A Research Study Investigating Mim8 in People With Haemophilia A

NCT04204408 | Completed Phase 2 FDA Drug

• 3 other identifiers: NN7769-4513U1111-1227-42202019-000465-20
• Study Type: interventional
• Target: 275
• Locations: 12 countries
• Sites: 40

Brief Summary

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.

Conditions

Healthy Volunteers; Haemophilia A With or Without Inhibitors

Outcome Measures

Primary Outcomes (3)

• Part 1: Number of treatment emergent adverse events

  Count

  From time of dosing (Day 1) to Week 16

• Part 2: Number of treatment emergent adverse events

  Count

  From time of first dosing (Day 1) to Week 12

• Part 2, extension: Number of treatment emergent adverse events

  Count

  From Week 12 up to Week 176 (16 weeks after last dose)

Secondary Outcomes (24)

• Part 1: Number of injection site reactions

  From time of dosing (Day 1) to Week 16

• Part 1: Relative change in D-dimer

  From baseline (Day 1) to Week 16

• Part 1: Relative change in prothrombin fragment 1 and 2

  From baseline (Day 1) to Week 16

• Part 1: Relative change in fibrinogen

  From baseline (Day 1) to Week 16

• Part 1: Relative change in platelets

  From baseline (Day 1) to Week 16

Study Arms (3)

Single dose (part 1) Mim8

EXPERIMENTAL

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

Drug: NNC0365-3769 (Mim8)

Single dose (part 1) placebo

PLACEBO COMPARATOR

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

Drug: Placebo (Mim8)

Multiple dose (part 2)

EXPERIMENTAL

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.

Drug: NNC0365-3769 (Mim8)

Interventions

NNC0365-3769 (Mim8) DRUG

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

Multiple dose (part 2); Single dose (part 1) Mim8

Placebo (Mim8) DRUG

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

Single dose (part 1) placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (40)

Arizona H&T Phoenix Child Hosp

Phoenix, Arizona, 85016-7710, United States

Location

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, 90027, United States

Location

Children's Healthcare Atlanta

Atlanta, Georgia, 30322, United States

Location

Rush University Med. Cntr

Chicago, Illinois, 60612, United States

Location

University of Iowa_Iowa City

Iowa City, Iowa, 52242, United States

Location

University Of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

Michigan State University

East Lansing, Michigan, 48823, United States

Location

St. Jude Clinic Novant Health

Charlotte, North Carolina, 28204, United States

Location

Cincinnati Child's Hsp Med Ctr

Cincinnati, Ohio, 45229, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Dayton Children Hemostati Ctr

Dayton, Ohio, 45404, United States

Location

Penn State MS Hershey Med Ctr

Hershey, Pennsylvania, 17033-2360, United States

Location

Vanderbilt Hemostasis Thrombosis Clinic

Nashville, Tennessee, 37232, United States

Location

Versiti, CCBD

Milwaukee, Wisconsin, 53226, United States

Location

Universitätsklinik für Innere Medizin V

Innsbruck, 6020, Austria

Location

Universitätsklinik für Innere Medizin V

Innsbruck, A 6020, Austria

Location

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

Sofia, 1527, Bulgaria

Location

Charité - Campus Charité Mitte - Charité Research Organisation GmbH

Berlin, 10117, Germany

Location

Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain

Berlin, 10249, Germany

Location

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

Milan, MI, 20124, Italy

Location

Policlinico Umberto I Sezione Ematologia

Roma, 00161, Italy

Location

Nagoya University Hospital_Blood Transfusion

Aichi, 466-8560, Japan

Location

Nara Medical University Hospital_Pediatrics

Nara, 634-8522, Japan

Location

Tokyo Medical Univ. Hospital_Laboratory Medicine

Tokyo, 160-0023, Japan

Location

Uniwersytecki Szpital Kliniczny W Poznaniu

Poznan, Greater Poland Voivodeship, 60-569, Poland

Location

Instytut Hematologii i Transfuzjologii

Warsaw, Masovian Voivodeship, 02-776, Poland

Location

Szpital Uniwersytecki, Oddzial Kliniczny Hematologii

Krakow, 30-688, Poland

Location

Charlotte Maxeke Johannesburg Academic Hospital

Parktown, Johannesburg, Gauteng, 2193, South Africa

Location

Pietersburg Hospital

Polokwane, Limpopo, 0699, South Africa

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Regional Universitario de Málaga

Málaga, 29010, Spain

Location

Hospital La Fe - Hemostasia y Trombosis

Valencia, 46026, Spain

Location

Universitätsklinik für Hämatologie

Bern, 3010, Switzerland

Location

Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie

Zurich, 8091, Switzerland

Location

Hacettepe Üniversitesi Hastanesi- Endokrinoloji

Ankara, 06230, Turkey (Türkiye)

Location

Trakya Üniversitesi Tıp Fakültesi Hastanesi- Kardiyoloji

Edirne, 22030, Turkey (Türkiye)

Location

Ege Üniversitesi Hastanesi- Hematoloji

Izmir, 35100, Turkey (Türkiye)

Location

Royal Free Haemophilia Comprehensive Care Centre

London, NW3 2QG, United Kingdom

Location

Oxford Haemophilia Comprehensive Care Center

Oxford, OX3 7LJ, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 41246456 DERIVED

Study Officials

• Clinical Reporting Anchor and Disclosure (1452)

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Sponsor staff involved in the clinical trial is masked according to company standard procedures
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Part 1 placebo-controlled double-blind within cohorts (phase 1) Part 2 open-label (phase 2)

Study Record Dates

• First Submitted: December 17, 2019
• First Posted: December 19, 2019
• Study Start: January 10, 2020
• Primary Completion: October 6, 2023
• Study Completion: October 6, 2023
• Last Updated: December 19, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

GB (2); TU (3); BU (1); AU (2); US (15); DE (2); IT (2); JP (3); CH (2); ES (3); PL (3); ZA (2)