A Research Study Investigating Mim8 in People With Haemophilia A

Completed · Phase 2 · Has a placebo group

Conditions studied: Healthy Volunteers, Haemophilia A With or Without Inhibitors

In brief

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.

Key facts

Study ID

NCT04204408

Run by

Novo Nordisk A/S

People needed

275

Starts

2020-01-10

Expected to finish

2023-10-06

Last updated by the study team

2025-12-19

Who can join

Age: 12 and older. Sex: male. Healthy volunteers: accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• 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
• 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
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Dayton Children Hemostati Ctr — Dayton, Ohio, United States
• Penn State MS Hershey Med Ctr — Hershey, Pennsylvania, United States
• Vanderbilt Hemostasis Thrombosis Clinic — Nashville, Tennessee, United States
• Versiti, CCBD — Milwaukee, Wisconsin, United States
• Universitätsklinik für Innere Medizin V — Innsbruck, Austria
• Universitätsklinik für Innere Medizin V — Innsbruck, Austria
• UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology — Sofia, Bulgaria
• Charité - Campus Charité Mitte - Charité Research Organisation GmbH — Berlin, Germany
• Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain — Berlin, Germany
• Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano — Milan, MI, Italy
• Policlinico Umberto I Sezione Ematologia — Roma, Italy
• Nagoya University Hospital_Blood Transfusion — Aichi, Japan
• Nara Medical University Hospital_Pediatrics — Nara, Japan
• Tokyo Medical Univ. Hospital_Laboratory Medicine — Tokyo, Japan

Full record on ClinicalTrials.gov

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