophilia A

U.S. FDA Resources

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

Primary Outcome Measures:

Incidence of inhibitory antibodies against coagulation factor VIII (FVIII) equal to or above 0.6 Bethesda units [ Time Frame: During the main phase of the trial (from 0-26 weeks of treatment) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Frequency of adverse events including serious adverse events reported during the trial period [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ] [ Designated as safety issue: No ]
Haemostatic effect of N8-GP when used for treatment of bleeding episodes and assessed as: Excellent, Good, Moderate, or None [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ] [ Designated as safety issue: No ]
Number of bleeding episodes during prophylactic treatment with N8-GP (annualised bleeding rate) [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ] [ Designated as safety issue: No ]
Consumption of N8-GP per bleeding episode (number of injections) [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ] [ Designated as safety issue: No ]
Consumption of N8-GP per bleeding episode (U/kg) [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ] [ Designated as safety issue: No ]
Consumption of N8-GP during prophylaxis (number of injections) [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ] [ Designated as safety issue: No ]
Consumption of N8-GP during prophylaxis ( U/kg per month and year) [ Time Frame: For the main phase (from 0-26 weeks of treatment) and the extension phase of the trial (from 26 weeks to the last patient has completed the trial) ] [ Designated as safety issue: No ]
Incremental recovery (defined as the peak level recorded 60 min after end of injection) evaluated for FVIII product [ Time Frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product. ] [ Designated as safety issue: No ]
Incremental recovery (defined as the peak level recorded 60 min after end of injection) evaluated for N8-GP [ Time Frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP ] [ Designated as safety issue: No ]
Area under the curve evaluated for FVIII product [ Time Frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product. ] [ Designated as safety issue: No ]
Area under the curve evaluated for N8-GP [ Time Frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP ] [ Designated as safety issue: No ]
Terminal half-life evaluated for FVIII product [ Time Frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product. ] [ Designated as safety issue: No ]
Terminal half-life evaluated for N8-GP [ Time Frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP ] [ Designated as safety issue: No ]
Clearance evaluated for FVIII product [ Time Frame: 2-6 weeks prior to initial dosing with N8-GP and up to 30 hours after administration of previous FVIII product. ] [ Designated as safety issue: No ]
Clearance evaluated for N8-GP [ Time Frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP ] [ Designated as safety issue: No ]


Enrollment:	68
Study Start Date:	February 2013
Estimated Study Completion Date:	May 2018
Estimated Primary Completion Date:	May 2018 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: N8-GP	Drug: turoctocog alfa pegol Fixed dose of turoctocog alfa pegol for intravenous injections (i.v.) twice weekly for prophylaxis. In addition, turoctocog alfa pegol will be administered to treat bleeding episodes during the trial period. Bleeding episodes will be treated with doses of 20-75 U/kg body weight. Other Names: NNC 0129-0000-1003 N8-GP

Eligibility


Ages Eligible for Study:	up to 11 Years (Child)
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Male patients with severe congenital haemophilia A (FVIII activity level below 1%)
Weight above or equal to 10 kg
Documented history of 150 exposure days (ED) to FVIII products for patients aged 6-11 years and above 50 ED to FVIII products for patients aged 0-5 years

Exclusion Criteria:

Any history of FVIII inhibitors

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: NCT01731600

Show 38 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:

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


Responsible Party:	Novo Nordisk A/S
ClinicalTrials.gov Identifier:	NCT01731600 History of Changes
Other Study ID Numbers:	NN7088-3885 U1111-1129-6009 2012-001711-23 JapicCTI-132214
Study First Received:	November 9, 2012
Last Updated:	July 27, 2016
Health Authority:	Canada: Health Canada France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Germany: Paul-Ehrlich-Institut Greece: National Organization for Medicines Israel: Israeli Health Ministry Pharmaceutical Administration Italy: The Italian Medicines Agency Japan: Ministry of Health, Labor and Welfare Lithuania: Ministry of Health Malaysia: Ministry of Health Portugal: INFARMED Switzerland: Federal Office of Public Health Turkey: Ministry of Health Drug and Pharmaceutical Department Ukraine: Ministry of Health Ukraine United Kingdom: Medicines and Healthcare Products Regulatory Agency 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

ClinicalTrials.gov processed this record on September 27, 2016

A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A (pathfinder™5)