A Multi-centre, Open-label Trial Evaluating Efficacy, Safety and Pharmacokinetics of Turoctocog Alfa Pegol (N8-GP) When Used for Treatment and Prophylaxis of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A

NN7088-4595

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.

2021-09-27

2022-12-28

2022-12-28

Completed

Early phase I

36

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.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 36, 'type': 'ACTUAL'}}

2024-01-18