A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors

• Status: Not yet recruiting
• Trial end date: 2024-03-31
• Target enrollment: 0
• Participant gender: All

Summary

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. The study will last for 72-124 weeks (17-29 months) depending on how long participants will be followed before it is decided when they start receiving Mim8 - the period before this is decided is called the 'run-in'. Participants will have 13-17 clinic visits. Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novo Nordisk A/S

Criteria

Inclusion Criteria:

• Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial

• Male or female with diagnosis of congenital haemophilia A of any severity based on medical records

• Patient has been prescribed, or in need of, treatment with factor VIII or bypassing agent in the last 26 weeks prior to screening

• Age above or equal to 12 years at the time of signing informed consent, Japan (The legal age in Japan is 20 years or above) and Taiwan (The legal age in Taiwan is 20 years or above)

• Body weight greater than or equal to 30 kg

• Applicable to patients on emicizumab prophylaxis: patient is willing to discontinue emicizumab at the time of screening

• Applicable to patients treated with no prophylaxis prior to enrolment: greater than or equal to 5 bleeds in the last 26 weeks prior to screening visit

• Applicable to patients with FVIII activity greater than 1 percentage who are on prophylactic treatment: greater than or equal to 1 bleed in the last 26 weeks prior to screening visit

• Willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires

Exclusion Criteria:

• Previous participation in this trial. Participation is defined as signed informed consent

• Participation in any clinical trial of an approved or non-approved investigational medicinal product, within 30 days (or 5 half-lives of the investigational medicinal product, whichever is greater) before screening

• Female who is pregnant, breast-feeding or intends to become pregnant or is of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures as defined in the protocol or as required by local regulation or practice). Breast feeding is allowed only during the run-in period

• Any disorder, except for conditions associated with haemophilia A, which in the investigator's opinion might jeopardise subject's safety or compliance with the protocol

• Known or suspected hypersensitivity to trial product(s), any constituents of the product or to related products

• Receipt of gene therapy at any given time point

• Ongoing or planned ITI (immune tolerance induction) therapy

• Major surgery planned at the time of screening

• Known congenital or acquired coagulation disorders other than haemophilia A

• Hepatic dysfunction defined as AST (aspartate aminotransferase) and/or ALT (alanine aminotransferase) greater than 3 times the upper limit combined with total bilirubin greater than 1.5 times the upper limit measured at screening

• Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) below or equal to 30 ml/min/1.73 m^2 for serum creatinine measured at screening

• Previous or current thromboembolic disease or events(Includes arterial and venous thrombosis including myocardial infarction, thrombotic microangiography (TMA), pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion.) (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or risk of thromboembolic disease, as evaluated by investigator

• Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation

• Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis as evaluated by the investigator