4514 (Frontier 2): Mim8 in adults and adolescents with haemophilia A
Research type
Research Study
Full title
A multinational, open-label, randomised, controlled study to investigate efficacy and safety of NNC0365-3769 (Mim8) in adults and adolescents with haemophilia A with or without inhibitors.
IRAS ID
290498
Contact name
Nicola Curry
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2020-001048-24
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
UTN, U1111-1249-4378
Duration of Study in the UK
2 years, 3 months, 4 days
Research summary
The purpose of this phase 3 trial is to confirm the effect and investigate safety of Mim8 prophylaxis (preventative) treatment in a larger population of patients with haemophilia A, with or without factor VIII inhibitors. This confirmatory trial will serve as the basis for market authorisation application.
This is a randomised open-labelled four arm trial in haemophilia A patients including a lead-in period of up to 52 weeks duration. This lead-in period serves the purpose of enabling collection of high-quality bleeding and treatment data in patients on prophylaxis coagulation factor treatment. Patients on prophylaxis treatment must continue on the same drug and dosing frequency for at least 26 weeks of the lead-in period before they can be randomised into the main part of the trial. Patients will be randomised to Mim8 prophylaxis treatment or no prophylaxis treatment, based on their treatment regimen over the prior 26 weeks. Patients not taking prophylaxis treatment will be randomised 1:2 to continue on no prophylaxis treatment (i.e. taking on-demand treatment with their usual product), or on Mim8 once-weekly prophylaxis treatment (arms 1 and 2). Patients on coagulation factor prophylaxis treatment during the lead-in will be randomised 1:1 to one of two Mim8 prophylaxis treatment regimens: once-weekly or once-monthly (arms 3 and 4). Only patients who are on prophylaxis treatment will enter the trial in the UK, so there will only be patients in arms 3 and 4.
Each patient will complete the main part of the trial when they have completed 26 weeks of participation (excluding screening and the lead-in period). After the main part of the trial, all patients will be offered to continue into an extension where they will receive treatment with Mim8 for 26 weeks.REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
20/YH/0320
Date of REC Opinion
21 Jan 2021
REC opinion
Further Information Favourable Opinion