4513 (Frontier 1) Investigating Mim8 in people with haemophilia A
Research type
Research Study
Full title
Safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple subcutaneous doses of NNC0365-3769 (Mim8) in healthy subjects and in subjects with haemophilia A with or without factor VIII inhibitors.
IRAS ID
272557
Contact name
Nicola Curry
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2019-000465-20
Clinicaltrials.gov Identifier
U1111-1227-4220, UTN
Duration of Study in the UK
1 years, 1 months, 27 days
Research summary
Haemophilia is a bleeding disorder that some people are born with. The most common type of haemophilia is called haemophilia A. This means the person has too little clotting factor VIII in the blood.\nMim8 is being developed to address a need for effective and convenient bleeding prevention in haemophilia A patients with or without FVIII inhibitors. Mim8 restores the function of missing activated FVIII clotting factor.\nThis is the first clinical trial with Mim8, it consists of a phase 1 single\nascending dose (SAD) and a phase 2 multiple ascending dose (MAD) part.\nMim8 will be given subcutaneously, usually products to treat haemophilia are injections into the vein.\nThe MAD part includes four cohorts, three receiving once-weekly doses\nand one cohort receiving once-monthly doses. There are 22 planned visits to the trial site for cohorts 1 to 3,and 19 planned visits for cohort 4. The total trial duration for a subject will be approximately 28 weeks for MAD cohorts with once-weekly dosing and 25 weeks for the MAD cohort with once-monthly dosing, plus a screening period of up to 28 days. The treatment period includes 12 once-weekly doses or 3 once-monthly doses, with a follow-up period of 16 weeks after last dose.\nAdditionally, haemophilia A subjects treated with emicizumab (approved treatment for Haemophilia A with & without inhibitors), will be enrolled in a separate cohort to obtain laboratory data which is expected to be useful for benchmarking against an approved product with the same mode of action. This cohort will consist of 5 visits to the trial site. The total trial duration for an individual subject will be 12 weeks, plus screening period of up to 28 days. There will be no follow-up.\nUK will participate in phase 2 MAD part and emicizumab cohorts at specialist Haemophilia centres.\n
REC name
South Central - Oxford B Research Ethics Committee
REC reference
19/SC/0621
Date of REC Opinion
21 Jan 2020
REC opinion
Further Information Favourable Opinion