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)

Study Description

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Brief Summary:

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 or disease	Intervention/treatment	Phase
Congenital Bleeding Disorder; Haemophilia A	Drug: turoctocog alfa pegol	Phase 3

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	68 participants
Allocation:	Non-Randomized
Intervention Model:	Single Group Assignment
Masking:	None (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
Actual Study Start Date :	February 20, 2013
Actual Primary Completion Date :	September 15, 2014
Estimated Study Completion Date :	September 30, 2018

Arms and Interventions

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Arm	Intervention/treatment
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

Outcome Measures

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Primary Outcome Measures :

1. 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) ]

Secondary Outcome Measures :

1. 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) ]
2. 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) ]
3. 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) ]
4. 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) ]
5. 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) ]
6. 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) ]
7. 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) ]
8. 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. ]
9. 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 ]
10. 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. ]
11. 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 ]
12. 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. ]
13. 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 ]
14. 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. ]
15. Clearance evaluated for N8-GP [ Time Frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP ]

Eligibility Criteria

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Ages Eligible for Study:	up to 11 Years (Child)
Sexes 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

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  Show 51 Study Locations

Sponsors and Collaborators

Novo Nordisk A/S

Investigators

Study Director:	Global Clinical Registry (GCR, 1452)	Novo Nordisk A/S

More Information

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Additional Information:

Clinical Trials at Novo Nordisk 

Responsible Party:	Novo Nordisk A/S
ClinicalTrials.gov Identifier:	NCT01731600 History of Changes
Other Study ID Numbers:	NN7088-3885; U1111-1129-6009 ( Other Identifier: WHO ); 2012-001711-23 ( EudraCT Number ); JapicCTI-132214 ( Registry Identifier: JAPIC )
First Posted:	November 22, 2012
Last Update Posted:	April 9, 2018
Last Verified:	April 2018

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