A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors
NCT ID: NCT05306418
Last Updated: 2025-11-13
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE3
70 participants
INTERVENTIONAL
2022-04-04
2024-11-13
Brief Summary
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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.
Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Mim8
52-week treatment period with a part 1 and part 2, where all participants receive Mim8 prophylaxis
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
Interventions
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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
Eligibility Criteria
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Inclusion Criteria
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
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.)
1 Year
11 Years
ALL
No
Sponsors
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Novo Nordisk A/S
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Transparency (dept. 2834)
Role: STUDY_DIRECTOR
Novo Nordisk A/S
Locations
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Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
Univ of Colorado Sch of Med
Aurora, Colorado, United States
St Joseph's Hospital Foundation
Tampa, Florida, United States
Children's Healthcare Atlanta
Atlanta, Georgia, United States
Children's Hospital at Memorial Health
Savannah, Georgia, United States
University of Iowa_Iowa City
Iowa City, Iowa, United States
Univ Hosp Cleveland Med Ctr
Cleveland, Ohio, United States
Penn State MS Hershey Med Ctr
Hershey, Pennsylvania, United States
St Christopher Hosp for Child
Philadelphia, Pennsylvania, United States
McMaster Children's Hospital
Hamilton, Ontario, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, China
Beijing Children's Hospital，Capital Medical University
Beijing, Beijing Municipality, China
Nanfang Hospital, Southern Medical University-Haematology
Guangzhou, Guangdong, China
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan, China
Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology
Tianjin, Tianjin Municipality, China
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
Bonn, , Germany
Nirmal Hospital Pvt. Ltd.
Surat, Gujarat, India
Seth GS Medical College & KEM Hospital
Mumbai, Maharashtra, India
Sahyadri Super Speciality Hospital
Pune, Maharashtra, India
J K Lon Hospital
Jaipur, Rajasthan, India
Post Graduate Institute of Child Health
Noida, Uttar Pradesh, India
Sheba MC - The Israeli National Hemophilia Center
Tel Litwinsky, , Israel
Ospedale Pediatrico Bambino Ges
Rome, , Italy
A.O.U. Città della Salute e della Scienza di Torino-Ospedale
Torino, , Italy
Ota Memorial Hospital_Pediatrics
Gunma, , Japan
Sapporo Tokushukai Hospital_Pediatrics
Hokkaido, , Japan
Saitama Children's Med Centre_Hematology-Oncology
Saitama, , Japan
Ogikubo Hospital_Pediatries & Blood
Tokyo, , Japan
Children Oncohaematology department Children's Hospital,
Vilnius, , Lithuania
Academisch Medisch Centrum
Amsterdam, , Netherlands
UMC Utrecht, Van Creveldkliniek
Utrecht, , Netherlands
Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego
Wroclaw, Lower Silesian Voivodeship, Poland
CSK UM Uniwersyteckie Centrum Pediatrii im. M. Konopnickiej
Lodz, , Poland
Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
Lublin, , Poland
Unidade Local de Saúde São José EPE- Hospital D. Estefânia
Lisbon, , Portugal
ULS São João, E.P.E.
Porto, , Portugal
SAHI Kuzbass Hospital(former Regional clinical hospital)
Kemerovo, , Russia
Children Regional Clinical Hospital
Krasnodar, , Russia
Morozovskaya municipal children hospital
Moscow, , Russia
Republican Hospital n.a. V. A. Baranov
Petrozavodsk, , Russia
Charlotte Maxeke Johannesburg Academic Hospital
Parktown, Johannesburg, Gauteng, South Africa
Daejeon Eulji Medical Center, Eulji University
Daejeon, , South Korea
Severance Hospital, Yonsei University Health System
Seoul, , South Korea
Hospital Sant Joan de Déu
Esplugues Llobregat, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Universitätsklinik für Kinderheilkunde
Bern, , Switzerland
Pädiatrische Onkologie-Hämatologie
Lucerne, , Switzerland
National Taiwan University Children's Hospital
Taipei, , Taiwan
Arthur Bloom Haemophilia Centre
Cardiff, , United Kingdom
St Thomas' Hospital - Haemostasis and Thrombosis Centre
London, , United Kingdom
Countries
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Other Identifiers
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U1111-1255-1540
Identifier Type: OTHER
Identifier Source: secondary_id
2020-003467-26
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
jRCT2031220670
Identifier Type: REGISTRY
Identifier Source: secondary_id
NN7769-4516
Identifier Type: -
Identifier Source: org_study_id