A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER 5)

NCT05878938 | Completed Phase 3 FDA Drug

• 3 other identifiers: NN7769-4728U1111-1281-93232022-003053-66
• Study Type: interventional
• Target: 61
• Locations: 12 countries
• Sites: 36

Brief Summary

This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

Conditions

Haemophilia A; Haemophilia A With Inhibitors

Outcome Measures

Primary Outcomes (1)

• Number of treatment-emergent adverse events

  Measured as count of events.

  From Visit 2 (week 0) until week 26

Secondary Outcomes (2)

• Device handling experience using the Hemophilia Device Handling and Preference Assessment (HDHPA) questionnaire

  Visit 8 (after 26 weeks of treatment)

• Change in participants' treatment burden using the Hemophilia treatment experience measure (Hemo-TEM) total score

  From Visit 2 (week 0) until end of treatment (up to 26 weeks)

Study Arms (1)

NNC0365-3769 (Mim8) PPX

EXPERIMENTAL

Participants will receive Mim8 prophylaxis (PPX) subcutaneous (s.c.) injection using a prefilled fixed dose DV3407-C1 pen-injector.

Drug: NNC0365-3769 (Mim8) PPX

Interventions

NNC0365-3769 (Mim8) PPX DRUG

Participants will receive Mim8 PPX once-weekly dosing (QW), once every two weeks dosing (Q2W), or once-monthly dosing s.c. injection using a prefilled fixed dose DV3407-C1 pen-injector for 26 weeks.

NNC0365-3769 (Mim8) PPX

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Informed consent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.
• Male or female with diagnosis of congenital haemophilia A of any severity based on medical records.
• Age 12 years or above at the time of signing the informed consent.
• Participants treated with emicizumab once-weekly (QW), once every two weeks (Q2W), or once every four weeks (Q4W) according to the label for at least 8 weeks prior to screening.
• Participants choosing to discontinue emicizumab treatment and switch to Mim8 QW, Q2W, or once-monthly (QM) treatment for 26 weeks from start of treatment (Visit 2).
• Participant and/or caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of an electronic diary and patient-reported outcomes (PRO) questionnaires.

You may not qualify if:

• Participation (i.e., signed informed consent) in any interventional, clinical study, with the exception of emicizumab, with receipt of the last dose within 8 weeks (or 5 half-lives of the investigational medicinal product \[IMP\], whichever is longer) before screening.
• Any disorder, which in the investigator's opinion might jeopardise the participant's compliance with the protocol or safety, including ongoing Adverse Events (AEs) associated with emicizumab.
• Previous participation in this study. Participation is defined as signed informed consent.
• Known congenital or acquired coagulation disorders other than haemophilia A.
• Previous or current thromboembolic disease or events (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.
• Neutralising antibodies towards emicizumab have been detected or, for patients adherent to emicizumab therapy, are suspected based on clinical and laboratory assessments.
• Receipt of FVIII gene therapy at any time.
• Ongoing or planned immune tolerance induction therapy.
• Minor or major surgery planned to take place after screening and during the 26-week treatment period.
• Known or suspected hypersensitivity to study intervention, related products, any constituents of the product or to other monoclonal antibodies.
• Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) greater than (\>) 3 times the upper limit combined with total bilirubin \>1.5 times the upper limit measured at screening.
• Renal impairment defined as estimated glomerular filtration rate (eGFR) lesser than or equal to (≤) 30 milliliter per minute per 1.73 square meter (mL/min/1.73 m\^2) for serum creatinine measured at screening.
• Female who is pregnant, breast-feeding or intends to become pregnant or is of childbearing potential and not using highly effective contraceptive method.
• 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.

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (36)

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, 90027, United States

Location

UC Davis Medical Center

Sacramento, California, 95817, United States

Location

UC Denver Hemoph & Thrombo Ctr

Aurora, Colorado, 80045, United States

Location

St Joseph's Hospital Foundation

Tampa, Florida, 33607, United States

Location

Augusta Univ/Childrens Hosp-GA

Augusta, Georgia, 30912, 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

Central Michigan University

Detroit, Michigan, 48201, United States

Location

Michigan State University

East Lansing, Michigan, 48824, United States

Location

Univ Hosp Cleveland Med Ctr

Cleveland, Ohio, 44106, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Penn State MS Hershey Med Ctr

Hershey, Pennsylvania, 17033-2360, United States

Location

Vanderbilt U Med Ctr_Nashville

Nashville, Tennessee, 37212, United States

Location

Universitätsklinik für Innere Medizin V

Innsbruck, 6020, Austria

Location

AKH - Klin. Abt. f. Haematologie u. Haemostaseologie

Vienna, 1090, Austria

Location

Cliniques universitaires Saint-Luc - Service Hématologie

Brussels, 1200, Belgium

Location

McMaster University

Hamilton, Ontario, L8N 3Z5, Canada

Location

Hospices Civils de Lyon- Hopital Louis Pradel-1

Bron, 69500, France

Location

Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain

Berlin, 10249, Germany

Location

Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie

Bonn, 53127, Germany

Location

AOU Careggi Firenze

Florence, Tuscany, 50134, Italy

Location

Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Azienda Ospedaliera di Rilievo Nazionale Santobono Pausilipon

Naples, 80122, Italy

Location

Azienda Ospedaliera Santobono Pausilipon - U.S.D. Centro Regionale Pediatrico Malattie della Coagulazione

Naples, 80122, Italy

Location

Nara Medical University Hospital_Pediatrics

Nara, 634-8522, Japan

Location

Charlotte Maxeke Johannesburg Academic Hospital

Parktown, Johannesburg, Gauteng, 2193, South Africa

Location

Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea

Location

Kyung Hee University Hospital at Gangdong

Seoul, 05278, South Korea

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Regional Universitario de Málaga

Málaga, 29010, Spain

Location

Belfast City Hospital

Belfast, BT9 78B, United Kingdom

Location

Birmingham Children's Hospital

Birmingham, United Kingdom

Location

Arthur Bloom Haemophilia Centre

Cardiff, CF14 4XW, United Kingdom

Location

Royal Free Haemophilia Comprehensive Care Center

London, NW3 2QG, United Kingdom

Location

Royal Free Haemophilia Comprehensive Care Centre

London, NW3 2QG, United Kingdom

Location

Royal Hallamshire Hospital

Sheffield, S10 2JF, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

protein phosphatase 4

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Transparency (dept. 2834)

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 12, 2023
• First Posted: May 30, 2023
• Study Start: June 26, 2023
• Primary Completion: July 18, 2024
• Study Completion: July 19, 2024
• Last Updated: December 8, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

BE (1); AU (2); CA (1); DE (2); GB (6); IT (4); US (13); ZA (1); ES (2); FR (1); JP (1); KR (2)