Haemophilia APhase 3FRONTIER4NCT05685238
A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A
A Phase 3 study testing Mim8 in people with Haemophilia A and Haemophilia A With Inhibitors, run by Novo Nordisk A/S at 141 sites in 32 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haemophilia A, Haemophilia A With Inhibitors
- What is being tested?
- Mim8
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 451 participants
- Where
- 141 sitesin 32 countries
- Study dates
- 13 Feb 2023to 30 Jun 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
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- 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).
- Participant's parent(s)/LAR(s) willingness and ability to comply with scheduled visits and Arm 3 (infant) procedures, including the completion of diary and patient reported outcome (PRO) questionnaire.
- Participants <3 months of age must show no signs of active intracranial haemorrhage at screening. This is confirmed by cranial ultrasound performed according to local practice and regardless of delivery method.
- Receipt of vitamin K prophylaxis (as per local standard practice).
- Availability of historical results in medical records for:
- activated partial thromboplastin time (aPTT)
- FVIII levels.
- Availability of historical results in medical records or pre-dose sample taken for:
- fibrinogen
- haematology parameters
- biochemistry parameters (aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT), bilirubin and creatinine).
Who cannot join
- 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.
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- 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:
- Known or suspected hypersensitivity to trial product or related products.
- Previous participation in study 4532. Participation is defined as signed informed consent.
- Participation (i.e., signed informed consent) in any interventional clinical study with receipt of the last dose within 6 months (or 5 half-lives of the investigational medicinal product, whichever is shorter) before planned enrolment.
- Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months (or 5 half-lives of the medicinal product, whichever is shorter) before planned enrolment.
- Known congenital or acquired coagulation disorders other than haemophilia A.
- Other conditions (e.g., autoimmune disease) or laboratory abnormality that may increase the risk of bleeding or thrombosis, as evaluated by the investigator. Any disorder, except for conditions associated with haemophilia A, that in the investigator's opinion might jeopardise the participant's safety or compliance with the protocol.
- Lack of adequate parental/legally acceptable representative (LAR) support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary.
- Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease.
- Any planned major surgery, during part 1 of Arm 3 (infant). For definition of major surgery.
- Immune tolerance induction planned to take place after treatment initiation.
- Hepatic dysfunction defined as AST and/or ALT greater than (>) 3 times the upper limit of normal (ULN) combined with total bilirubin >1.5 times the ULN.
- Serum creatinine above 1.5 times the ULN.
Trial locations and contacts
141 sites in 32 countries.
141 sites in 32 countries
United States15 sites
Children's Healthcare Atlanta
Atlanta, Georgia, 30329
UC Denver Hemoph & Thrombo Ctr
Aurora, Colorado, 80045
Rush University Med. Cntr
Chicago, Illinois, 60612
Rush University Medical Center
Chicago, Illinois, 60612
Univ Hosp Cleveland Med Ctr
Cleveland, Ohio, 44106
Dayton Children Hemostati Ctr
Dayton, Ohio, 45404
Central Michigan University
Detroit, Michigan, 48201
Penn State MS Hershey Med Ctr
Hershey, Pennsylvania, 17033-2360
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, 46260
University of Iowa_Iowa City
Iowa City, Iowa, 52242
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, 90027
Univ of Miami/SCCC
Miami, Florida, 33136
Vanderbilt U Med Ctr_Nashville
Nashville, Tennessee, 37212
St Christopher Hosp for Child
Philadelphia, Pennsylvania, 19134
St Joseph's Hospital Foundation
Tampa, Florida, 33607
China13 sites
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, 100045
Beijing Children's Hospital,Capital Medical University
Beijing, Beijing Municipality, 100045
Xiangya Hospital Central-South University
Changsha, Hunan, 410008
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan, 610074
Haemotology, Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
Nanfang Hospital, Southern Medical University-Haematology
Guangzhou, Guangdong, 510515
Children's Hospital, Zhejiang University School of Medicine-Hematology
Huzhou, Zhejiang, 313200
Jinan Central Hospital Affiliated to Shandong University
Jinan, Shandong, 250062
Jinan Central Hospital
Jinan, Shandong, 250062
Jinan Central Hospital
Jinan, 250062
Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology
Tianjin, Tianjin Municipality, 300020
Tongji Hospital, Tongji Medical College of HUST
Wuhan, Hubei, 430030
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology-Hematology
Wuhan, Hubei, 430030
Turkey (Türkiye)13 sites
Acıbadem Adana Hastanesi-Hematoloji
Adana, 01130
Gazi University
Ankara, Beşevler/Ankara, 06500
Gazi Üniversitesi Hastanesi- Hematoloji
Ankara, Beşevler/Ankara, 06500
Hacettepe Universitesi
Ankara, 06100
Hacettepe Üniversitesi Hastanesi- Hematoloji
Ankara, 06100
Akdeniz Üniversitesi Hastanesi- Hematoloji
Antalya, Konyaaltı/ Antalya, 01010
Akdeniz Universitesi
Antalya, 01010
Ege Universitesi Tip Fakultesi
Bornova-IZMIR, 35100
Ege Üniversitesi Hastanesi- Hematoloji
Bornova-IZMIR, 35100
Istanbul University Oncology Institute
Capa-ISTANBUL, Capa-ISTANBUL, 34093
İstanbul Üniversitesi İstanbul Tıp Fakültesi Hastanesi- Onkoloji Enstitüsü
Capa-ISTANBUL, Capa-ISTANBUL, 34093
Trakya University
Edirne, 22030
Trakya Üniversitesi Tıp Fakültesi Hastanesi-Hematoloji
Edirne, 22030
Italy9 sites
AOU Careggi Firenze
Florence, 50134
IRCCS Humanitas Research Hospital - Centro Trombosi e Malattie Emorragiche
Milan, 20089
Fondazione IRCSS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122
Azienda Ospedaliera di Rilievo Nazionale Santobono Pausilipon
Naples, 80122
Azienda Ospedaliera Santobono Pausilipon - U.S.D. Centro Regionale Pediatrico Malattie della Coagulazione
Naples, 80122
Ospedale Pediatrico Bambino Ges
Roma, 00165
A.O.U. Policlinico Umberto I
Rome, Lazio, 00161
A.O.U. Policlinico Umberto I
Rome, 00161
AOU Città della Salute e della Scienza di Torino - Ospedale Regina Margherita
Torino, 10126
Japan9 sites
Nagoya University Hospital_Blood Transfusion
Aichi, 466-8560
Ota Memorial Hospital_Pediatrics
Gunma, 373-8585
Nara Medical University Hospital_Pediatrics
Nara, 634-8522
Saitama Children's Med Centre_Hematology-Oncology
Saitama, 330-8777
Jichi Medical University Hospital_Hematology
Tochigi, 329-0498
Jichi Medical University Hospital_Pediatrics
Tochigi, 329-0498
National Center for Child Health and Development_Hematology
Tokyo, 157-8535
Tokyo Medical Univ. Hospital_Laboratory Medicine
Tokyo, 160-0023
Ogikubo Hospital_Pediatries & Blood
Tokyo, 167-0035
India8 sites
SMS Medical College & Hospital
Jaipur, Rajasthan, 302004
Christian Medical College and Hospital
Ludhiana, Punjab, 141008
Seth GS medical college and KEM Hospital
Mumbai, Maharashtra, 400012
Seth GS medical college and KEM Hospital
Mumbai, 400012
Post Graduate Institute of Child Health
Noida, Uttar Pradesh, 201303
Sahyadri Clinical Research And Development Center
Pune, Maharashtra, 411004
CMCV
Ranipet, Tamil Nadu, 632517
Nirmal Hospital Pvt. Ltd.
Surat, Gujarat, 395002
France7 sites
Hospices Civils de Lyon- Hopital Louis Pradel
Bron, 69500
Ap-Hp-Hopital de Bicetre-1
Le Kremlin-Bicêtre, 94275
Ap-Hp-Hopital de Bicetre
Le Kremlin-Bicêtre, 94275
Centre Hospitalier Universitaire de Lille-Institut Coeur Poumon
Lille, 59037
INSTITUT COEUR POUMON_Service d'hémostase et transfusion
Lille, 59037
Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu
Nantes, 44000
Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu
Nantes, Nantes
Poland7 sites
Szpital Uniwersytecki, Oddzial Kliniczny Hematologii
Krakow, 30-688
Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
Lublin, 20-093
Uniwersytecki Szpital Kliniczny W Poznaniu
Poznan, Greater Poland Voivodeship, 60-569
Instytut Hematologii i Transfuzjologii
Warsaw, Masovian Voivodeship, 02-776
Intytut Hematologii i Transfuzjologii
Warsaw, Masovian Voivodeship, 02-776
Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego
Wroclaw, Lower Silesian Voivodeship, 50-556
Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego
Wroclaw, 50-556
United Kingdom7 sites
Belfast City Hospital
Belfast, BT9 78B
University Hospital of Wales - Haemophilia
Cardiff, CF14 4XW
Royal Free Haemophilia Comprehensive Care Center
London, NW3 2QG
Royal Free Hospital - Haemophilia
London, NW3 2QG
Evelina London Children's Hospital - Haemophilia
London, SE1 7EH
St Thomas' Hospital - Haemostasis and Thrombosis Centre
London, SE1 7EH
Royal Hallamshire Hospital
Sheffield, S10 2JF
Spain5 sites
Hospital Vall d'Hebron
Barcelona, 08035
Hospital Sant Joan de Déu
Esplugues Llobregat, 08950
Hospital Universitario La Paz
Madrid, 28046
Hospital Carlos Haya
Málaga, 29010
Hospital Regional Universitario de Málaga
Málaga, 29010
Portugal4 sites
Unidade De Coimbra, E.P.E. - Hospital da Universidade de Coimbra
Coimbra, 3000-459
ULS De São José, E.P.E.- Hospital D. Estefânia
Lisbon, 1169-045
ULS De Santo António, E.P.E. _H.Santo António_ Imunohemoterapia
Porto, 4050-342
ULS São João, E.P.E._H.São João_Imunohemoterapia
Porto, 4200-319
South Korea4 sites
Daejeon Eulji Medical Center, Eulji University
Daejeon, 35233
Severance Hospital, Yonsei University Health System
Seoul, 03722
Gangdong Kyung Hee University Hospital
Seoul, 05278
Kyung Hee University Hospital at Gangdong
Seoul, 05278
Switzerland4 sites
Universitätsklinik für Hämatologie
Bern, 3010
Pädiatrische Onkologie-Hämatologie
Lucerne, 6000
Zentrum für Labormedizin
Sankt Gallen, 9008
Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie
Zurich, 8091
Taiwan4 sites
Chung Shan Medical University Hospital
Taichung, 402
Taichung Veterans General Hospital
Taichung, 407
National Taiwan University Hospital_main
Taipei, 100
National Taiwan University Hospital
Taipei, 100
Austria3 sites
Universitätsklinik für Innere Medizin V
Innsbruck, 6020
Universitätsklinik für Innere Medizin V
Innsbruck, A 6020
AKH - Klin. Abt. f. Haematologie u. Haemostaseologie
Vienna, 1090
Germany3 sites
Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain
Berlin, 10249
Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
Bonn, 53127
HZRM Haemophilie-Zentrum Rhein Main GmbH
Frankfurt am Main, 60596
Slovakia3 sites
Univerzitna Nemocnica Martin
Martin, 036 59
Vseobecna nemocnica Rimavska Sobota
Rimavská Sobota, 979 01
Vranovska nemocnica, a.s.
Vranov nad Topľou, 09327
Belgium2 sites
Cliniques universitaires Saint-Luc - Service Hématologie
Brussels, 1200
UZ Leuven - Kindergeneeskunde
Leuven, 3000
Canada2 sites
McMaster University
Hamilton, Ontario, L8N 3Z5
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
Latvia2 sites
Stradini Clinic of Oncology
Riga, 1002
Children University Clinical Hospital
Riga, 1004
Lithuania2 sites
Vilnius University hospital Santaros klinikos
Vilnius, 08661
Centre of Oncology and Hematology, Vilnius University
Vilnius, LT-08406
Malaysia2 sites
Hospital Ampang
Ampang, Selangor, Selangor, 68000
Hospital Queen Elizabeth 1
Kota Kinabalu, Sabah, 88586
Netherlands2 sites
AmsterdamUMC AMC
Amsterdam, 1105 AZ
UMC Utrecht, Van Creveldkliniek
Utrecht, 3584 CX
Serbia2 sites
Clinical Centre of Serbia, Institute for Haematology
Belgrade, 11000
Clinical Centre of Vojvodina
Novi Sad, 21000
South Africa2 sites
Wits Bara Clinical Trial Site
Johannesburg, Gauteng, 2013
Charlotte Maxeke Johannesburg Academic Hospital
Parktown, Johannesburg, Gauteng, 2193
Bulgaria1 site
SHAT of Haematological Diseases EAD, Clinic of Paediatric Clinical Haematology
Sofia, 1797
Denmark1 site
Rigshospitalet - Department of Haematology, 2081
København Ø, 2100
Israel1 site
Sheba MC - The Israeli National Hemophilia Center
Tel Litwinsky, 52621
Mexico1 site
Centro Multidisciplinario Para El Desarrollo Especializado De La Investigación Clínica En Yucatán S.C.P. (CEMDEICY S.C.P.)
Mérida, 97130
Romania1 site
Institut Oncologic "Prof. Dr. Ion Chiricuta" Cluj Napoca
Cluj-Napoca, 400015
Saudi Arabia1 site
King Faisal Specialist Hospital & Research Centre, Riyadh
Riyadh, 12713
About this study
Official title: Open-label, Long-term Safety and Efficacy Study of Mim8 in Participants With Haemophilia A With or Without Inhibitors
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.
- Conditions
- Haemophilia A, Haemophilia A With Inhibitors
- Being tested
- Mim8
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mim8Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Participants 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. Receives: Drug: Mim8 |
| Arm 2 | Experimental | Participants 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. Receives: Drug: Mim8 |
| Arm 3 | Experimental | In part 1 and 2, participants will receive Mim8 PPX s.c. administration using DV3407 pen-injector. Receives: Drug: Mim8 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Arm 1 and 2: Number of treatment emergent adverse events
Measured as count of events.
Measured: From week 0 until end of study (up to 283 weeks)
Arm 3: Number of treatment emergent adverse events
Measured as count of events.
Measured: 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
Measured as count of reactions.
Measured: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Occurrence of anti Mim8 antibodies
Measured as count of participants.
Measured: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated bleeding episodes
Measured as count of bleeds.
Measured: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated spontaneous bleeding episodes
Measured as count of bleeds.
Measured: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated traumatic bleeding episodes
Measured as count of bleeds.
Measured: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated joint bleeding episodes
Measured as count of bleeds.
Measured: From week 0 until end of treatment (up to 262 weeks)
Arm 2: Number of treated target joint bleeding episodes
Measured as count of bleeds.
Measured: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Mim8 plasma concentration
Measured as micrograms per milliliter (µg/mL).
Measured: From week 0 until end of treatment (up to 262 weeks)
Arm 2: Device handling using haemophilia device assessment tool (HDAT) (applicable for participants in arm 2 only)
Measured as percentage of participants.
Measured: From week 26 until end of treatment (up to 262 weeks)
Arm 3: Number of injection site reactions
Measured as count of reactions.
Measured: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Occurrence of anti Mim8 antibodies
Measured as count of participants.
Measured: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Number of treated bleeding episodes
Measured as count of bleeds.
Measured: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Number of treated spontaneous bleeding episodes
Measured as count of bleeds.
Measured: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Number of treated traumatic bleeding episodes
Measured as count of bleeds.
Measured: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Mim8 plasma concentration
Measured as µg/mL.
Measured: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Device handling using haemophilia device assessment tool (HDAT)
Measured as percentage of participants.
Measured: At week 26 and week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jan 2023
- Study start13 Feb 2023
- Primary completion30 Jun 2028 (estimated)
- Study completion30 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Haemophilia AStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 451 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Transparency dept. 2834, Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05685238
- Sponsor's protocol ID
- NN7769-4532
- Other IDs
- U1111-1274-4426, 2022-502215-10
- Acronym
- FRONTIER4
- Status last verified
- Sep 2026
- First submitted
- 4 Jan 2023
- Last updated
- 9 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com.
Publications
Matsushita T, Banchev A, Bovet J, Chowdary P, Garcia Fernandez LM, Jimenez-Yuste V, Kavakli K, Kremer Hovinga JA, Windyga J, Young G. Denecimig (Mim8) prophylaxis once-every-2-weeks for hemophilia A with or without inhibitors - 26-week results from FRONTIER. Blood Adv. 2026 Jul 17:bloodadvances.2025019095. doi: 10.1182/bloodadvances.2025019095. Online ahead of print.
PubMed 42469164
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label, Long-term Safety and Efficacy Study of Mim8 in Participants With Haemophilia A With or Without Inhibitors
- ClinicalTrials.gov ID
- NCT05685238
- Acronym / sponsor ID
- FRONTIER4 / NN7769-4532
- Conditions
- Haemophilia A, Haemophilia A With Inhibitors
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 451 participants (estimated)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 13 Feb 2023 (actual)
- Primary completion
- 30 Jun 2028 (estimated)
- Study completion
- 30 Jun 2028 (estimated)
- First posted
- 17 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Drug: Mim8Participants 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 | Experimental | Drug: Mim8Participants 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 | Experimental | Drug: Mim8In part 1 and 2, participants will receive Mim8 PPX s.c. administration using DV3407 pen-injector. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (22 listed)
- 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 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.
- 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.
Key exclusion criteria (21 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mim8Participants 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. | Drug | Arm 1, Arm 2, Arm 3 |
Primary outcome
Arm 1 and 2: Number of treatment emergent adverse events
Time frame: From week 0 until end of study (up to 283 weeks)
Measured as count of events.
Arm 3: Number of treatment emergent adverse events
Time frame: From treatment initiation (week 0) until end of study (up to 124 weeks)
Measured as count of events.
16 secondary outcomes
Arm 1 and 2: Number of injection site reactions
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Occurrence of anti Mim8 antibodies
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated bleeding episodes
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated spontaneous bleeding episodes
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated traumatic bleeding episodes
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Number of treated joint bleeding episodes
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 2: Number of treated target joint bleeding episodes
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 1 and 2: Mim8 plasma concentration
Time frame: From week 0 until end of treatment (up to 262 weeks)
Arm 2: Device handling using haemophilia device assessment tool (HDAT) (applicable for participants in arm 2 only)
Time frame: From week 26 until end of treatment (up to 262 weeks)
Arm 3: Number of injection site reactions
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Occurrence of anti Mim8 antibodies
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Number of treated bleeding episodes
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Number of treated spontaneous bleeding episodes
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Number of treated traumatic bleeding episodes
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Mim8 plasma concentration
Time frame: From treatment initiation (week 0) until end of treatment (up to 103 weeks)
Arm 3: Device handling using haemophilia device assessment tool (HDAT)
Time frame: At week 26 and week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2026.
- Derived from this study: PubMed 42469164Matsushita T, Banchev A, Bovet J, Chowdary P, Garcia Fernandez LM, Jimenez-Yuste V, Kavakli K, Kremer Hovinga JA, Windyga J, Young G. Denecimig (Mim8) prophylaxis once-every-2-weeks for hemophilia A with or without inhibitors - 26-week results from FRONTIER. Blood Adv. 2026 Jul 17:bloodadvances.2025019095. doi: 10.1182/bloodadvances.2025019095. Online ahead of print.
- Individual participant dataSharing planned: Yes. According to the Novo Nordisk disclosure commitment on novonordisk-trials.com.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Transparency dept. 2834 study director
Novo Nordisk A/S
141 sites in 32 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mim8 in people with Haemophilia A and Haemophilia A With Inhibitors, run by Novo Nordisk A/S at 141 sites in 32 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 22 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 141 sites in United States, China, Turkey (Türkiye), Italy, Japan and 27 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Arm 1 and 2: Number of treatment emergent adverse events (time frame: From week 0 until end of study (up to 283 weeks)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05685238, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Sep 2026.