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

Inclusion criteria:
1. Informed consent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study
2. Male or female with diagnosis of congenital haemophilia A of any severity based on medical records
3. Participants has been prescribed treatment with factor VIII concentrates or bypassing agent in the last 26 weeks prior to screening
4. Age above or equal to 12 years at the time of signing informed consent.
5. Body weight above or equal to 30 kg
6. Applicable to participants treated with on-demand/no prophylaxis prior to enrolment: more than or equal to 5 bleeds in the last 26 weeks prior to screening visit, for which factor VIII concentrates or bypassing agent has been prescribed
7. Applicable to participants with FVIII activity above or equal to 1% who are on prophylactic treatment: more than or equal to 1 bleed in the last 26 weeks prior to screening visit, for which factor VIII concentrates or bypassing agent has been prescribed
8. 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 204
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 12

Exclusion criteria:
1. Previous participation in this study. Participation is defined as signed informed consent
2. Participation in any clinical study 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
3. Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months (or 5 half-lives of the medicinal product, whichever is shorter) prior to planned first dose, for participants not included in the run-in.
4. 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. Breast feeding is allowed only during the run-in period
5. Any disorder, except for conditions associated with haemophilia A, which in the investigator’s opinion might jeopardise participant’s safety or compliance with the protocol
6. Known or suspected hypersensitivity to study product(s), any constituents of the product or to related products
7. Receipt of gene therapy at any given time point
8. Ongoing or planned ITI therapy
9. Major surgery planned at the time of screening.
10. Known congenital or acquired coagulation disorders other than haemophilia A
11. Hepatic dysfunction defined as AST and/or ALT more than 3 times the upper limit combined with total bilirubin more than 1.5 times the upper limit measured at screening
12. Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) less than or equal to 30 ml/min/1.73 m^2 for serum creatinine measured at screening
13. Previous or current thromboembolic disease or events (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
14. Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation
15. Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis as evaluated by the investigator