This study is investigating how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medication that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected with a thin needle in the skin of the stomach, using a pen-injector. The study will last for up to 44 months. It consists of a main phase (part 1 and part 2) and an extension phase. In part 1, participants will be injected only once with either Mim8 or a "dummy" medicine (placebo) - which one will be decided by chance. In part 2 and the extension phase participants will get an Mim8 injection weekly or monthly.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
275
Mim8 administered subcutaneously (s.c., under the skin). The treatment period will consist of 12 once-weekly doses or 3 once-monthly doses
Mim8 placebo administered subcutaneously (s.c., under the skin)
Arizona H&T Phoenix Child Hosp
Phoenix, Arizona, United States
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
Children's Healthcare Atlanta
Atlanta, Georgia, United States
Rush University Med. Cntr
Chicago, Illinois, United States
University of Iowa_Iowa City
Iowa City, Iowa, United States
Part 1: Number of treatment emergent adverse events
Count
Time frame: From time of dosing (Day 1) to Week 16
Part 2: Number of treatment emergent adverse events
Count
Time frame: From time of first dosing (Day 1) to Week 12
Part 2, extension: Number of treatment emergent adverse events
Count
Time frame: From Week 12 up to Week 176 (16 weeks after last dose)
Part 1: Number of injection site reactions
Count
Time frame: From time of dosing (Day 1) to Week 16
Part 1: Relative change in D-dimer
Percent
Time frame: From baseline (Day 1) to Week 16
Part 1: Relative change in prothrombin fragment 1 and 2
Percent
Time frame: From baseline (Day 1) to Week 16
Part 1: Relative change in fibrinogen
Percent
Time frame: From baseline (Day 1) to Week 16
Part 1: Relative change in platelets
Percent
Time frame: From baseline (Day 1) to Week 16
Part 1: Cmax, SD: the maximum concentration of Mim8 after a single dose
μg/mL
Time frame: From baseline (Day 1) to Week 16
Part 1: AUC0-inf, SD: the area under the Mim8 concentration-time curve from time 0 to infinity after a single dose
μg\*day/mL
Time frame: From baseline (Day 1) to Week 16
Part 1: t1/2, SD: the terminal half-life of Mim8 after a single dose
Days
Time frame: From baseline (Day 1) to Week 16
Part 1: tmax, SD: the time to maximum concentration of Mim8 after a single dose
Days
Time frame: From baseline (Day 1) to Week 16
Part 1: Change in activated partial thromboplastin time
Seconds
Time frame: From baseline (Day 1) to Week 16
Part 2 (weekly and monthly dosing): Number of injection site reactions
Count
Time frame: From time of first dosing (Day 1) to Week 12
Part 2 (weekly and monthly dosing): Occurrence of anti-Mim8 antibodies
Count
Time frame: From baseline (Day 1) to Week 12
Part 2 (weekly and monthly dosing): Relative change in D-dimer
Percent
Time frame: From baseline (Day 1) to Week 12
Part 2 (weekly and monthly dosing): Relative change in prothrombin fragment 1 and 2
Percent
Time frame: From baseline (Day 1) to Week 12
Part 2 (weekly and monthly dosing): Relative change in fibrinogen
Percent
Time frame: From baseline (Day 1) to Week 12
Part 2 (weekly and monthly dosing): Relative change in platelets
Percent
Time frame: From baseline (Day 1) to Week 12
Part 2 PK session 2 (weekly dosing): Cmax, MD: the maximum concentration of Mim8 after multiple doses
μg/mL
Time frame: From Day 57 to Day 64
Part 2 PK session 2 (weekly dosing): AUCτ, MD: the area under the Mim8 concentration-time curve in the dosing interval after multiple doses
μg\*day/mL
Time frame: From Day 57 to Day 64
Part 2 PK session 2 (monthly dosing): Cmax, MD: the maximum concentration of Mim8 after multiple doses
μg/mL
Time frame: From Day 57 to Day 85
Part 2 PK session 2 (monthly dosing): AUCτ, MD: the area under the Mim8 concentration-time curve in the dosing interval after multiple doses
μg\*day/mL
Time frame: From Day 57 to Day 85
Part 2 (weekly dosing): Mean of maximum thrombin generation (peak height)
nM
Time frame: From Day 57 to Day 64
Part 2 (monthly dosing): Mean of maximum thrombin generation (peak height)
nM
Time frame: From Day 57 to Day 85
Part 2, extension: Number of injection site reactions
Count
Time frame: From Week 12 up to Week 176 (16 weeks after last dose)
Part 2, extension: Occurrence of anti-Mim8 antibodies
Count
Time frame: From Week 12 up to Week 176 (16 weeks after last dose)
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University Of Michigan
Ann Arbor, Michigan, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Michigan State University
East Lansing, Michigan, United States
St. Jude Clinic Novant Health
Charlotte, North Carolina, United States
Cincinnati Child's Hsp Med Ctr
Cincinnati, Ohio, United States
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