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New Once-Weekly shot could prevent bleeds in kids with hemophilia

NCT ID NCT05306418

Disease control Sponsor: Novo Nordisk A/S Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a new medicine called Mim8, given as a shot under the skin, to prevent bleeding episodes in children aged 1-11 with hemophilia A (with or without inhibitors). The trial involved 70 participants and compared once-weekly and once-monthly dosing over about 54-98 weeks. The goal was to see if Mim8 is safe and effective at reducing bleeds compared to other treatments.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Mim8 (a medicine injected under the skin to prevent bleeding episodes)
What this could lead to
If successful, Mim8 could offer a convenient, less frequent injection option to prevent bleeds in children with hemophilia A, reducing the burden of daily or frequent infusions.
What could go wrong
This is a relatively small Phase 3 trial (70 children) and results may not apply to all patients. Side effects or lack of efficacy compared to existing treatments remain possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

70 people

The number who actually took part.

Started

Apr 2022

Finished

Nov 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year to 11 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.)

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Conditions

The condition(s) this trial relates to.

hemophilia A

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Haemophilia A with or without inhibitors

Contacts and locations

Locations

  • A.O.U. Città della Salute e della Scienza di Torino-Ospedale

    Torino, 10126, Italy

  • Academisch Medisch Centrum

    Amsterdam, 1105 AZ, Netherlands

  • Arthur Bloom Haemophilia Centre

    Cardiff, CF14 4XW, United Kingdom

  • Beijing Children's Hospital, Capital Medical University

    Beijing, Beijing Municipality, 100045, China

  • Beijing Children's Hospital，Capital Medical University

    Beijing, Beijing Municipality, 100045, China

  • CSK UM Uniwersyteckie Centrum Pediatrii im. M. Konopnickiej

    Lodz, 91-738, Poland

  • Charlotte Maxeke Johannesburg Academic Hospital

    Parktown, Johannesburg, Gauteng, 2193, South Africa

  • Chengdu Women's and Children's Central Hospital

    Chengdu, Sichuan, 610000, China

  • Children Oncohaematology department Children's Hospital,

    Vilnius, 08406, Lithuania

  • Children Regional Clinical Hospital

    Krasnodar, 350007, Russia

  • Children's Healthcare Atlanta

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Los Angeles - Endocrinology

    Los Angeles, California, 90027, United States

  • Children's Hospital at Memorial Health

    Savannah, Georgia, 31404, United States

  • Children's Hospital, Zhejiang University School of Medicine

    Hangzhou, Zhejiang, 310052, China

  • Daejeon Eulji Medical Center, Eulji University

    Daejeon, 35233, South Korea

  • Hospital Sant Joan de Déu

    Esplugues Llobregat, 08950, Spain

  • Hospital Universitario La Paz

    Madrid, 28046, Spain

  • Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology

    Tianjin, Tianjin Municipality, 300020, China

  • J K Lon Hospital

    Jaipur, Rajasthan, 302004, India

  • McMaster Children's Hospital

    Hamilton, Ontario, L8N 3Z5, Canada

  • Morozovskaya municipal children hospital

    Moscow, 119049, Russia

  • Nanfang Hospital, Southern Medical University-Haematology

    Guangzhou, Guangdong, 510515, China

  • National Taiwan University Children's Hospital

    Taipei, 100, Taiwan

  • Nirmal Hospital Pvt. Ltd.

    Surat, Gujarat, 395002, India

  • Ogikubo Hospital_Pediatries & Blood

    Tokyo, 167-0035, Japan

  • Ospedale Pediatrico Bambino Ges

    Rome, 00165, Italy

  • Ota Memorial Hospital_Pediatrics

    Gunma, 373-8585, Japan

  • Penn State MS Hershey Med Ctr

    Hershey, Pennsylvania, 17033-2360, United States

  • Post Graduate Institute of Child Health

    Noida, Uttar Pradesh, 201303, India

  • Pädiatrische Onkologie-Hämatologie

    Lucerne, 6000, Switzerland

  • Republican Hospital n.a. V. A. Baranov

    Petrozavodsk, 185019, Russia

  • SAHI Kuzbass Hospital(former Regional clinical hospital)

    Kemerovo, 650066, Russia

  • Sahyadri Super Speciality Hospital

    Pune, Maharashtra, 411004, India

  • Saitama Children's Med Centre_Hematology-Oncology

    Saitama, 330-8777, Japan

  • Sapporo Tokushukai Hospital_Pediatrics

    Hokkaido, 004-0041, Japan

  • Seth GS Medical College & KEM Hospital

    Mumbai, Maharashtra, 400012, India

  • Severance Hospital, Yonsei University Health System

    Seoul, 03722, South Korea

  • Sheba MC - The Israeli National Hemophilia Center

    Tel Litwinsky, 52621, Israel

  • St Christopher Hosp for Child

    Philadelphia, Pennsylvania, 19134, United States

  • St Joseph's Hospital Foundation

    Tampa, Florida, 33607, United States

  • St Thomas' Hospital - Haemostasis and Thrombosis Centre

    London, SE1 7EH, United Kingdom

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • ULS São João, E.P.E.

    Porto, 4200-319, Portugal

  • UMC Utrecht, Van Creveldkliniek

    Utrecht, 3584 CX, Netherlands

  • Unidade Local de Saúde São José EPE- Hospital D. Estefânia

    Lisbon, 1169-045, Portugal

  • Univ Hosp Cleveland Med Ctr

    Cleveland, Ohio, 44106, United States

  • Univ of Colorado Sch of Med

    Aurora, Colorado, 80045, United States

  • University of Iowa_Iowa City

    Iowa City, Iowa, 52242, United States

  • Universitätsklinik für Kinderheilkunde

    Bern, 3010, Switzerland

  • Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie

    Bonn, 53127, Germany

  • Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa

    Lublin, 20-093, Poland

  • Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego

    Wroclaw, Lower Silesian Voivodeship, 50-556, Poland

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