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)

Conditions: Haemophilia A

Summary: 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.


Eligibility 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 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.