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)
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ClinicalTrials.gov Identifier: NCT01731600 |
Recruitment Status
:
Active, not recruiting
First Posted
: November 22, 2012
Last Update Posted
: April 9, 2018
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Congenital Bleeding Disorder Haemophilia A | Drug: turoctocog alfa pegol | Phase 3 |
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 |

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:
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- 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) ]
- 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) ]
- 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) ]
- 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) ]
- 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) ]
- 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) ]
- 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) ]
- 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) ]
- 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. ]
- 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 ]
- 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. ]
- 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 ]
- 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. ]
- 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 ]
- 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. ]
- Clearance evaluated for N8-GP [ Time Frame: From 1 hour prior to and up to 96 hours after initial administration of N8-GP ]

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

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): NCT01731600

Study Director: | Global Clinical Registry (GCR, 1452) | Novo Nordisk A/S |
Additional Information:
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 Key Record Dates |
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 |