A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors

Safety, Efficacy and Exposure of Subcutaneously Administered NNC0365-3769 (Mim8) Prophylaxis in Children With Haemophilia A With or Without FVIII Inhibitors

This study is looking at how Mim8 works compared to other medicines in children with haemophilia A, who either have inhibitors or do not have inhibitors.

Mim8 is a new medicine that will be used for prevention of bleeds. Mim8 will be injected with a thin needle into the skin. The study will last for about 54-98 weeks, from screening to follow-up visit, In case the participant experiences bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor.

Study Overview

• Status: Completed
• Conditions: Haemophilia A With or Without Inhibitors
• Intervention / Treatment: Drug: Mim8

• Study Type: Interventional
• Enrollment (Actual): 70
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Ontario
    • Hamilton, Ontario, Canada, L8N 3Z5
      • McMaster Children's Hospital
    • Toronto, Ontario, Canada, M5G 1X8
      • The Hospital for Sick Children
• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100045
      • Beijing Children's Hospital, Capital Medical University
    • Beijing, Beijing Municipality, China, 100045
      • Beijing Children's Hospital，Capital Medical University
  • Guangdong
    • Guangzhou, Guangdong, China, 510515
      • Nanfang Hospital, Southern Medical University-Haematology
  • Sichuan
    • Chengdu, Sichuan, China, 610000
      • Chengdu Women's and Children's Central Hospital
  • Tianjin Municipality
    • Tianjin, Tianjin Municipality, China, 300020
      • Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310052
      • Children's Hospital, Zhejiang University School of Medicine
• Germany
  • Bonn, Germany, 53127
    • Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
• India
  • Gujarat
    • Surat, Gujarat, India, 395002
      • Nirmal Hospital Pvt. Ltd.
  • Maharashtra
    • Mumbai, Maharashtra, India, 400012
      • Seth GS Medical College & KEM Hospital
    • Pune, Maharashtra, India, 411004
      • Sahyadri Super Speciality Hospital
  • Rajasthan
    • Jaipur, Rajasthan, India, 302004
      • J K Lon Hospital
  • Uttar Pradesh
    • Noida, Uttar Pradesh, India, 201303
      • Post Graduate Institute of Child Health
• Israel
  • Tel Litwinsky, Israel, 52621
    • Sheba MC - The Israeli National Hemophilia Center
• Italy
  • Rome, Italy, 00165
    • Ospedale Pediatrico Bambino Ges
  • Torino, Italy, 10126
    • A.O.U. Città della Salute e della Scienza di Torino-Ospedale
• Japan
  • Gunma, Japan, 373-8585
    • Ota Memorial Hospital_Pediatrics
  • Hokkaido, Japan, 004-0041
    • Sapporo Tokushukai Hospital_Pediatrics
  • Saitama, Japan, 330-8777
    • Saitama Children's Med Centre_Hematology-Oncology
  • Tokyo, Japan, 167-0035
    • Ogikubo Hospital_Pediatries & Blood
• Lithuania
  • Vilnius, Lithuania, 08406
    • Children Oncohaematology department Children's Hospital,
• Netherlands
  • Amsterdam, Netherlands, 1105 AZ
    • Academisch Medisch Centrum
  • Utrecht, Netherlands, 3584 CX
    • UMC Utrecht, Van Creveldkliniek
• Poland
  • Lodz, Poland, 91-738
    • CSK UM Uniwersyteckie Centrum Pediatrii im. M. Konopnickiej
  • Lublin, Poland, 20-093
    • Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
  • Lower Silesian Voivodeship
    • Wroclaw, Lower Silesian Voivodeship, Poland, 50-556
      • Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego
• Portugal
  • Lisbon, Portugal, 1169-045
    • Unidade Local de Saúde São José EPE- Hospital D. Estefânia
  • Porto, Portugal, 4200-319
    • ULS São João, E.P.E.
• Russia
  • Kemerovo, Russia, 650066
    • SAHI Kuzbass Hospital(former Regional clinical hospital)
  • Krasnodar, Russia, 350007
    • Children Regional Clinical Hospital
  • Moscow, Russia, 119049
    • Morozovskaya municipal children hospital
  • Petrozavodsk, Russia, 185019
    • Republican Hospital n.a. V. A. Baranov
• South Africa
  • Gauteng
    • 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
• Spain
  • Esplugues Llobregat, Spain, 08950
    • Hospital Sant Joan de Déu
  • Madrid, Spain, 28046
    • Hospital Universitario La Paz
• Switzerland
  • Bern, Switzerland, 3010
    • Universitätsklinik für Kinderheilkunde
  • Lucerne, Switzerland, 6000
    • Pädiatrische Onkologie-Hämatologie
• Taiwan
  • Taipei, Taiwan, 100
    • National Taiwan University Children's Hospital
• United Kingdom
  • Cardiff, United Kingdom, CF14 4XW
    • Arthur Bloom Haemophilia Centre
  • London, United Kingdom, SE1 7EH
    • St Thomas' Hospital - Haemostasis and Thrombosis Centre
• United States
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles - Endocrinology
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Univ of Colorado Sch of Med
  • Florida
    • Tampa, Florida, United States, 33607
      • St Joseph's Hospital Foundation
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Children's Healthcare Atlanta
    • Savannah, Georgia, United States, 31404
      • Children's Hospital at Memorial Health
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa_Iowa City
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • Univ Hosp Cleveland Med Ctr
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033-2360
      • Penn State MS Hershey Med Ctr
    • Philadelphia, Pennsylvania, United States, 19134
      • St Christopher Hosp for Child

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 11 years (Child)
• 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 and female participants with the diagnosis of congenital haemophilia A of any severity based on medical records.
3. Aged 1-11 years (both inclusive) at the time of signing informed consent.

4. For previously treated participants :

   1. Participant has been prescribed treatment with FVIII concentrate or bypassing agent in the last 26 weeks prior to screening.
   2. Participants with endogenous FVIII activity greater than or equal to 1%, based on medical records, must have at least 1 treated bleed during the previous 26 weeks before screening for which factor VIII concentrate or bypassing agent has been prescribed (no requirements for participants with FVIII activity below 1%).

5. For previously untreated participants:

   a. Diagnosis of severe haemophilia A (endogenous FVIII activity below 1%) based on medical records.

6. Child and parent/caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires.( For China mainland; assessed at the investigator's discretion unless otherwise stated.)

Exclusion criteria:

1. Known or suspected hypersensitivity to trial product or related products.(For China mainland; assessed at the investigator's discretion unless otherwise stated.)
2. Previous participation in this study. Participation is defined as signed informed consent.
3. Participation (i.e., signed informed consent) in any interventional clinical study with receipt of last dose within 6 months (or 5 half-lives of the investigational medicinal product, whichever is shorter) before planned randomisation.
4. 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.
5. Known congenital or acquired coagulation disorders other than haemophilia A.
6. Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of bleeding or thrombosis, as evaluated by the investigator.(For China mainland; assessed at the investigator's discretion unless otherwise stated.)
7. 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.(For China mainland; assessed at the investigator's discretion unless otherwise stated.)
8. Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate understanding and cooperation.(For China mainland; assessed at the investigator's discretion unless otherwise stated.)
9. Lack of adequate parental/caregiver support to enter accurately and timely information regarding treatment and bleeding episodes into an (electronic) diary.(For China mainland; assessed at the investigator's discretion unless otherwise stated.)
10. 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.
11. Major surgery planned to take place after screening.(For China mainland; assessed at the investigator's discretion unless otherwise stated.)
12. Immune tolerance induction planned to take place after treatment initiation.(For China mainland; assessed at the investigator's discretion unless otherwise stated.)
13. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) greater than 3 times the upper limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal measured at screening.
14. Serum creatinine above 1.5 x upper limit of normal (ULN), measured at screening.
15. Pregnancy (female participants).(Will be assessed at investigator's discretion, according to suspicion of pregnancy.)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Mim8; 52-week treatment period with a part 1 and part 2, where all participants receive Mim8 prophylaxis	Drug: Mim8; For treatment part 1, all participants will start on once-weekly treatment and continue on this regimen until week 26. For treatment part 2, starting at week 26, all participants will be offered the choice to remain on once-weekly or switch to once-monthly dosing. Mim8 will be injected with a thin needle into the skin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of treatment emergent adverse events	Count of events	From treatment initiation to follow up visit (week 0 to week 72)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of treated bleeds	Count of bleeds	From treatment initiation to end of treatment (week 0 to week 52)
Number of treated spontaneous bleeds	Count of bleeds	From treatment initiation to end of treatment (week 0 to week 52)
Number of treated traumatic bleeds	Count of bleeds	From treatment initiation to end of treatment (week 0 to week 52)
Number of treated joint bleeds	Count of bleeds	From treatment initiation to end of treatment (week 0 to week 52)
Number of treated target joint bleeds	Count of bleeds	From treatment initiation to end of treatment (week 0 to week 52)
Number of injection site reactions	Count of reactions	From treatment initiation to end of treatment (week 0 to week 52)
Consumption of factor product per bleed treatment (number of injections)	Count of injections	From run-in initiation to end of treatment (week -26 to week 52)
Occurrence of anti-Mim8 antibodies	Count of participants	From treatment initiation to end of treatment (week 0 to week 52)
Mim8 plasma concentration	µg/mL	From treatment initiation to end of treatment (week 0 to week 52)
Change in physical function domain of PEDS QL (Paediatric Quality of Life inventory) Generic Core Scales	Score on a scale 0-100 (applies for scale scores and total score). A higher score indicates a better health-related quality of life	From treatment initiation to end of treatment (week 0 to week 52)
Treatment preference for Mim8 versus previous treatment using Caregiver H PPQ (Caregiver Haemophilia Patient Preference )	Percentage of participants	Once during treatment (week 26)
Change in participants' treatment burden using the Hemo TEM (Haemophilia treatment experience measure)	Score on a scale 0-100 (applies for scale scores and total score). A lower score indicates a lower treatment burden.	From treatment initiation to end of treatment (week 0 to week 52)

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): April 4, 2022
• Primary Completion (Actual): November 13, 2024
• Study Completion (Actual): November 13, 2024

Study Registration Dates

• First Submitted: March 23, 2022
• First Submitted That Met QC Criteria: March 23, 2022
• First Posted (Actual): April 1, 2022

Study Record Updates

• Last Update Posted (Estimated): November 13, 2025
• Last Update Submitted That Met QC Criteria: November 11, 2025
• Last Verified: November 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; Hemophilia A
• Other Study ID Numbers: NN7769-4516; U1111-1255-1540 (Other Identifier: World Health Organization (WHO); 2020-003467-26 (EudraCT Number); jRCT2031220670 (Registry Identifier: jRCT)

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