A Research Study Investigating Mim8 in People With Haemophilia A

• Status: Recruiting
• Trial end date: 2024-01-29
• Target enrollment: 0
• Participant gender: Male

Summary

This study consists of 2 parts. Part 1 looks at the safety and tolerability when the study medicine Mim8 is given to healthy subjects for the first time. The obtained results will help to perform a second part of the study with patients who suffer from a bleeding disorder called haemophilia A. Haemophilia A is an inherited condition caused by a lack of a protein called factor VIII. Mim8 works in the body for a longer time than most other products used by patients with haemophilia A. Mim8 is designed for once weekly or once monthly administration. In Part 1, participants will be injected only once with either Mim8 or a "dummy" medicine (placebo) under the tummy skin - which one will be decided by chance. In this study, Mim8 will be used for the first time in humans. Mim8 is a new medicine and cannot be prescribed by doctors in any country. Mim8 belongs to a group of medicines called antibodies. Mim8 is designed to take over the function of the missing factor VIII in haemophilia A patients. Part 1 of the study will last for up to 20 weeks. Participants will attend a screening visit, followed by a 12-day in-house visit where they will have to stay in the study unit. This is followed by 10 ambulatory visits with the study doctor throughout the remaining part of the study. Assessments will include several blood tests and electrocardiograms (ECGs). In part 2 of the study, people with haemophilia A - with or without inhibitors - will be given injections with a thin needle in the skin of their stomach, either weekly or monthly. Part 2 of the study will last for 120 weeks.

• Phase: Phase 2
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Novo Nordisk A/S

Criteria

Inclusion Criteria:

Single ascending dose part 1:

• Male, aged 18-45 years (both inclusive) at the time of signing informed consent

• Considered to be generally healthy based on the medical history, physical examination, and the results of vital signs, electrocardiogram and clinical laboratory tests performed during the screening visit, as judged by the investigator

Multiple ascending dose part 2:

• Male, aged 12-64 years (both inclusive) at the time of signing informed consent (Germany and Japan have local requirements)

• Diagnosis of congenital haemophilia A with FVIII activity below 1% based on medical records

Exploratory biomarker cohort:

• Male, aged equal to or above 12 years at the time of signing informed consent (Germany and Japan have local requirements)

• Diagnosis of congenital haemophilia A with FVIII activity below 1% based on medical recordsv

Exclusion Criteria:

Part 1:

• Factor VIII activity equal to or above 150% at screening

• Increased risk of thrombosis, e.g. known history of personal or first degree relative(s) with unprovoked deep vein thrombosis

• Any clinical signs or established diagnosis of venous or arterial thromboembolic disease

Part 2:

• Known congenital or acquired coagulation disorders other than haemophilia A

• Increased risk of thrombosis as evaluated by the investigator. E.g. known history of personal or first degree relative(s) with unprovoked deep vein thrombosis with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing

• Any clinical signs or established diagnosis of venous or arterial thromboembolic disease with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing

• Advanced atherosclerotic disease (e.g. known history of ischemic heart disease, ischemic stroke) as evaluated by the investigator

• Any autoimmune disease that may increase the risk of thrombosis

• Receipt of emicizumab or drugs with similar modes of action within 5 half-lives before trial product administration

• Ongoing or planned immune tolerance induction therapy

Exploratory biomarker cohort:

• Known congenital or acquired coagulation disorders other than haemophilia A

• Increased risk of thrombosis as evaluated by the investigator. E.g. known history of personal or first degree relative(s) with unprovoked deep vein thrombosis with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing

• Any clinical signs or established diagnosis of venous or arterial thromboembolic disease with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing

• Advanced atherosclerotic disease (e.g. known history of ischemic heart disease, ischemic stroke) as evaluated by the investigator

• Any autoimmune disease that may increase the risk of thrombosis

• Ongoing or planned immune tolerance induction therapy