This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant 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
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
61
Participants will receive Mim8 PPX once-weekly dosing (QW), once every two weeks dosing (Q2W), or once-monthly dosing s.c. injection using a prefilled fixed dose DV3407-C1 pen-injector for 26 weeks.
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
UC Davis Medical Center
Sacramento, California, United States
UC Denver Hemoph & Thrombo Ctr
Aurora, Colorado, United States
St Joseph's Hospital Foundation
Tampa, Florida, United States
Augusta Univ/Childrens Hosp-GA
Augusta, Georgia, United States
Number of treatment-emergent adverse events
Measured as count of events.
Time frame: From Visit 2 (week 0) until week 26
Device handling experience using the Hemophilia Device Handling and Preference Assessment (HDHPA) questionnaire
Measured as percentage of participants. HDHPA measures device handling experience and device preference. The measure consists of 26 items that are reported individually. it is measures in units: Percentage of participants = the distribution of participant answers within each response category, for each of the 26 individual items.
Time frame: Visit 8 (after 26 weeks of treatment)
Change in participants' treatment burden using the Hemophilia treatment experience measure (Hemo-TEM) total score
Measured as score points. Hemo-TEM measures treatment burden. The measure consists of 26 items yielding 5 domain scores and 1 total score. Domain scores (score range): Injection difficulties (0-100), physical impact (0-100), treatment bother (0-100), interference with daily life (0-100), and emotional impact (0-100). Total score ranges 0-100. Higher scores indicate greater treatment burden.
Time frame: From Visit 2 (week 0) until end of treatment (up to 26 weeks)
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Rush University Med. Cntr
Chicago, Illinois, United States
University of Iowa_Iowa City
Iowa City, Iowa, United States
Central Michigan University
Detroit, Michigan, United States
Michigan State University
East Lansing, Michigan, United States
Univ Hosp Cleveland Med Ctr
Cleveland, Ohio, United States
...and 26 more locations