A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A

Recruiting now · Phase 3

Conditions studied: Haemophilia A, Haemophilia A With Inhibitors

In brief

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.

Key facts

Study ID

NCT05685238

Run by

Novo Nordisk A/S

People needed

451

Starts

2023-02-13

Expected to finish

2028-06-30

Last updated by the study team

2026-06-30

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

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:
• Inclusion criteria
• 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

Where it is running

• Christian Medical College and Hospital — Ludhiana, Punjab, India (enrolling)
• Ota Memorial Hospital_Pediatrics — Gunma, Japan (enrolling)
• Jinan Central Hospital — Jinan, China (enrolling)
• Sahyadri Clinical Research And Development Center — Pune, Maharashtra, India (enrolling)
• Azienda Ospedaliera di Rilievo Nazionale Santobono Pausilipon — Naples, Italy (enrolling)
• Ospedale Pediatrico Bambino Ges — Roma, Italy (enrolling)
• Beijing Children's Hospital，Capital Medical University — Beijing, Beijing Municipality, China (enrolling)
• Institute of hematology and Blood Diseases Hospital, Tianjin-Hematology — Tianjin, Tianjin Municipality, China (enrolling)
• Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain — Berlin, Germany (enrolling)
• Nirmal Hospital Pvt. Ltd. — Surat, Gujarat, India (enrolling)
• Sheba MC - The Israeli National Hemophilia Center — Tel Litwinsky, Israel (enrolling)
• Penn State MS Hershey Med Ctr — Hershey, Pennsylvania, United States (enrolling)
• St Christopher Hosp for Child — Philadelphia, Pennsylvania, United States (enrolling)
• Vanderbilt U Med Ctr_Nashville — Nashville, Tennessee, United States (enrolling)
• UC Denver Hemoph & Thrombo Ctr — Aurora, Colorado, United States (enrolling)
• Children's Hospital Los Angeles - Endocrinology — Los Angeles, California, United States (enrolling)
• University of Iowa_Iowa City — Iowa City, Iowa, United States (enrolling)
• Tongji Hospital, Tongji Medical College of HUST — Wuhan, Hubei, China (enrolling)
• Central Michigan University — Detroit, Michigan, United States (enrolling)
• Children's Healthcare Atlanta — Atlanta, Georgia, United States (enrolling)
• Univ Hosp Cleveland Med Ctr — Cleveland, Ohio, United States (enrolling)
• Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie — Bonn, Germany (enrolling)
• Dayton Children Hemostati Ctr — Dayton, Ohio, United States (enrolling)
• Univ of Miami/SCCC — Miami, Florida, United States (enrolling)
• Nara Medical University Hospital_Pediatrics — Nara, Japan (enrolling)

Full record on ClinicalTrials.gov

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