Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A

• Register: ClinicalTrials.gov
• Last refreshed on: 29 April 2024
• Main ID: NCT02137850
• Date of registration: 28/03/2014
• Prospective Registration: Yes
• Primary sponsor: Novo Nordisk A/S
• Public title: Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A pathfinder™6
• Scientific title: An Open-label Single-arm Multicentre Non-controlled Phase 3a Trial Investigating Safety and Efficacy of N8-GP in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Paediatric Patients With Severe Haemophilia A
• Date of first enrolment: June 26, 2014
• Target sample size: 125
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02137850
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Algeria, Argentina, Australia, Austria, Bulgaria, Canada, France, Germany, Greece, Israel, Italy, Japan, Malaysia, Romania, Spain, Taiwan, Thailand, United States

Contacts

• Name: Global Clinical Registry (GCR, 1452)
• Affiliation: Novo Nordisk A/S

Key inclusion & exclusion criteria

Inclusion Criteria:

- Informed consent obtained before any trial-related activities. Trial-related
activities are any procedures that are carried out as part of the trial, including
activities to determine suitability for the trial

- Male, age below 6 years of age at the time of signing informed consent

- Diagnosis of severe haemophilia A (FVIII activity level 1%) based on medical records
or central laboratory results

- No prior use of purified clotting factor products (5 previous exposures to blood
components is acceptable)

Exclusion Criteria:

- Any history of FVIII inhibitor (defined by medical records) - Known or suspected
hypersensitivity to trial product or related products

- Previous participation in this trial. Participation is defined as first dose
administered of trial product

- Receipt of any investigational medicinal product within 30 days before screening

- Congenital or acquired coagulation disorder other than haemophilia A

- Any chronic disorder or severe disease which, in the opinion of the Investigator,
might jeopardise the patient's safety or compliance with the protocol

- Patient's parent(s')/legally acceptable representative (LAR(s')) mental incapacity,
unwillingness to cooperate, or a language barrier precluding adequate understanding
and cooperation



Age minimum: 0 Years
Age maximum: 6 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Congenital Bleeding Disorder

Haemophilia A

Intervention(s)

Drug: turoctocog alfa pegol

Primary Outcome(s)

Incidence of inhibitory antibodies against coagulation factor VIII (FVIII) [Time Frame: When the first 50 PUP have reached at least 50 exposure dates. (Expected to reach between 6 - 18 months)]

Incidence of inhibitory antibodies against coagulation factor VIII (FVIII) [Time Frame: At the end of the trial. End of trial will be up to 4 years after the patient has reached 100 exposure dates. (Expected to reach between 12 - 60 months)]

Secondary Outcome(s)

Consumption of N8-GP for treatment of bleeding episodes (number of injections and U/Kg required per bleeding episode) [Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]

Total consumption of N8-GP per patient (prevention and treatment of bleeding episodes) per month and annualised value [Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]

Incidence of confirmed high titre inhibitors (defined as inhibitor titre above 5 Bethesda Units (BU) [Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]

Consumption of N8-GP for prophylaxis (number of injections and U/Kg per month and per year) [Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]

Haemostatic effect of N8-GP in treatment of bleeding episodes, assessed by a predefined 4-point haemostatic response scale ("excellent", "good", "moderate" and "none") [Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]

Number of breakthrough bleeding episodes during prophylaxis with turoctocog alfa pegol (N8-GP) (annualised bleeding rate) [Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]

Outcome of immune tolerance induction (ITI), assessed by a predefined 4-point ITI outcome scale ("success", "partial success", "failure", "other") [Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]

Frequency of adverse events including serious adverse events and medical events of special interest [Time Frame: When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]

Secondary ID(s)

JapicCTI-142577

2013-004025-88

REec-2014-0898

NN7088-3908

U1111-1148-1897

Source(s) of Monetary Support

Please refer to primary and secondary sponsors