None listed
Conditions
Interventions
Drug : After confirmed eligibility (see Section 5), participants will switch to Mim8 prophylaxis (QW, Q2W, or QM) which they will receive for 26 weeks. Treatment frequency will be chosen based on pati
Sponsors
Novo Nordisk
Eligibility
Sex/Gender
All
Inclusion criteria
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. Age 12 years or above at the time of signing the informed consent. 4. Patients treated with emicizumab QW, Q2W, or Q4W according to the label for at least 8 weeks prior to screening. 5. Patients choosing to discontinue emicizumab treatment and switch to Mim8 QW, Q2W, or QM treatment for 26 weeks from start of treatment (Visit 2). 6. Participant and/or caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of an electronic diary and patient-reported outcomes (PRO) questionnaires.
Exclusion criteria
Exclusion criteria: 1. Participation (i.e., signed informed consent) in any interventional, clinical study, with the exception of emicizumab, with receipt of the last dose within 8 weeks (or 5 half-lives of the IMP, whichever is longer) before screening 2. Any disorder, which in the investigator’s opinion might jeopardise the participant’s compliance with the protocol or safety, including ongoing AEs associated with emicizumab. 3. Previous participation in this study. Participation is defined as signed informed consent. 4. Known congenital or acquired coagulation disorders other than haemophilia A 5. 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. 6. Neutralising antibodies towards emicizumab have been detected or, for patients adherent to emicizumab therapy, are suspected based on clinical and laboratory assessments. 7. Receipt of FVIII gene therapy at any time 8. Ongoing or planned immune tolerance induction therapy 9. Minor or major surgery planned to take place after screening and during the 26-week treatment period. 10. Known or suspected hypersensitivity to study intervention, related products, any constituents of the product or to other monoclonal antibodies 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. Female who is pregnant, breast-feeding or intends to become pregnant or is of childbearing potential and not using highly effective contraceptive method, as defined in Appendix 4 (Section 10.4). 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 treatment-emergent adverse events | — |
Secondary
| Measure | Time frame |
|---|---|
| Device handling experience using H-DAT questionnaire;Change in participants’ treatment burden using the Hemo-TEM total score;Mean change from baseline peak thrombin generation;Change in participants’ joint pain score using Joint Pain Rating Scale;Change in physical functioning scale of PedsQL | — |
Countries
Korea, Republic of
Contacts
Public ContactSeung Min Hahn
Yonsei University Health System, Severance Hospital
Outcome results
None listed