A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors
- Conditions
- Haemophilia A With or Without Inhibitors
- Registration Number
- NCT05306418
- Lead Sponsor
- Novo Nordisk A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 70
Inclusion Criteria:<br><br> 1. Informed consent obtained before any study-related activities. Study-related<br> activities are any procedures that are carried out as part of the study, including<br> activities to determine suitability for the study.<br><br> 2. Male and female participants with the diagnosis of congenital haemophilia A of any<br> severity based on medical records.<br><br> 3. Aged 1-11 years (both inclusive) at the time of signing informed consent.<br><br> 4. For previously treated participants :<br><br> 1. Participant has been prescribed treatment with FVIII concentrate or bypassing<br> agent in the last 26 weeks prior to screening.<br><br> 2. Participants with endogenous FVIII activity greater than or equal to 1%, based<br> on medical records, must have at least 1 treated bleed during the previous 26<br> weeks before screening for which factor VIII concentrate or bypassing agent has<br> been prescribed (no requirements for participants with FVIII activity below<br> 1%).<br><br> 5. For previously untreated participants:<br><br> a. Diagnosis of severe haemophilia A (endogenous FVIII activity below 1%) based on<br> medical records.<br><br> 6. Child and parent/caregiver willingness and ability to comply with scheduled visits<br> and study procedures, including the completion of diary and patient-reported<br> outcomes questionnaires.( For China mainland; assessed at the investigator's<br> discretion unless otherwise stated.)<br><br>Exclusion criteria:<br><br> 1. Known or suspected hypersensitivity to trial product or related products.(For China<br> mainland; assessed at the investigator's discretion unless otherwise stated.)<br><br> 2. Previous participation in this study. Participation is defined as signed informed<br> consent.<br><br> 3. Participation (i.e., signed informed consent) in any interventional clinical study<br> with receipt of last dose within 6 months (or 5 half-lives of the investigational<br> medicinal product, whichever is shorter) before planned randomisation.<br><br> 4. Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months<br> (or 5 half-lives of the medicinal product, whichever is shorter) before planned<br> randomisation, for participants not included in the run-in.<br><br> 5. Known congenital or acquired coagulation disorders other than haemophilia A.<br><br> 6. Other conditions (e.g. autoimmune disease) or laboratory abnormality that may<br> increase risk of bleeding or thrombosis, as evaluated by the investigator.(For China<br> mainland; assessed at the investigator's discretion unless otherwise stated.)<br><br> 7. Any disorder, except for conditions associated with haemophilia A, that in the<br> investigator's opinion might jeopardise the participant's safety or compliance with<br> the protocol.(For China mainland; assessed at the investigator's discretion unless<br> otherwise stated.)<br><br> 8. Mental incapacity, unwillingness to cooperate or a language barrier precluding<br> adequate understanding and cooperation.(For China mainland; assessed at the<br> investigator's discretion unless otherwise stated.)<br><br> 9. Lack of adequate parental/caregiver support to enter accurately and timely<br> information regarding treatment and bleeding episodes into an (electronic)<br> diary.(For China mainland; assessed at the investigator's discretion unless<br> otherwise stated.)<br><br> 10. Previous or current treatment for thromboembolic disease (with the exception of<br> previous catheter-associated thrombosis for which anti-thrombotic treatment is not<br> currently ongoing) or signs of thromboembolic disease.<br><br> 11. Major surgery planned to take place after screening.(For China mainland; assessed at<br> the investigator's discretion unless otherwise stated.)<br><br> 12. Immune tolerance induction planned to take place after treatment initiation.(For<br> China mainland; assessed at the investigator's discretion unless otherwise stated.)<br><br> 13. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine<br> aminotransferase (ALT) greater than 3 times the upper limit of normal combined with<br> total bilirubin greater than 1.5 times the upper limit of normal measured at<br> screening.<br><br> 14. Serum creatinine above 1.5 x upper limit of normal (ULN), measured at screening.<br><br> 15. Pregnancy (female participants).(Will be assessed at investigator's discretion,<br> according to suspicion of pregnancy.)
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of treatment emergent adverse events
- Secondary Outcome Measures
Name Time Method 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 (number of injections);Occurrence of anti-Mim8 antibodies;Mim8 plasma concentration;Change in physical function domain of PEDS QL (Paediatric Quality of Life inventory) Generic Core Scales;Treatment preference for Mim8 versus previous treatment using Caregiver H PPQ (Caregiver Haemophilia Patient Preference );Change in participants' treatment burden using the Hemo TEM (Haemophilia treatment experience measure)