A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors

A Multinational, Open-label, Randomised, Controlled Study to Investigate Efficacy and Safety of NNC0365-3769 (Mim8) in Adults and Adolescents With Haemophilia A With or Without Inhibitors

This study is investigating how Mim8 works compared to other medicines in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII).

When and how often participants will receive Mim8 is dependent on their previous treatment - but is otherwise decided by chance. Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.

The study will last 54-124 weeks (12-29 months) depending on how long participants will be followed in run-in before they start treatment and if they continue in the follow period or transfer to an open label extension study. Participants will have 12-17 clinic visits.

Study Overview

• Status: Completed
• Conditions: Haemophilia A With Inhibitors; Haemophilia A
• Intervention / Treatment: Drug: NNC0365-3769 (Mim8)

• Study Type: Interventional
• Enrollment (Actual): 281
• Phase: Phase 3

Contacts and Locations

Study Locations

• Austria
  • Innsbruck, Austria, 6020
    • Universitätsklinik für Innere Medizin V
  • Vienna, Austria, 1090
    • AKH - Klin. Abt. f. Haematologie u. Haemostaseologie
• Belgium
  • Brussels, Belgium, 1200
    • Cliniques universitaires Saint-Luc - Service Hématologie
  • Edegem, Belgium, 2650
    • UZ Antwerpen - UZA - Kinderhemato-Oncologie
  • Leuven, Belgium, 3000
    • UZ Leuven - Kindergeneeskunde
• Canada
  • Manitoba
    • Winnipeg, Manitoba, Canada, R3T 2N2
      • University of Manitoba
  • New Brunswick
    • Saint John, New Brunswick, Canada, E2L 4L4
      • St. John Regional Hospital
  • Newfoundland and Labrador
    • St. John's, Newfoundland and Labrador, Canada, A1B 3V6
      • Eastern Health Authority
  • Ontario
    • Hamilton, Ontario, Canada, L8N 3Z5
      • McMaster University Medical Center
    • Hamilton, Ontario, Canada, L8N 3Z5
      • McMaster University
    • London, Ontario, Canada, N6A 5W9
      • London Health Sciences Center Ontario
    • Toronto, Ontario, Canada, M5G 1X8
      • The Hospital for Sick Children
  • Quebec
    • Montreal, Quebec, Canada, HIT 2M4
      • Hopital Maisonneuve Rosemont
  • Saskatchewan
    • Saskatoon, Saskatchewan, Canada, S7N 0W8
      • Royal University Hospital
• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100045
      • Beijing Children's Hospital, Capital Medical University
  • Guangdong
    • Guangzhou, Guangdong, China, 510515
      • Haemotology, Nanfang Hospital, Southern Medical University
    • Guangzhou, Guangdong, China, 510515
      • Nanfang Hospital, Southern Medical University-Haematology
  • Hubei
    • Wuhan, Hubei, China, 430030
      • Tongji Hospital, Tongji Medical College of HUST
    • Wuhan, Hubei, China, 430030
      • Tongji Hospital, Tongji Medical College of HUST-Hematology
  • Hunan
    • Changsha, Hunan, China, 410008
      • Xiangya Hospital Central-South University
  • Jiangsu
    • Suzhou, Jiangsu, China, 215006
      • the First Affiliated Hospital of Soochow University
  • Shandong
    • Ji'Nan, Shandong, China, 250000
      • Jinan Central Hospital
    • Jinan, Shandong, China, 250000
      • Jinan Central Hospital
  • Sichuan
    • Chengdu, Sichuan, China, 610074
      • Chengdu Women's and Children's Central Hospital
  • Tianjin Municipality
    • Tianjin, Tianjin Municipality, China, 300020
      • Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology
  • Yunnan
    • Kunming, Yunnan, China, 650101
      • The Second Affiliated Hospital of Kunming Medical University
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310052
      • The Children's Hospital, Zhejiang University School of Medicine
    • Hangzhou, Zhejiang, China, 310052
      • Children's Hospital, Zhejiang University School of Medicine
• Czechia
  • Prague, Czechia, 128 20
    • Ustav Hematologie a krevni tranfuze
• Denmark
  • København Ø, Denmark, 2100
    • Rigshospitalet - Department of Haematology, 2081
• France
  • Bordeaux, France, 33076
    • Centre Hospitalier Universitaire de Bordeaux-Hopital Pellegrin
  • Bron, France, 69500
    • Hôpital cardiologique Louis Pradel
  • Bron, France, 69500
    • Hospices Civils de Lyon- Hopital Louis Pradel-1
  • Le Kremlin-Bicêtre, France, 94275
    • Ap-Hp-Hopital de Bicetre-1
  • Lille, France, 59037
    • Centre Hospitalier Universitaire de Lille-Institut Coeur Poumon
  • Nantes, France, Nantes
    • Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu
  • Paris, France, 75015
    • Ap-Hp-Hopital Necker-1
  • Rennes, France, 35033
    • Centre Hospitalier Universitaire de Rennes - Hôpital Pontchaillou
• Germany
  • Berlin, Germany, 10249
    • Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain
  • Bonn, Germany, 53127
    • Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
  • Frankfurt am Main, Germany, 60596
    • HZRM Haemophilie-Zentrum Rhein Main GmbH
• India
  • Gujarat
    • Surat, Gujarat, India, 395002
      • Nirmal Hospital Pvt. Ltd.
  • Maharashtra
    • Mumbai, Maharashtra, India, 400012
      • Seth GS Medical College & KEM Hospital
  • Punjab
    • Ludhiana, Punjab, India, 141008
      • Christian Medical College
  • Tamil Nadu
    • Ranipet, Tamil Nadu, India, 632517
      • CMCV
  • Uttar Pradesh
    • Noida, Uttar Pradesh, India, 201303
      • Post Graduate Institute of Child Health
• Ireland
  • Dublin, Ireland, D12 N512
    • CHI Crumlin Haematology
  • Leinster
    • Dublin, Leinster, Ireland, D08 A978
      • St James's CRF
• Israel
  • Tel Litwinsky, Israel, 52621
    • Sheba MC - The Israeli National Hemophilia Center
• Italy
  • Florence, Italy, 50134
    • Dipartimento di Ematologia Univ. Firenze
  • Milan, Italy, 20089
    • IRCCS Humanitas Research Hospital - Centro Trombosi e Malattie Emorragiche
• Japan
  • Aichi, Japan, 466-8560
    • Nagoya University Hospital_Blood Transfusion
  • Kitakyusyu-shi, Fukuoka, Japan, 807 8555
    • Hospital of the University of Occupational And Environmental Health Japan, Pediatrics
  • Okinawa, Japan, 901-1193
    • Nanbu Medical Center & Children's Medical Center
  • Saitama, Japan, 330-8777
    • Saitama Children's Med Centre_Hematology-Oncology
  • Shizuoka, Japan, 420-8660
    • Shizuoka Children's Hospital, Hematology-Oncology
  • Tochigi, Japan, 329-0498
    • Jichi Medical University Hospital_Hematology
  • Tochigi, Japan, 329-0498
    • Jichi Medical University Hospital_Pediatrics
  • Tokyo, Japan, 167-0035
    • Ogikubo Hospital_Pediatries & Blood
  • Tokyo, Japan, 157-8535
    • National Center for Child Health and Development_Hematology
  • Tokyo, Japan, 160-0023
    • Tokyo Medical Univ. Hospital_Laboratory Medicine
• Latvia
  • Riga, Latvia, 1002
    • Stradini Clinic of Oncology
  • Riga, Latvia, 1004
    • Children University Clinical Hospital
• Lithuania
  • Kaunas, Lithuania, LT-50161
    • Hospital of LUHS "Kauno Klinikos"
  • Vilnius, Lithuania, LT-08661
    • Vilnius University Hospital Santaros Klinikos
  • Vilnius, Lithuania, 08406
    • Children Oncohaematology department Children's Hospital,
• Malaysia
  • Selangor Darul Ehsan, Malaysia, 68000
    • Hospital Ampang
  • Sabah
    • Kota Kinabalu, Sabah, Malaysia, 88586
      • Hospital Queen Elizabeth 1
  • Selangor
    • Ampang, Selangor, Malaysia, 68000
      • Hospital Ampang
• Mexico
  • Mérida, Mexico, 97130
    • Centro Multidisciplinario Para El Desarrollo Especializado De La Investigación Clínica En Yucatán S.C.P. (CEMDEICY S.C.P.)
• Netherlands
  • Amsterdam, Netherlands, 1105 AZ
    • Academisch Medisch Centrum
  • Rotterdam, Netherlands, 3015 GD
    • Erasmus MC
  • Utrecht, Netherlands, 3584 CX
    • UMC Utrecht, Van Creveldkliniek
• Poland
  • Lublin, Poland, 20-093
    • Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
  • Lesser Poland Voivodeship
    • Krakow, Lesser Poland Voivodeship, Poland, 30-688
      • Szpital Uniwersytecki, Oddzial Kliniczny Hematologii
  • Lower Silesian Voivodeship
    • Wroclaw, Lower Silesian Voivodeship, Poland, 50-556
      • Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego
• Portugal
  • Coimbra, Portugal, 3000-561
    • Unidade Local de Saúde de Coimbra, E.P.E.
  • Porto, Portugal, 4200-319
    • Centro Hospitalar de São João_Porto
  • Porto, Portugal, 4099-001
    • Unidade Local de Saúde de Santo António, E.P.E
  • Porto, Portugal, 4200-319
    • ULS São João, E.P.E.
• Romania
  • Bucharest, Romania, 022328
    • Institutul Clinic Fundeni
  • Cluj-Napoca, Romania, 400015
    • Institut Oncologic "Prof. Dr. Ion Chiricuta" Cluj Napoca
  • Timișoara, Romania, 300254
    • Spitalul Clinic Municipal de Urgenta Timisoara
  • Dolj
    • Craiova, Dolj, Romania, 200143
      • Spitalul Clinic Municipal Filantropia Craiova
• Russia
  • Barnaul, Russia, 656024
    • Reg. State Budget Healthc. Inst. Regional Clinical Hospital
  • Kemerovo, Russia, 650066
    • SAHI Kuzbass Hospital(former Regional clinical hospital)
  • Krasnodar, Russia, 350007
    • Children Regional Clinical Hospital
  • Moscow, Russia, 119049
    • Morozovskaya municipal children hospital
  • Moscow, Russia, 125167
    • National Medical Research institution of haemotology
  • Petrozavodsk, Russia, 185019
    • Republican Hospital n.a. V. A. Baranov
  • Saint Petersburg, Russia, 191186
    • City out-patient clinic 37, City Hemophilia Centre
• Saudi Arabia
  • Riyadh, Saudi Arabia, 12713
    • King Faisal Specialist Hospital & Research Centre, Riyadh
• Serbia
  • Belgrade, Serbia, 11000
    • Clinical Centre of Serbia, Institute for Haematology
  • Novi Sad, Serbia, 21000
    • Clinical Centre of Vojvodina
• Slovakia
  • Bratislava, Slovakia, 851 07
    • Nemocnica sv. Cyrila a Metoda, UNB,Klinika hemat. a transfuz
  • Košice, Slovakia, 04001
    • Unilabs Slovensko, s. r. o.
  • Martin, Slovakia, 036 59
    • Univerzitna nemocnica MARTIN
  • Rimavská Sobota, Slovakia, 979 12
    • Vseobecna nemocnica Rimavska Sobota
  • Vranov nad Topľou, Slovakia, 09327
    • Vranovska nemocnica, a.s.
• South Africa
  • Gauteng
    • Johannesburg, Gauteng, South Africa, 2013
      • Wits Bara Clinical Trial Site
    • Parktown, Johannesburg, Gauteng, South Africa, 2193
      • Charlotte Maxeke Johannesburg Academic Hospital
• South Korea
  • Daejeon, South Korea, 35233
    • Daejeon Eulji Medical Center, Eulji University
  • Seoul, South Korea, 03722
    • Severance Hospital, Yonsei University Health System
  • Seoul, South Korea, 05278
    • Kyung Hee University Hospital at Gangdong
• Spain
  • Barcelona, Spain, 08035
    • Hospital Vall d'Hebron
  • Madrid, Spain, 28046
    • Hospital Universitario La Paz
  • Málaga, Spain, 29009
    • Hospital Regional Universitario de Málaga
• Switzerland
  • Bern, Switzerland, 3010
    • Universitätsklinik für Hämatologie
  • Sankt Gallen, Switzerland, 9008
    • Zentrum für Labormedizin
  • Zurich, Switzerland, 8091
    • Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie
• Taiwan
  • Kaohsiung City, Taiwan, 807
    • Kaohsiung Medical University Chung-Ho Memorial Hospital_Dept of Pediatrics
  • Taichung, Taiwan, 402
    • Chung Shan Medical University Hospital
  • Taichung, Taiwan, 407
    • Taichung Veterans General Hospital
  • Taipei, Taiwan, 100
    • National Taiwan University Hospital_Dept of Hematology
• Turkey (Türkiye)
  • Adana, Turkey (Türkiye), 01130
    • Acıbadem Adana Hastanesi-Hematoloji
  • Antalya, Turkey (Türkiye), 01010
    • Akdeniz Universitesi
  • Bornova-IZMIR, Turkey (Türkiye), 35100
    • Ege Universitesi Tip Fakultesi
  • Bornova-IZMIR, Turkey (Türkiye), 35100
    • Ege Üniversitesi Hastanesi- Hematoloji
  • Beşevler/Ankara
    • Ankara, Beşevler/Ankara, Turkey (Türkiye), 06500
      • Gazi University
    • Ankara, Beşevler/Ankara, Turkey (Türkiye), 06500
      • Gazi Üniversitesi Hastanesi- Hematoloji
  • Capa-ISTANBUL
    • Capa-ISTANBUL, Capa-ISTANBUL, Turkey (Türkiye), 34093
      • Istanbul University Oncology Institute
    • Capa-ISTANBUL, Capa-ISTANBUL, Turkey (Türkiye), 34093
      • İstanbul Üniversitesi İstanbul Tıp Fakültesi Hastanesi- Onkoloji Enstitüsü
  • Konyaaltı/ Antalya
    • Antalya, Konyaaltı/ Antalya, Turkey (Türkiye), 01010
      • Akdeniz Üniversitesi Hastanesi- Hematoloji
• Ukraine
  • Dnipro, Ukraine, 49102
    • City Clinical Hospital #4 (Dnipro) - Haematology centre
  • Lviv, Ukraine, 79044
    • Institute of blood pathology and transfusion medicine of NAMSU - General and haematol. surgery
• United Kingdom
  • Cardiff, United Kingdom, CF14 4XW
    • Arthur Bloom Haemophilia Centre
  • London, United Kingdom, NW3 2QG
    • Royal Free Haemophilia Comprehensive Care Centre
  • London, United Kingdom, SE1 7EH
    • St Thomas' Hospital - Haemostasis and Thrombosis Centre
  • Oxford, United Kingdom, OX3 7LJ
    • Oxford Haemophilia Comprehensive Care Center
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85016-7710
      • Arizona H&T Phoenix Child Hosp
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles - Endocrinology
    • Los Angeles, California, United States, 90007
      • Hemophilia Treatment Center
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Univ of Colorado Sch of Med
  • Florida
    • Miami, Florida, United States, 33136
      • Univ of Miami/SCCC
    • Orlando, Florida, United States, 32806
      • Arnold Palmer Hospital for Children
    • Tampa, Florida, United States, 33607
      • St Joseph's Hospital Foundation
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Children HC Atlanta-Adv Pediat
    • Augusta, Georgia, United States, 30912
      • Augusta University
    • Savannah, Georgia, United States, 31404
      • Memorial Health Univ Med Ctr
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Med. Cntr
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Indiana Hemophilia-Thromb Ctr
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa_Iowa City
  • Louisiana
    • New Orleans, Louisiana, United States, 70118-5720
      • Children's Hosp-New Orleans
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan
    • East Lansing, Michigan, United States, 48823
      • Michigan State University
  • Missouri
    • St Louis, Missouri, United States, 63110
      • Washington University School of Medicine_St. Louis
  • Nebraska
    • Omaha, Nebraska, United States, 68105
      • Univ of NE Med Center_Omaha
  • Nevada
    • Las Vegas, Nevada, United States, 89135
      • Cure 4 the Kids Foundation
  • North Carolina
    • Charlotte, North Carolina, United States, 28203
      • Torrence Hemby Ped Hem/Onc Ctr
    • Charlotte, North Carolina, United States, 28204
      • St. Jude Clinic Novant Health
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Child's Hsp Med Ctr
    • Cleveland, Ohio, United States, 44106
      • Univ Hosp Cleveland Med Ctr
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
    • Dayton, Ohio, United States, 45404
      • Dayton Children Hemostati Ctr
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033-2360
      • Penn State MS Hershey Med Ctr
    • Philadelphia, Pennsylvania, United States, 19134
      • St Christopher Hosp for Child
  • Tennessee
    • Kingsport, Tennessee, United States, 37660
      • Balad Hlth Sys-Welmont CardCVA
    • Nashville, Tennessee, United States, 37232-9830
      • Vanderbilt Hemostasis Thrombosis Clinic
  • Texas
    • Dallas, Texas, United States, 75235
      • Children's Medical Center_Dallas
    • Houston, Texas, United States, 77030
      • Univ TX Hlth Sci Ctr Houston
  • Washington
    • Spokane, Washington, United States, 99204
      • Providence Sacred Heart Medical Center & Children's Hospital
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Children's Hospital of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. 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.
2. Male or female participants with diagnosis of congenital haemophilia A of any severity based on medical records.
3. Participant has been prescribed treatment with factor VIII concentrates or bypassing agent in the last 26 weeks prior to screening.
4. Age above or equal to 12 years at the time of signing informed consent.
5. Body weight greater than or equal to 30 kg.
6. Applicable to participants treated with on-demand/no prophylaxis prior to enrolment: ≥5 bleeds in the last 26 weeks prior to screening visit, for which factor VIII concentrates or bypassing agent has been prescribed.
7. Applicable to participants with FVIII activity ≥1% who are on prophylactic treatment: ≥1 bleed in the last 26 weeks prior to screening visit, for which factor VIII concentrates or bypassing agent has been prescribed.
8. Willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires.

Exclusion Criteria:

1. Previous participation in this study. Participation is defined as signed informed consent.
2. 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 randomisation.
3. 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 randomisation, for participants not included in the run-in.
4. Female who is pregnant, breast-feeding or intends to become pregnant or is of child-bearing potential and not using a highly effective contraceptive method. Breast feeding is allowed only during the run-in period.
5. 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.
6. Known or suspected hypersensitivity to trial product(s), any constituents of the product or to related products.
7. Receipt of gene therapy at any given time point.
8. Ongoing or planned immune tolerance induction (ITI) therapy.
9. Major surgery planned to take place after screening.
10. Known congenital or acquired coagulation disorders other than haemophilia A.
11. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) above 3 times the upper limit combined with total bilirubin above1.5 times the upper limit measured at screening.
12. Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) below or equal to 30 ml/min/1.73 m^2 for serum creatinine measured at screening.
13. 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.
14. Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation.
15. Other conditions (e.g., autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis as evaluated by the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: no PPX- no PPX - Mim8 PPXQW/QM; Participants not receiving prophylaxis will not enter the run-in period. In arm 1, participants will be randomised to continue no prophylaxis (on-demand treatment with their Standard of Care products) or Mim8 once-weekly or once-monthly prophylaxis in agreement with investigators in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) in agreement with the investigator, either weekly or monthly Mim8 prophylaxis regimen.	Drug: NNC0365-3769 (Mim8); Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.
Experimental: no PPX - Mim8 PPXQW - Mim8 PPXQW; Participants not receiving prophylaxis will not enter the run-in period. In arm 2a, participants will be randomised to Mim8 once-weekly prophylaxis in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-weekly Mim8 prophylaxis regimen.	Drug: NNC0365-3769 (Mim8); Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.
Experimental: no PPX - Mim8 PPXQM - Mim8 PPXQM; Participants not receiving prophylaxis will not enter the run-in period. In arm 2b, participants will be randomised to Mim8 once-monthly prophylaxis in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-monthy Mim8 prophylaxis regimen.	Drug: NNC0365-3769 (Mim8); Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.
Experimental: PPX - Mim8 PPXQW; Participants on coagulation factor prophylaxis prior to enrolment will preferably continue the same product type and dosing frequency in the run-in period for at least 26 weeks before they can be randomised into the main part of the study. These participants will only be allowed to receive coagulation factor prophylaxis. In arm 3, participants will be randomised to once-weekly Mim8 prophylaxis regimen in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-weekly Mim8 prophylaxis regimen.	Drug: NNC0365-3769 (Mim8); Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.
Experimental: PPX- Mim8 PPXQM; Participants on coagulation factor prophylaxis prior to enrolment will preferably continue the same product type and dosing frequency in the run-in period for at least 26 weeks before they can be randomised into the main part of the study. These participants will only be allowed to receive coagulation factor prophylaxis. In arm 4, participants will be randomised to once-monthly Mim8 prophylaxis regimen in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) on once-monthly Mim8 prophylaxis regimen.	Drug: NNC0365-3769 (Mim8); Mim8 will be injected into a skinfold on the stomach with a thin needle either once a week or once a month.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of treated bleeds	Count	No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (week 26)
Number of treated bleeds	Count	Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (week 26)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of injection site reactions	Count	All participants receiving Mim8 (Arms 2a, 2b, 3 and 4): From randomisation (week 0) to end of main (week 26)
Occurrence of anti-Mim8 antibodies	Count	All participants receiving Mim8 (Arms 2a, 2b, 3 and 4): From randomisation (week 0) to end of extension (week 52)
Number of treated spontaneous bleeds	Count	No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)
Number of treated joint bleeds	Count	No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)
Number of treated traumatic bleeds	Count	No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)
Number of target joint bleeds	Count	No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)
Consumption of factor product per bleed treatment (number of injections)	Count	No prophylaxis treatment (Arms 1, 2a and 2b): From randomisation (week 0) to end of main (Week 26) Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from week 0 to end of main (week 26)
Change in physical function domain of PEDS-QL (pediatric quality of life inventory)	Score points Minimum score per question (best) = 0 Maximum score per question (worst) = 4 Total score for 13 questions: 0 (best) to 92 (worst)	All participants (Arms 1, 2a, 2b, 3 and 4): From randomisation (week 0) to the end of the main part (week 26)
Change in patient's treatment burden using the Hemo-TEM (haemophilia treatment experience measure)	Score points Ranges from 0 (best) - 4 (worst) representing answers ranging: 'Not at all difficult' - 'Extremely difficult' 'Never' - 'always' 'Not at all bothered' - 'Extremely bothered' 'Not at all interfering' - 'Extremely interfering' 'Not at all burdened' - 'Extremely burdened'	All participants (Arms 1, 2a, 2b, 3 and 4): From randomisation (week 0) to the end of the main part (week 26)
Change in patient's joint pain score using Joint Pain Rating Scale	Score points ranges from 0 = 'not at all' (best) to 4 = 'extremely' (worst)	All participants (Arms 1, 2a, 2b, 3 and 4): From randomisation (week 0) to the end of the main part (week 26)

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S
• Investigators: Study Director: Clinical Transparency (dept. 2834), Novo Nordisk A/S

Publications and helpful links

General Publications

• Ong Clausen WH, Latendorf T, Stehr R, Ezban M, Lund J. Comparative ex vivo analysis of Mim8 in thrombin generation assays triggered by activated factor XI or tissue factor. Res Pract Thromb Haemost. 2025 Oct 10;9(7):103209. doi: 10.1016/j.rpth.2025.103209. eCollection 2025 Oct.

Study record dates

Study Major Dates

• Study Start (Actual): December 2, 2021
• Primary Completion (Actual): March 11, 2024
• Study Completion (Actual): December 17, 2024

Study Registration Dates

• First Submitted: September 13, 2021
• First Submitted That Met QC Criteria: September 13, 2021
• First Posted (Actual): September 22, 2021

Study Record Updates

• Last Update Posted (Actual): December 8, 2025
• Last Update Submitted That Met QC Criteria: December 5, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia A
• Other Study ID Numbers: NN7769-4514; U1111-1249-4378 (Other Identifier: World Health Organization (WHO)); 2020-001048-24 (Registry Identifier: European Medicines Agency (EudraCT)); jRCT2031210643 (Registry Identifier: JAPIC)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No