Haemophilia A Haemophilia A with inhibitors MedDRA version: 20.0 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10053751 Term: Hemophilia A with anti factor VIII System Organ Class: 100000004850
Conditions
Interventions
Product Name: NNC0365-3769 B 2.0 mg/mL
Pharmaceutical Form: Solution for injection
INN or Proposed INN: N/A
Other descriptive name: Mim8
Concentration unit: mg/ml milligram(s)/millilitre
Concentration
Sponsors
Novo Nordisk A/S
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 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Known or suspected hypersensitivity to trial product or related products. 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. 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. 8. Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate understanding and cooperation. 9. Lack of adequate parental/caregiver support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary. 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 definition of major surgery, see Table ?6-7. 12. Immune tolerance induction planned to take place after treatment initiation. 13. 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. 14. Serum creatinine above 1.5 x upper limit of normal (ULN), measured at screening. 15. Pregnancy (female participants).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To investigate the safety of Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.;Secondary Objective: Investigate the efficacy of Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors. Evaluate the consumption of coagulation factor replacement product per bleed treatment (number of injections) with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors. Evaluate the development of anti-Mim8 antibodies with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors. Investigate exposure of Mim8 after once-weekly and once-monthly subcutaneous dosing in children with haemophilia A with or without FVIII inhibitors. Evaluate treatment burden with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors, as reported by their caregivers. Investigate treatment preference among caregivers of previously treated children with haemophilia A with or without FVIII inhibitors. Evaluate aspects of physical functioning with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.;Primary end point(s): 1. Number of treatment emergent adverse events;Timepoint(s) of evaluation of this end point: 1. From treatment initiation to follow up visit (week 0 to week 72) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Number of treated bleeds 2. Number of treated spontaneous bleeds 3. Number of treated traumatic bleeds 4. Number of treated joint bleeds 5. Number of treated target joint bleeds 6. Number of injection site reactions 7. Consumption of factor product per bleed treatment 8. Occurrence of anti-Mim8 antibodies 9. Mim8 plasma concentration 10. Change in physical function domain of Paediatric Quality of Life inventory (PEDS-QL) Generic Core Scales 11. Treatment preference for Mim8 versus previous treatment using Caregiver Haemophilia Patient Preference Questionnaire (Caregiver H-PPQ) 12. Change in participants’ treatment burden using the Haemophilia treatment experience measure (Hemo TEM);Timepoint(s) of evaluation of this end point: 1-6 . From treatment initiation to end of treatment (week 0 to week 52) 7. From run-in initiation to end of treatment (week -26 to week 52) 8-10. From treatment initiation to end of treatment (week 0 to week 52) 11. Once during treatment (week 26) 12. From treatment initiation to end of treatment (week 0 to week 52) | — |
Countries
Canada, China, European Union, India, Israel, Italy, Japan, Korea, Republic of, Lithuania, Netherlands, Poland, Portugal, Russian Federation, South Africa, Spain, Switzerland, Taiwan, United Kingdom, United States
Contacts
Public ContactClinical Transparency (2834)
Novo Nordisk A/S
Outcome results
None listed