Eine Forschungsstudie zur Untersuchung von Mim8 bei Erwachsenen und Jugendlichen mit Hämophilie A mit oder ohne Hemmkörper

Product Code: NNC0365-3769 A
Pharmaceutical Form: Solution for injection
INN or Proposed INN: N/A
CAS Number: *MASKED*
Current Sponsor code: NNC0365-3769
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: range
Concentration number: 10-100

Gender:
Female: yes
Male: yes

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 or female with diagnosis of congenital haemophilia A of any severity based on medical records
- Patient has been prescribed, or in need of, treatment with factor VIII or bypassing agent in the last 26 weeks prior to screening
- Age above or equal to 12 years at the time of signing informed consent. Germany, Japan and Taiwan: Local requirements apply
- Body weight above or equal to 30 kg
- Applicable to patients on emicizumab prophylaxis: patient is willing to discontinue emicizumab at the time of screening
- Applicable to patients treated with no prophylaxis prior to enrolment: 5 or more bleeds in the last 26 weeks prior to screening visit
- Applicable to patients with FVIII activity above 1% who are on prophylactic treatment: 1 or more bleeds in the last 26 weeks prior to screening visit
- Willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 190
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion criteria:
- 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 30 days (or 5 half-lives of the investigational medicinal product, whichever is greater) before screening
- Female who is pregnant, breast-feeding or intends to become pregnant or is of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures as defined in the protocol or as required by local regulation or practice). Breast feeding is allowed only during the run-in period
- Any disorder, except for conditions associated with haemophilia A, which in the investigator?s opinion might jeopardise subject?s safety or compliance with the protocol
- Known or suspected hypersensitivity to trial product(s), any constituents of the product or to related products
- Receipt of gene therapy at any given time point
- Ongoing or planned immune tolerance induction (ITI) therapy
- Major surgery planned at the time of screening
- Known congenital or acquired coagulation disorders other than haemophilia A
- Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) above 3 times the upper limit combined with total bilirubin above 1.5 times the upper limit measured at screening
- 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
- Previous or current thromboembolic disease or events (includes arterial and venous thrombosis including myocardial infarction, thrombotic microangiography (TMA), pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion) (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or risk of thromboembolic disease, as evaluated by investigator
- Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation
- Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis as evaluated by the investigator