Haemophilia A With or Without Inhibitors
Conditions
Brief summary
This study is looking at how Mim8 works compared to other medicines in children with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeds. Mim8 will be injected with a thin needle into the skin. The study will last for about 54-98 weeks, from screening to follow-up visit, In case the participant experiences bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor.
Interventions
DRUGMim8
For treatment part 1, all participants will start on once-weekly treatment and continue on this regimen until week 26. For treatment part 2, starting at week 26, all participants will be offered the choice to remain on once-weekly or switch to once-monthly dosing. Mim8 will be injected with a thin needle into the skin
Sponsors
Novo Nordisk A/S
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 11 Years
Healthy volunteers
No
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 : 1. Participant has been prescribed treatment with FVIII concentrate or bypassing agent in the last 26 weeks prior to screening. 2. Participants with endogenous FVIII activity greater than or equal to 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 below 1%). 5. For previously untreated participants: a. Diagnosis of severe haemophilia A (endogenous FVIII activity below 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.( For China mainland; assessed at the investigator's discretion unless otherwise stated.)
Exclusion criteria
1. Known or suspected hypersensitivity to trial product or related products.(For China mainland; assessed at the investigator's discretion unless otherwise stated.) 2. Previous participation in this study. Participation is defined as signed informed consent. 3. Participation (i.e., signed informed consent) in any interventional clinical study with receipt of last dose within 6 months (or 5 half-lives of the investigational medicinal product, whichever is shorter) before planned randomisation. 4. Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months (or 5 half-lives of the medicinal product, whichever is shorter) before planned randomisation, for participants not included in the run-in. 5. Known congenital or acquired coagulation disorders other than haemophilia A. 6. Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis, as evaluated by the investigator.(For China mainland; assessed at the investigator's discretion unless otherwise stated.) 7. 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.(For China mainland; assessed at the investigator's discretion unless otherwise stated.) 8. Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate understanding and cooperation.(For China mainland; assessed at the investigator's discretion unless otherwise stated.) 9. Lack of adequate parental/caregiver support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary.(For China mainland; assessed at the investigator's discretion unless otherwise stated.) 10. 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. 11. Major surgery planned to take place after screening.(For China mainland; assessed at the investigator's discretion unless otherwise stated.) 12. Immune tolerance induction planned to take place after treatment initiation.(For China mainland; assessed at the investigator's discretion unless otherwise stated.) 13. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) greater than 3 times the upper limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal measured at screening. 14. Serum creatinine above 1.5 x upper limit of normal (ULN), measured at screening. 15. Pregnancy (female participants).(Will be assessed at investigator's discretion, according to suspicion of pregnancy.)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of treatment emergent adverse events | From treatment initiation to follow up visit (week 0 to week 72) | Count of events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of injection site reactions | From treatment initiation to end of treatment (week 0 to week 52) | Count of reactions |
| Number of treated bleeds | From treatment initiation to end of treatment (week 0 to week 52) | Count of bleeds |
| Number of treated spontaneous bleeds | From treatment initiation to end of treatment (week 0 to week 52) | Count of bleeds |
| Number of treated traumatic bleeds | From treatment initiation to end of treatment (week 0 to week 52) | Count of bleeds |
| Number of treated joint bleeds | From treatment initiation to end of treatment (week 0 to week 52) | Count of bleeds |
| Number of treated target joint bleeds | From treatment initiation to end of treatment (week 0 to week 52) | Count of bleeds |
| Occurrence of anti-Mim8 antibodies | From treatment initiation to end of treatment (week 0 to week 52) | Count of participants |
| Mim8 plasma concentration | From treatment initiation to end of treatment (week 0 to week 52) | µg/mL |
| Change in physical function domain of PEDS QL (Paediatric Quality of Life inventory) Generic Core Scales | From treatment initiation to end of treatment (week 0 to week 52) | Score on a scale 0-100 (applies for scale scores and total score). A higher score indicates a better health-related quality of life |
| Treatment preference for Mim8 versus previous treatment using Caregiver H PPQ (Caregiver Haemophilia Patient Preference ) | Once during treatment (week 26) | Percentage of participants |
| Change in participants' treatment burden using the Hemo TEM (Haemophilia treatment experience measure) | From treatment initiation to end of treatment (week 0 to week 52) | Score on a scale 0-100 (applies for scale scores and total score). A lower score indicates a lower treatment burden. |
| Consumption of factor product per bleed treatment (number of injections) | From run-in initiation to end of treatment (week -26 to week 52) | Count of injections |
Countries
Canada, China, Germany, India, Israel, Italy, Japan, Lithuania, Netherlands, Poland, Portugal, Russia, South Africa, South Korea, Spain, Switzerland, Taiwan, United Kingdom, United States
Outcome results
None listed