None listed
Conditions
Interventions
Drug : • If the subject has not had prophylaxis before the study, the subject will get Mim8 once a week for 26 weeks (Arm 1) or for 52 weeks (Arm 2).
• If the subject has had prophylaxis before the s
Sponsors
Yonsei University Health System, Severance Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. 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 2. Male or female with diagnosis of congenital haemophilia A of any severity based on medical records 3. Patient has been prescribed, or in need of, treatment with factor VIII 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 =30 kg 6. Applicable to patients treated on-demand/with no prophylaxis: =5 bleeds in the last 26 weeks prior to screening visit 7. Applicable to patients with FVIII activity >1% who are on prophylactic treatment: =1 bleed in the last 26 weeks prior to screening visit 8. Willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires
Exclusion criteria
Exclusion criteria: 1. Previous participation in this trial. Participation is defined as signed informed consent 2. 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 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 patients 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 (highly effective contraceptive measures as defined in protocol Section 10.4 or as required by local regulation or practice). 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 subject’s safety or compliance with the protocol 6. Known or suspected hypersensitivity to trial 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. For definition of major surgery see protocol Table 6 7 10. Known congenital or acquired coagulation disorders other than haemophilia A 11. Hepatic dysfunction defined as AST and/or ALT >3 times the upper limit combined with total bilirubin >1.5 times the upper limit measured at screening 12. Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) =30 ml/min/1.73 m2 for serum creatinine measured at screening 13. Previous or current thromboembolic disease or eventsa (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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of treated bleeds | — |
Secondary
| Measure | Time frame |
|---|---|
| All endpoints related to subgroups of bleeds | — |
Countries
Korea, Republic of
Contacts
Public ContactSeung Min Hahn
Yonsei University Health System, Severance Hospital
Outcome results
None listed