This study is investigating how Mim8 works compared to other medicines in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). When and how often participants will receive Mim8 is dependent on their previous treatment - but is otherwise decided by chance. Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month. The study will last 54-124 weeks (12-29 months) depending on how long participants will be followed in run-in before they start treatment and if they continue in the follow period or transfer to an open label extension study. Participants will have 12-17 clinic visits.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
281
Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.
Arizona H&T Phoenix Child Hosp
Phoenix, Arizona, United States
Hemophilia Treatment Center
Los Angeles, California, United States
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
Univ of Colorado Sch of Med
Aurora, Colorado, United States
Univ of Miami/SCCC
Miami, Florida, United States
Number of treated bleeds
Count
Time frame: No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (week 26)
Number of treated bleeds
Count
Time frame: Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (week 26)
Number of injection site reactions
Count
Time frame: All participants receiving Mim8 (Arms 2a, 2b, 3 and 4): From randomisation (week 0) to end of main (week 26)
Occurrence of anti-Mim8 antibodies
Count
Time frame: All participants receiving Mim8 (Arms 2a, 2b, 3 and 4): From randomisation (week 0) to end of extension (week 52)
Number of treated spontaneous bleeds
Count
Time frame: No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)
Number of treated joint bleeds
Count
Time frame: No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)
Number of treated traumatic bleeds
Count
Time frame: No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)
Number of target joint bleeds
Count
Time frame: No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)
Consumption of factor product per bleed treatment (number of injections)
Count
Time frame: No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)
Change in physical function domain of PEDS-QL (pediatric quality of life inventory)
Score points Minimum score per question (best) = 0 Maximum score per question (worst) = 4 Total score for 13 questions: 0 (best) to 92 (worst)
Time frame: All participants (Arms 1, 2a, 2b, 3 and 4): From randomisation (week 0) to the end of the main part (week 26)
Change in patient's treatment burden using the Hemo-TEM (haemophilia treatment experience measure)
Score points Ranges from 0 (best) - 4 (worst) representing answers ranging: 'Not at all difficult' - 'Extremely difficult' 'Never' - 'always' 'Not at all bothered' - 'Extremely bothered' 'Not at all interfering' - 'Extremely interfering' 'Not at all burdened' - 'Extremely burdened'
Time frame: All participants (Arms 1, 2a, 2b, 3 and 4): From randomisation (week 0) to the end of the main part (week 26)
Change in patient's joint pain score using Joint Pain Rating Scale
Score points ranges from 0 = 'not at all' (best) to 4 = 'extremely' (worst)
Time frame: All participants (Arms 1, 2a, 2b, 3 and 4): From randomisation (week 0) to the end of the main part (week 26)
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Arnold Palmer Hospital for Children
Orlando, Florida, United States
St Joseph's Hospital Foundation
Tampa, Florida, United States
Children HC Atlanta-Adv Pediat
Atlanta, Georgia, United States
Augusta University
Augusta, Georgia, United States
Memorial Health Univ Med Ctr
Savannah, Georgia, United States
...and 153 more locations