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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
70
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
Number of treatment emergent adverse events
Count of events
Time frame: From treatment initiation to follow up visit (week 0 to week 72)
Number of treated bleeds
Count of bleeds
Time frame: From treatment initiation to end of treatment (week 0 to week 52)
Number of treated spontaneous bleeds
Count of bleeds
Time frame: From treatment initiation to end of treatment (week 0 to week 52)
Number of treated traumatic bleeds
Count of bleeds
Time frame: From treatment initiation to end of treatment (week 0 to week 52)
Number of treated joint bleeds
Count of bleeds
Time frame: From treatment initiation to end of treatment (week 0 to week 52)
Number of treated target joint bleeds
Count of bleeds
Time frame: From treatment initiation to end of treatment (week 0 to week 52)
Number of injection site reactions
Count of reactions
Time frame: From treatment initiation to end of treatment (week 0 to week 52)
Consumption of factor product per bleed treatment (number of injections)
Count of injections
Time frame: From run-in initiation to end of treatment (week -26 to week 52)
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Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
Univ of Colorado Sch of Med
Aurora, Colorado, United States
St Joseph's Hospital Foundation
Tampa, Florida, United States
Children's Healthcare Atlanta
Atlanta, Georgia, United States
Children's Hospital at Memorial Health
Savannah, Georgia, United States
University of Iowa_Iowa City
Iowa City, Iowa, United States
Univ Hosp Cleveland Med Ctr
Cleveland, Ohio, United States
Penn State MS Hershey Med Ctr
Hershey, Pennsylvania, United States
St Christopher Hosp for Child
Philadelphia, Pennsylvania, United States
McMaster Children's Hospital
Hamilton, Ontario, Canada
...and 42 more locations
Occurrence of anti-Mim8 antibodies
Count of participants
Time frame: From treatment initiation to end of treatment (week 0 to week 52)
Mim8 plasma concentration
µg/mL
Time frame: From treatment initiation to end of treatment (week 0 to week 52)
Change in physical function domain of PEDS QL (Paediatric Quality of Life inventory) Generic Core Scales
Score on a scale 0-100 (applies for scale scores and total score). A higher score indicates a better health-related quality of life
Time frame: From treatment initiation to end of treatment (week 0 to week 52)
Treatment preference for Mim8 versus previous treatment using Caregiver H PPQ (Caregiver Haemophilia Patient Preference )
Percentage of participants
Time frame: Once during treatment (week 26)
Change in participants' treatment burden using the Hemo TEM (Haemophilia treatment experience measure)
Score on a scale 0-100 (applies for scale scores and total score). A lower score indicates a lower treatment burden.
Time frame: From treatment initiation to end of treatment (week 0 to week 52)