A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A (FRONTIER 4)
FRONTIER4
Open-label, Long-term Safety and Efficacy Study of Mim8 in Participants With Haemophilia A With or Without Inhibitors
3 other identifiers
interventional
451
32 countries
140
Brief Summary
This study is looking at how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used to avoid bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). The study will last for up to 5.5 years. The duration of the study depends on when the participant enrolled in this study. The study will end if Mim8 is approved and marketed in participant's country during the study, or the study will end in June 2028, whichever comes first. Participants will get up to 262 injections; the number of injections depends on how often participants will get injections and how long time participants take part in the study. While taking part in this study, there are some restrictions about what medicine participants 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Feb 2023
Longer than P75 for phase_3
140 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 4, 2023
CompletedFirst Posted
Study publicly available on registry
January 17, 2023
CompletedStudy Start
First participant enrolled
February 13, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 30, 2028
March 24, 2026
March 1, 2026
5.4 years
January 4, 2023
March 23, 2026
Conditions
Outcome Measures
Primary Outcomes (2)
Arm 1 and 2: Number of treatment emergent adverse events
Measured as count of events.
From week 0 until end of study (up to 283 weeks)
Arm 3: Number of treatment emergent adverse events
Measured as count of events.
From treatment initiation (week 0) until end of study (up to 124 weeks)
Secondary Outcomes (16)
Arm 1 and 2: Number of injection site reactions
From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Occurrence of anti Mim8 antibodies
From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated bleeding episodes
From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated spontaneous bleeding episodes
From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated traumatic bleeding episodes
From week 0 until end of treatment (up to 262 weeks)
- +11 more secondary outcomes
Study Arms (3)
Arm 1
EXPERIMENTALParticipants entering from the multiple ascending dose (MAD) part of study NN7769-4513. In part 1, participants will receive Mim8 prophylaxis (PPX) subcutaneous (s.c.) administration using enhanced cartridge for 26 weeks. In part 2, participants will receive Mim8 PPX s.c. administration using enhanced cartridge or DV3407 pen-injector once it is approved.
Arm 2
EXPERIMENTALParticipants entering from study NN7769-4514, NN7769-4728 and NN7769-4516. In part 1, participants will receive Mim8 PPX s.c. administration using DV3407 pen-injector for 26 weeks. In part 2, participants will receive Mim8 PPX s.c. administration using DV3407 pen-injector.
Arm 3
EXPERIMENTALIn part 1 and 2, participants will receive Mim8 PPX s.c. administration using DV3407 pen-injector.
Interventions
Participants in arm 1 will administer Mim8 using an enhanced cartridge and switch to the DV3407 pen-injector once it is approved. Participants in arm 2 and 3 will use the DV3407 pen injector.
Eligibility Criteria
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 based on medical records.
- Ongoing participation in study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728 at the time of transfer. Participant should qualify either of the following criteria:
- Participant from study NN7769-4513, who has participated in the extension part of the study for at least 12 weeks prior to enrolment in study NN7769-4532, or,
- Participant has completed the end of treatment visit for study NN7769-4514, NN7769-4516 or NN7769-4728.
- Participant and/or participant's parent(s)/participant's Legally acceptable representative (LAR) willingness and ability to comply with scheduled visits and study procedures, including the completion of diary.
You may not qualify if:
- Any disorder, except for conditions associated with haemophilia A, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.
- Participant who has discontinued or been withdrawn from studies NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728.
- Previous participation in this study. Participation is defined as signed informed consent.
- Female who is pregnant, breast-feeding or intends to become pregnant.
- Female of child-bearing potential and not using a highly effective contraceptive method (highly effective contraceptive measures or as required by local regulation or practice).
- Participation (i.e., signed informed consent) in any interventional, clinical study (except from study NN7769-4513, NN7769-4514, NN7769-4516, or NN7769-4728) of an approved or non-approved investigational medicinal product.
- Any planned major surgery, during part 1 of the study.
- Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.
- Arm 3:
- 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 severe haemophilia A (endogenous FVIII activity less than (\<) 1 percentage \[%\]) with or without FVIII inhibitors based on medical records.
- Aged \<1 year at the time of signing informed consent.
- Body weight greater than or equal to (≥) 3.2 kilograms at the time of signing informed consent.
- previously untreated patients (PUPs) or minimally treated patients (MTPs) (i.e., up to 5 days of exposure to haemophilia-related treatment such as plasma-derived FVIII, recombinant FVIII, fresh frozen plasma, cryoprecipitate, or whole blood products).
- Full-term pregnancy (gestational age ≥37 weeks).
- +21 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Novo Nordisk A/Slead
Study Sites (140)
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, 90027, United States
UC Denver Hemoph & Thrombo Ctr
Aurora, Colorado, 80045, United States
Univ of Miami/SCCC
Miami, Florida, 33136, United States
St Joseph's Hospital Foundation
Tampa, Florida, 33607, United States
Children's Healthcare Atlanta
Atlanta, Georgia, 30329, United States
Rush University Med. Cntr
Chicago, Illinois, 60612, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, 46260, United States
University of Iowa_Iowa City
Iowa City, Iowa, 52242, United States
Central Michigan University
Detroit, Michigan, 48201, United States
Univ Hosp Cleveland Med Ctr
Cleveland, Ohio, 44106, United States
Dayton Children Hemostati Ctr
Dayton, Ohio, 45404, United States
Penn State MS Hershey Med Ctr
Hershey, Pennsylvania, 17033-2360, United States
St Christopher Hosp for Child
Philadelphia, Pennsylvania, 19134, United States
Vanderbilt U Med Ctr_Nashville
Nashville, Tennessee, 37212, United States
Universitätsklinik für Innere Medizin V
Innsbruck, 6020, Austria
Universitätsklinik für Innere Medizin V
Innsbruck, A 6020, Austria
AKH - Klin. Abt. f. Haematologie u. Haemostaseologie
Vienna, 1090, Austria
Cliniques universitaires Saint-Luc - Service Hématologie
Brussels, 1200, Belgium
UZ Leuven - Kindergeneeskunde
Leuven, 3000, Belgium
SHAT of Haematological Diseases EAD, Clinic of Paediatric Clinical Haematology
Sofia, 1797, Bulgaria
McMaster University
Hamilton, Ontario, L8N 3Z5, Canada
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, 100045, China
Beijing Children's Hospital，Capital Medical University
Beijing, Beijing Municipality, 100045, China
Haemotology, Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515, China
Nanfang Hospital, Southern Medical University-Haematology
Guangzhou, Guangdong, 510515, China
Tongji Hospital, Tongji Medical College of HUST-Hematology
Wuhan, Hubei, 430030, China
Tongji Hospital, Tongji Medical College of HUST
Wuhan, Hubei, 430030, China
Xiangya Hospital Central-South University
Changsha, Hunan, 410008, China
Jinan Central Hospital Affiliated to Shandong University
Jinan, Shandong, 250062, China
Jinan Central Hospital
Jinan, Shandong, 250062, China
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan, 610074, China
Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology
Tianjin, Tianjin Municipality, 300020, China
Children's Hospital, Zhejiang University School of Medicine-Hematology
Huzhou, Zhejiang, 313200, China
Jinan Central Hospital
Jinan, 250062, China
Rigshospitalet - Department of Haematology, 2081
København Ø, 2100, Denmark
Hospices Civils de Lyon- Hopital Louis Pradel
Bron, 69500, France
Ap-Hp-Hopital de Bicetre-1
Le Kremlin-Bicêtre, 94275, France
Ap-Hp-Hopital de Bicetre
Le Kremlin-Bicêtre, 94275, France
Centre Hospitalier Universitaire de Lille-Institut Coeur Poumon
Lille, 59037, France
Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu
Nantes, 44000, France
Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu
Nantes, Nantes, France
Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain
Berlin, 10249, Germany
Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
Bonn, 53127, Germany
HZRM Haemophilie-Zentrum Rhein Main GmbH
Frankfurt am Main, 60596, Germany
Nirmal Hospital Pvt. Ltd.
Surat, Gujarat, 395002, India
Seth GS medical college and KEM Hospital
Mumbai, Maharashtra, 400012, India
Sahyadri Clinical Research And Development Center
Pune, Maharashtra, 411004, India
Christian Medical College and Hospital
Ludhiana, Punjab, 141008, India
SMS Medical College & Hospital
Jaipur, Rajasthan, 302004, India
CMCV
Ranipet, Tamil Nadu, 632517, India
Post Graduate Institute of Child Health
Noida, Uttar Pradesh, 201303, India
Seth GS medical college and KEM Hospital
Mumbai, 400012, India
St James's CRF
Dublin, Leinster, D08 A978, Ireland
Sheba MC - The Israeli National Hemophilia Center
Tel Litwinsky, 52621, Israel
A.O.U. Policlinico Umberto I
Rome, Lazio, 00161, Italy
AOU Careggi Firenze
Florence, 50134, Italy
IRCCS Humanitas Research Hospital - Centro Trombosi e Malattie Emorragiche
Milan, 20089, Italy
Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122, Italy
Azienda Ospedaliera di Rilievo Nazionale Santobono Pausilipon
Naples, 80122, Italy
Azienda Ospedaliera Santobono Pausilipon - U.S.D. Centro Regionale Pediatrico Malattie della Coagulazione
Naples, 80122, Italy
Ospedale Pediatrico Bambino Ges
Roma, 00165, Italy
A.O.U. Policlinico Umberto I
Rome, 00161, Italy
A.O.U. Città della Salute e della Scienza di Torino-Ospedale
Torino, 10126, Italy
Nagoya University Hospital_Blood Transfusion
Aichi, 466-8560, Japan
Ota Memorial Hospital_Pediatrics
Gunma, 373-8585, Japan
Nara Medical University Hospital_Pediatrics
Nara, 634-8522, Japan
Saitama Children's Med Centre_Hematology-Oncology
Saitama, 330-8777, Japan
Jichi Medical University Hospital_Hematology
Tochigi, 329-0498, Japan
Jichi Medical University Hospital_Pediatrics
Tochigi, 329-0498, Japan
National Center for Child Health and Development_Hematology
Tokyo, 157-8535, Japan
Tokyo Medical Univ. Hospital_Laboratory Medicine
Tokyo, 160-0023, Japan
Ogikubo Hospital_Pediatries & Blood
Tokyo, 167-0035, Japan
Stradini Clinic of Oncology
Riga, 1002, Latvia
Children University Clinical Hospital
Riga, 1004, Latvia
Vilnius University hospital Santaros klinikos
Vilnius, 08661, Lithuania
Centre of Oncology and Hematology, Vilnius University
Vilnius, LT-08406, Lithuania
Hospital Queen Elizabeth 1
Kota Kinabalu, Sabah, 88586, Malaysia
Hospital Ampang
Ampang, Selangor, Selangor, 68000, Malaysia
Centro Multidisciplinario Para El Desarrollo Especializado De La Investigación Clínica En Yucatán S.C.P. (CEMDEICY S.C.P.)
Mérida, 97130, Mexico
AmsterdamUMC AMC
Amsterdam, 1105 AZ, Netherlands
UMC Utrecht, Van Creveldkliniek
Utrecht, 3584 CX, Netherlands
Uniwersytecki Szpital Kliniczny W Poznaniu
Poznan, Greater Poland Voivodeship, 60-569, Poland
Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego
Wroclaw, Lower Silesian Voivodeship, 50-556, Poland
Instytut Hematologii i Transfuzjologii
Warsaw, Masovian Voivodeship, 02-776, Poland
Intytut Hematologii i Transfuzjologii
Warsaw, Masovian Voivodeship, 02-776, Poland
Szpital Uniwersytecki, Oddzial Kliniczny Hematologii
Krakow, 30-688, Poland
Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
Lublin, 20-093, Poland
Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego
Wroclaw, 50-556, Poland
Unidade Local De Saúde De Coimbra, E.P.E.
Coimbra, 3000-459, Portugal
Unidade Local De Saúde De São José, E.P.E.- Hospital D. Estefânia
Lisbon, 1169-045, Portugal
Centro Hospitalar Universitario De Santo Antonio E.P.E
Porto, 4050-342, Portugal
ULS De São João, E.P.E. - Hospital de São João
Porto, 4200-319, Portugal
Institut Oncologic "Prof. Dr. Ion Chiricuta" Cluj Napoca
Cluj-Napoca, 400015, Romania
King Faisal Specialist Hospital & Research Centre, Riyadh
Riyadh, 12713, Saudi Arabia
Clinical Centre of Serbia, Institute for Haematology
Belgrade, 11000, Serbia
Clinical Centre of Vojvodina
Novi Sad, 21000, Serbia
Univerzitna Nemocnica Martin
Martin, 036 59, Slovakia
Vseobecna nemocnica Rimavska Sobota
Rimavská Sobota, 979 01, Slovakia
Vranovska nemocnica, a.s.
Vranov nad Topľou, 09327, Slovakia
Wits Bara Clinical Trial Site
Johannesburg, Gauteng, 2013, South Africa
Charlotte Maxeke Johannesburg Academic Hospital
Parktown, Johannesburg, Gauteng, 2193, South Africa
Daejeon Eulji Medical Center, Eulji University
Daejeon, 35233, South Korea
Severance Hospital, Yonsei University Health System
Seoul, 03722, South Korea
Gangdong Kyung Hee University Hospital
Seoul, 05278, South Korea
Kyung Hee University Hospital at Gangdong
Seoul, 05278, South Korea
Hospital Vall d'Hebron
Barcelona, 08035, Spain
Hospital Sant Joan de Déu
Esplugues Llobregat, 08950, Spain
Hospital Universitario La Paz
Madrid, 28046, Spain
Hospital Carlos Haya
Málaga, 29010, Spain
Hospital Regional Universitario de Málaga
Málaga, 29010, Spain
Universitätsklinik für Hämatologie
Bern, 3010, Switzerland
Pädiatrische Onkologie-Hämatologie
Lucerne, 6000, Switzerland
Zentrum für Labormedizin
Sankt Gallen, 9008, Switzerland
Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie
Zurich, 8091, Switzerland
Chung Shan Medical University Hospital
Taichung, 402, Taiwan
Taichung Veterans General Hospital
Taichung, 407, Taiwan
National Taiwan University Hospital_main
Taipei, 100, Taiwan
National Taiwan University Hospital
Taipei, 100, Taiwan
Gazi University
Ankara, Beşevler/Ankara, 06500, Turkey (Türkiye)
Gazi Üniversitesi Hastanesi- Hematoloji
Ankara, Beşevler/Ankara, 06500, Turkey (Türkiye)
Istanbul University Oncology Institute
Capa-ISTANBUL, Capa-ISTANBUL, 34093, Turkey (Türkiye)
İstanbul Üniversitesi İstanbul Tıp Fakültesi Hastanesi- Onkoloji Enstitüsü
Capa-ISTANBUL, Capa-ISTANBUL, 34093, Turkey (Türkiye)
Akdeniz Üniversitesi Hastanesi- Hematoloji
Antalya, Konyaaltı/ Antalya, 01010, Turkey (Türkiye)
Acıbadem Adana Hastanesi-Hematoloji
Adana, 01130, Turkey (Türkiye)
Hacettepe Universitesi
Ankara, 06100, Turkey (Türkiye)
Hacettepe Üniversitesi Hastanesi- Hematoloji
Ankara, 06100, Turkey (Türkiye)
Akdeniz Universitesi
Antalya, 01010, Turkey (Türkiye)
Ege Universitesi Tip Fakultesi
Bornova-IZMIR, 35100, Turkey (Türkiye)
Ege Üniversitesi Hastanesi- Hematoloji
Bornova-IZMIR, 35100, Turkey (Türkiye)
Trakya University
Edirne, 22030, Turkey (Türkiye)
Trakya Üniversitesi Tıp Fakültesi Hastanesi-Hematoloji
Edirne, 22030, Turkey (Türkiye)
Belfast City Hospital
Belfast, BT9 78B, United Kingdom
University Hospital of Wales - Haemophilia
Cardiff, CF14 4XW, United Kingdom
Royal Free Haemophilia Comprehensive Care Center
London, NW3 2QG, United Kingdom
Royal Free Hospital - Haemophilia
London, NW3 2QG, United Kingdom
Evelina London Children's Hospital - Haemophilia
London, SE1 7EH, United Kingdom
St Thomas' Hospital - Haemostasis and Thrombosis Centre
London, SE1 7EH, United Kingdom
Royal Hallamshire Hospital
Sheffield, S10 2JF, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Clinical Transparency dept. 2834
Novo Nordisk A/S
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 4, 2023
First Posted
January 17, 2023
Study Start
February 13, 2023
Primary Completion (Estimated)
June 30, 2028
Study Completion (Estimated)
June 30, 2028
Last Updated
March 24, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will share
According to the Novo Nordisk disclosure commitment on novonordisk-trials.com.