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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
451
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.
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
UC Denver Hemoph & Thrombo Ctr
Aurora, Colorado, United States
Univ of Miami/SCCC
Miami, Florida, United States
St Joseph's Hospital Foundation
Tampa, Florida, United States
Children's Healthcare Atlanta
Atlanta, Georgia, United States
Arm 1 and 2: Number of treatment emergent adverse events
Measured as count of events.
Time frame: From week 0 until end of study (up to 283 weeks)
Arm 3: Number of treatment emergent adverse events
Measured as count of events.
Time frame: From treatment initiation (week 0) until end of study (up to 124 weeks)
Arm 1 and 2: Number of injection site reactions
Measured as count of reactions.
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Occurrence of anti Mim8 antibodies
Measured as count of participants.
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated bleeding episodes
Measured as count of bleeds.
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated spontaneous bleeding episodes
Measured as count of bleeds.
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated traumatic bleeding episodes
Measured as count of bleeds.
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated joint bleeding episodes
Measured as count of bleeds.
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 2: Number of treated target joint bleeding episodes
Measured as count of bleeds.
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Mim8 plasma concentration
Measured as micrograms per milliliter (µg/mL).
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 2: Device handling using haemophilia device assessment tool (HDAT) (applicable for participants in arm 2 only)
Measured as percentage of participants.
Time frame: From week 26 until end of treatment (up to 262 weeks)
Arm 3: Number of injection site reactions
Measured as count of reactions.
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Occurrence of anti Mim8 antibodies
Measured as count of participants.
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Number of treated bleeding episodes
Measured as count of bleeds.
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Number of treated spontaneous bleeding episodes
Measured as count of bleeds.
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Number of treated traumatic bleeding episodes
Measured as count of bleeds.
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Mim8 plasma concentration
Measured as µg/mL.
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Device handling using haemophilia device assessment tool (HDAT)
Measured as percentage of participants.
Time frame: At week 26 and week 52
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Rush University Med. Cntr
Chicago, Illinois, United States
Rush University Medical Center
Chicago, Illinois, United States
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, United States
University of Iowa_Iowa City
Iowa City, Iowa, United States
Central Michigan University
Detroit, Michigan, United States
...and 128 more locations