None listed
Conditions
Interventions
Drug : The study includes a run-in period of at least 26 weeks duration for participants previously treated on prophylaxis. There is no run-in period for PUPs and those on ITI. The run-in period is op
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 and female participants with the diagnosis of congenital haemophilia A of any severity based on medical records. 3. Aged 1-11 years (both inclusive) at the time of signing informed consent. 4. For previously treated participants : a. Participant has been prescribed treatment with FVIII concentrate or bypassing agent in the last 26 weeks prior to screening. b. Participants with endogenous FVIII activity =1%, based on medical records, must have at least 1 treated bleed during the previous 26 weeks before screening for which factor VIII concentrate or bypassing agent has been prescribed (No requirements for participants with FVIII activity <1%). 5. For previously untreated participants: a. Diagnosis of severe haemophilia A (endogenous FVIII activity < 1%) based on medical records. 6. Child and parent/caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires.a
Exclusion criteria
Exclusion criteria: 1. Known or suspected hypersensitivity to study product or related products. 2. Previous participation in this study. Participation is defined as signed informed consent. 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 participantsnot included in the run-in. 4. Known congenital or acquired coagulation disorders other than haemophilia A. 5. Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis, as evaluated by the investigator. 6. Any disorder, except for conditions associated with haemophilia A, that in the investigator’s opinion might jeopardise the participant’s safety or compliance with the protocol. 7. Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate understanding and cooperation.a 8. Lack of adequate parental/caregiver support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary. 9. Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease. 10. Major surgery planned to take place after screening. 11. Immune tolerance induction planned to take place after treatment initiation. 12. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) >3 times the upper limit of normal combined with total bilirubin >1.5 times the upper limit of normal measured at screening. 13. Serum creatinine above 1.5 x upper limit of normal (ULN), measured at screening. 14. Pregnancy (female participants).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of treatment emergent adverse events | — |
Secondary
| Measure | Time frame |
|---|---|
| Number of treated bleeds;Number of treated spontaneous bleeds;Number of treated traumatic bleeds;Number of treated joint bleeds;Number of treated target joint bleeds;Number of injection site reactions;Consumption of factor product per bleed treatment;Occurrence of anti-Mim8 antibodies;Mim8 plasma concentration;Change in physical function domain of PEDS-QL Generic Core Scales;Treatment preference for Mim8 versus previous treatment using Caregiver H-PPQ;Change in patients’ treatment burden using the Hemo-TEM | — |
Countries
Korea, Republic of
Contacts
Public ContactSeung Min Hahn
Yonsei University Health System, Severance Hospital
Outcome results
None listed