Efficacy and Safety of Turoctocog Alfa Pegol (N8-GP) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A (pathfinder10) (Pathfinder10)

Novo Nordisk

Status and phase: Completed; Phase 3

Conditions: Haemophilia A

Treatments: Drug: turoctocog alfa pegol (N8-GP)

Study type: Interventional

Funder types: Industry

Identifiers

NCT05082116

NN7088-4595

2020-003001-58 (EudraCT Number)

U1111-1235-5905 (Other Identifier)

Details and patient eligibility

About

The study investigates how well the medicine called turoctocog alfa pegol (N8-GP) works in previously treated Chinese patients with severe haemophilia A.

Participants will be treated with N8-GP. This is a medicine that doctors can already prescribe in other countries.

The medicine will be injected into a vein (intravenous injections) and blood samples will be collected.

The study will last for about 7-8 months. Participants will have between 8 and 15 visits to the clinic and possibly a number of phone calls with the study doctor.

Enrollment: 36 patients

Sex: Male

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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 Chinese patient with severe congenital haemophilia A with a FVIII activity below 1% according to medical records.
• Aged greater than or equal to 12 years at the time of signing informed consent.
• History of at least 150 exposure days (EDs) to other FVIII products.
• The patient and/or caregiver is capable of assessing a bleeding episode, keeping a diary, performing home treatment of bleeding episodes and otherwise following the trial procedures at the discretion of the investigator.

Exclusion criteria

• Known or suspected hypersensitivity to trial product or related products.
• Previous participation in this trial. Participation is defined as signed informed consent.
• Participation in any clinical trial of an approved or non-approved investigational medicinal product within 5 half-lives or 30 days from screening, whichever is longer.
• Known history of FVIII inhibitors based on existing medical records, laboratory report reviews and patient and/or caregiver interviews.
• Current FVIII inhibitors greater than or equal to 0.6 BU.
• Congenital or acquired coagulation disorder other than haemophilia According to medical records.
• HIV positive, defined by medical records, with CD4+ count less than or equal 200/L and a viral load greater than 200 particles/μl or greater than 400000 copies/mL within 6 months of the trial entry. If the data are not available in medical records within last 6 months, then the test must be performed at screening visit.
• Previous significant thromboembolic events (e.g. myocardial infarction, cerebrovascular disease or deep venous thrombosis) as defined by available medical records.
• Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) greater than 3 times limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal at screening, as defined by central laboratory
• Renal impairment defined as estimated glomerular filtration rate (eGFR) below or equal to 30 mL/min/1.73 m^2 for serum creatinine measured at screening, as defined by central laboratory.
• Platelet count below 50×109/L at screening based on central laboratory values at screening.
• Ongoing immune modulating or chemotherapeutic medication.
• Any disorder, except for conditions associated with haemophilia A, which in the investigator's opinion might jeopardise the patient's safety or compliance with the protocol.
• Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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36 participants in 1 patient group

N8-GP prophylaxis

Experimental group

Description:

All patients will receive prophylaxis with 50 IU/kg N8-GP every 4 days for a treatment period of at least 28 weeks (with the possibility of switching to twice-weekly dosing during the treatment period at the discretion of the investigator).

Treatment:

Drug: turoctocog alfa pegol (N8-GP)