Haemophilia A, Haemophilia A With Inhibitors
Conditions
Brief summary
This study is looking at how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used to avoid bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). The study will last for up to 5.5 years. The duration of the study depends on when the participant enrolled in this study. The study will end if Mim8 is approved and marketed in participant's country during the study, or the study will end in June 2028, whichever comes first. Participants will get up to 262 injections; the number of injections depends on how often participants will get injections and how long time participants take part in the study. While taking part in this study, there are some restrictions about what medicine participants can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.
Interventions
DRUGMim8
Participants in arm 1 will administer Mim8 using an enhanced cartridge and switch to the DV3407 pen-injector once it is approved. Participants in arm 2 and 3 will use the DV3407 pen injector.
Sponsors
Novo Nordisk A/S
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
Arm 1 \& 2: 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 based on medical records. 3. Ongoing participation in study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728 at the time of transfer. Participant should qualify either of the following criteria: 1. Participant from study NN7769-4513, who has participated in the extension part of the study for at least 12 weeks prior to enrolment in study NN7769-4532, or, 2. Participant has completed the end of treatment visit for study NN7769-4514, NN7769-4516 or NN7769-4728. 4. Participant and/or participant's parent(s)/participant's Legally acceptable representative (LAR) willingness and ability to comply with scheduled visits and study procedures, including the completion of diary.
Exclusion criteria
1. Any disorder, except for conditions associated with haemophilia A, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol. 2. Participant who has discontinued or been withdrawn from studies NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728. 3. Previous participation in this study. Participation is defined as signed informed consent. 4. Female who is pregnant, breast-feeding or intends to become pregnant. 5. Female of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures or as required by local regulation or practice). 6. Participation (i.e., signed informed consent) in any interventional, clinical study (except from study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728) of an approved or non-approved investigational medicinal product. 7. Any planned major surgery, during part 1 of the study. 8. Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation. Arm 3: 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 severe haemophilia A (endogenous FVIII activity less than (\<) 1 percentage \[%\]) with or without FVIII inhibitors based on medical records. 3. Aged \<1 year at the time of signing informed consent. 4. Body weight greater than or equal to (≥) 3.2 kilograms at the time of signing informed consent. 5. previously untreated patients (PUPs) or minimally treated patients (MTPs) (i.e., up to 5 days of exposure to haemophilia-related treatment such as plasma-derived FVIII, recombinant FVIII, fresh frozen plasma, cryoprecipitate, or whole blood products). 6. Full-term pregnancy (gestational age ≥37 weeks). 7. Participant's parent(s)/LAR(s) willingness and ability to comply with scheduled visits and Arm 3 (infant) procedures, including the completion of diary and patient reported outcome (PRO) questionnaire. 8. Participants \<3 months of age must show no signs of active intracranial haemorrhage at screening. This is confirmed by cranial ultrasound performed according to local practice and regardless of delivery method. 9. Receipt of vitamin K prophylaxis (as per local standard practice). 10. Availability of historical results in medical records for: 1. activated partial thromboplastin time (aPTT) 2. FVIII levels. 11. Availability of historical results in medical records or pre-dose sample taken for: 1. fibrinogen 2. haematology parameters 3. biochemistry parameters (aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT), bilirubin and creatinine).
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Arm 1 and 2: Number of treatment emergent adverse events | From week 0 until end of study (up to 283 weeks) | Measured as count of events. |
| Arm 3: Number of treatment emergent adverse events | From treatment initiation (week 0) until end of study (up to 124 weeks) | Measured as count of events. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Arm 1 and 2: Number of injection site reactions | From week 0 until end of treatment (up to 262 weeks) | Measured as count of reactions. |
| Arm 1 and 2: Occurrence of anti Mim8 antibodies | From week 0 until end of treatment (up to 262 weeks) | Measured as count of participants. |
| Arm 1 and 2: Number of treated bleeding episodes | From week 0 until end of treatment (up to 262 weeks) | Measured as count of bleeds. |
| Arm 1 and 2: Number of treated spontaneous bleeding episodes | From week 0 until end of treatment (up to 262 weeks) | Measured as count of bleeds. |
| Arm 1 and 2: Number of treated traumatic bleeding episodes | From week 0 until end of treatment (up to 262 weeks) | Measured as count of bleeds. |
| Arm 1 and 2: Number of treated joint bleeding episodes | From week 0 until end of treatment (up to 262 weeks) | Measured as count of bleeds. |
| Arm 2: Number of treated target joint bleeding episodes | From week 0 until end of treatment (up to 262 weeks) | Measured as count of bleeds. |
| Arm 1 and 2: Mim8 plasma concentration | From week 0 until end of treatment (up to 262 weeks) | Measured as micrograms per milliliter (µg/mL). |
| Arm 2: Device handling using haemophilia device assessment tool (HDAT) (applicable for participants in arm 2 only) | From week 26 until end of treatment (up to 262 weeks) | Measured as percentage of participants. |
| Arm 3: Number of injection site reactions | From treatment initiation (week 0) until end of treatment (up to 103 weeks) | Measured as count of reactions. |
| Arm 3: Occurrence of anti Mim8 antibodies | From treatment initiation (week 0) until end of treatment (up to 103 weeks) | Measured as count of participants. |
| Arm 3: Number of treated bleeding episodes | From treatment initiation (week 0) until end of treatment (up to 103 weeks) | Measured as count of bleeds. |
| Arm 3: Number of treated spontaneous bleeding episodes | From treatment initiation (week 0) until end of treatment (up to 103 weeks) | Measured as count of bleeds. |
| Arm 3: Number of treated traumatic bleeding episodes | From treatment initiation (week 0) until end of treatment (up to 103 weeks) | Measured as count of bleeds. |
| Arm 3: Mim8 plasma concentration | From treatment initiation (week 0) until end of treatment (up to 103 weeks) | Measured as µg/mL. |
| Arm 3: Device handling using haemophilia device assessment tool (HDAT) | At week 26 and week 52 | Measured as percentage of participants. |
Countries
Austria, Belgium, Bulgaria, Canada, China, Denmark, France, Germany, India, Ireland, Israel, Italy, Japan, Latvia, Lithuania, Malaysia, Mexico, Netherlands, Poland, Portugal, Romania, Saudi Arabia, Serbia, Slovakia, South Africa, South Korea, Spain, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom, United States
Contacts
STUDY_DIRECTORClinical Transparency dept. 2834
Novo Nordisk A/S
Outcome results
None listed