A Research Study Investigating Mim8 in People With Haemophilia A

NCT ID: NCT04204408

Last Updated: 2025-12-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

275 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-01-10

Study Completion Date

2023-10-06

Brief Summary

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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.

Detailed Description

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Conditions

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Healthy Volunteers Haemophilia A With or Without Inhibitors

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Part 1 placebo-controlled double-blind within cohorts (phase 1) Part 2 open-label (phase 2)
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Sponsor staff involved in the clinical trial is masked according to company standard procedures

Study Groups

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Single dose (part 1) Mim8

Blinded. Single doses in healthy volunteers. Dose escalation. In each of the 6 cohorts, 6 participants will receive Mim8.

Group Type EXPERIMENTAL

NNC0365-3769 (Mim8)

Intervention Type DRUG

Mim8 administered subcutaneously (s.c., under the skin). The treatment period will consist of 12 once-weekly doses or 3 once-monthly doses

Single dose (part 1) placebo

Blinded. Single doses in healthy volunteers. In each of the 6 cohorts, 2 participants will receive placebo.

Group Type PLACEBO_COMPARATOR

Placebo (Mim8)

Intervention Type DRUG

Mim8 placebo administered subcutaneously (s.c., under the skin)

Multiple dose (part 2)

Open-label. There will be 4 cohorts receiving once-weekly doses (part 2 cohorts 1, 2, 3 and 5) and one cohort receiving once-monthly doses (part 2 cohort 4). Participants will continue into the part 2 extension on the same treatment regimen.

Group Type EXPERIMENTAL

NNC0365-3769 (Mim8)

Intervention Type DRUG

Mim8 administered subcutaneously (s.c., under the skin). The treatment period will consist of 12 once-weekly doses or 3 once-monthly doses

Interventions

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NNC0365-3769 (Mim8)

Mim8 administered subcutaneously (s.c., under the skin). The treatment period will consist of 12 once-weekly doses or 3 once-monthly doses

Intervention Type DRUG

Placebo (Mim8)

Mim8 placebo administered subcutaneously (s.c., under the skin)

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

Yes

Sponsors

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Novo Nordisk A/S

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Reporting Anchor and Disclosure (1452)

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

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Arizona H&T Phoenix Child Hosp

Phoenix, Arizona, United States

Site Status

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, United States

Site Status

Children's Healthcare Atlanta

Atlanta, Georgia, United States

Site Status

Rush University Med. Cntr

Chicago, Illinois, United States

Site Status

University of Iowa_Iowa City

Iowa City, Iowa, United States

Site Status

University Of Michigan

Ann Arbor, Michigan, United States

Site Status

Children's Hospital of Michigan

Detroit, Michigan, United States

Site Status

Michigan State University

East Lansing, Michigan, United States

Site Status

St. Jude Clinic Novant Health

Charlotte, North Carolina, United States

Site Status

Cincinnati Child's Hsp Med Ctr

Cincinnati, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Dayton Children Hemostati Ctr

Dayton, Ohio, United States

Site Status

Penn State MS Hershey Med Ctr

Hershey, Pennsylvania, United States

Site Status

Vanderbilt Hemostasis Thrombosis Clinic

Nashville, Tennessee, United States

Site Status

Versiti, CCBD

Milwaukee, Wisconsin, United States

Site Status

Universitätsklinik für Innere Medizin V

Innsbruck, , Austria

Site Status

Universitätsklinik für Innere Medizin V

Innsbruck, , Austria

Site Status

UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology

Sofia, , Bulgaria

Site Status

Charité - Campus Charité Mitte - Charité Research Organisation GmbH

Berlin, , Germany

Site Status

Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain

Berlin, , Germany

Site Status

Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano

Milan, MI, Italy

Site Status

Policlinico Umberto I Sezione Ematologia

Roma, , Italy

Site Status

Nagoya University Hospital_Blood Transfusion

Aichi, , Japan

Site Status

Nara Medical University Hospital_Pediatrics

Nara, , Japan

Site Status

Tokyo Medical Univ. Hospital_Laboratory Medicine

Tokyo, , Japan

Site Status

Uniwersytecki Szpital Kliniczny W Poznaniu

Poznan, Greater Poland Voivodeship, Poland

Site Status

Instytut Hematologii i Transfuzjologii

Warsaw, Masovian Voivodeship, Poland

Site Status

Szpital Uniwersytecki, Oddzial Kliniczny Hematologii

Krakow, , Poland

Site Status

Charlotte Maxeke Johannesburg Academic Hospital

Parktown, Johannesburg, Gauteng, South Africa

Site Status

Pietersburg Hospital

Polokwane, Limpopo, South Africa

Site Status

Hospital Universitario La Paz

Madrid, , Spain

Site Status

Hospital Regional Universitario de Málaga

Málaga, , Spain

Site Status

Hospital La Fe - Hemostasia y Trombosis

Valencia, , Spain

Site Status

Universitätsklinik für Hämatologie

Bern, , Switzerland

Site Status

Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie

Zurich, , Switzerland

Site Status

Hacettepe Üniversitesi Hastanesi- Endokrinoloji

Ankara, , Turkey (Türkiye)

Site Status

Trakya Üniversitesi Tıp Fakültesi Hastanesi- Kardiyoloji

Edirne, , Turkey (Türkiye)

Site Status

Ege Üniversitesi Hastanesi- Hematoloji

Izmir, , Turkey (Türkiye)

Site Status

Royal Free Haemophilia Comprehensive Care Centre

London, , United Kingdom

Site Status

Oxford Haemophilia Comprehensive Care Center

Oxford, , United Kingdom

Site Status

Countries

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United States Austria Bulgaria Germany Italy Japan Poland South Africa Spain Switzerland Turkey (Türkiye) United Kingdom

References

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Chowdary P, Lentz SR, Gil L, Lopez-Jaime FJ, Windyga J, Ong Clausen WH, Laursen PN, Mahlangu J. FRONTIER1 multiple ascending dose extension: a safety, tolerability, pharmacokinetics, and pharmacodynamics study of Mim8 in people with hemophilia A. Res Pract Thromb Haemost. 2025 Oct 8;9(7):103207. doi: 10.1016/j.rpth.2025.103207. eCollection 2025 Oct.

Reference Type DERIVED
PMID: 41246456 (View on PubMed)

Other Identifiers

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U1111-1227-4220

Identifier Type: OTHER

Identifier Source: secondary_id

2019-000465-20

Identifier Type: REGISTRY

Identifier Source: secondary_id

NN7769-4513

Identifier Type: -

Identifier Source: org_study_id