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Haemophilia A With or Without InhibitorsPhase 3NCT05306418

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

A Phase 3 study testing Mim8 in people with Haemophilia A With or Without Inhibitors, run by Novo Nordisk A/S at 52 sites in 19 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 1 year to 11 years
Conditions the study is for
Haemophilia A With or Without Inhibitors
What is being tested?
Mim8
Could I receive a placebo?
No, everyone receives an active treatment
How many people
70 participants
Where
52 sitesin 19 countries
Study dates
4 Apr 2022to 13 Nov 2024
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 11 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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 and female participants with the diagnosis of congenital haemophilia A of any severity based on medical records.
  • Aged 1-11 years (both inclusive) at the time of signing informed consent.
  • For previously treated participants :
  • Participant has been prescribed treatment with FVIII concentrate or bypassing agent in the last 26 weeks prior to screening.
  • 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%).
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  • For previously untreated participants:
  • Diagnosis of severe haemophilia A (endogenous FVIII activity below 1%) based on medical records.
  • 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.)

Who cannot join

  • Known or suspected hypersensitivity to trial product or related products.(For China mainland; assessed at the investigator's discretion unless otherwise stated.)
  • Previous participation in this study. Participation is defined as signed informed consent.
  • 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.
  • 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.
  • Known congenital or acquired coagulation disorders other than haemophilia A.
  • 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.)
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  • 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.)
  • 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.)
  • 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.)
  • 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.
  • Major surgery planned to take place after screening.(For China mainland; assessed at the investigator's discretion unless otherwise stated.)
  • Immune tolerance induction planned to take place after treatment initiation.(For China mainland; assessed at the investigator's discretion unless otherwise stated.)
  • 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.
  • Serum creatinine above 1.5 x upper limit of normal (ULN), measured at screening.
  • Pregnancy (female participants).(Will be assessed at investigator's discretion, according to suspicion of pregnancy.)

Trial locations and contacts

52 sites in 19 countries.

52 sites in 19 countries

United States9 sites

  • Children's Healthcare Atlanta

    Atlanta, Georgia, 30322

  • Univ of Colorado Sch of Med

    Aurora, Colorado, 80045

  • Univ Hosp Cleveland Med Ctr

    Cleveland, Ohio, 44106

  • Penn State MS Hershey Med Ctr

    Hershey, Pennsylvania, 17033-2360

  • University of Iowa_Iowa City

    Iowa City, Iowa, 52242

  • Children's Hospital Los Angeles - Endocrinology

    Los Angeles, California, 90027

  • St Christopher Hosp for Child

    Philadelphia, Pennsylvania, 19134

  • Children's Hospital at Memorial Health

    Savannah, Georgia, 31404

  • St Joseph's Hospital Foundation

    Tampa, Florida, 33607

Other trials in United States

China6 sites

  • Beijing Children's Hospital, Capital Medical University

    Beijing, Beijing Municipality, 100045

  • Beijing Children's Hospital，Capital Medical University

    Beijing, Beijing Municipality, 100045

  • Chengdu Women's and Children's Central Hospital

    Chengdu, Sichuan, 610000

  • Nanfang Hospital, Southern Medical University-Haematology

    Guangzhou, Guangdong, 510515

  • Children's Hospital, Zhejiang University School of Medicine

    Hangzhou, Zhejiang, 310052

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

    Tianjin, Tianjin Municipality, 300020

Other trials in China

India5 sites

  • J K Lon Hospital

    Jaipur, Rajasthan, 302004

  • Seth GS Medical College & KEM Hospital

    Mumbai, Maharashtra, 400012

  • Post Graduate Institute of Child Health

    Noida, Uttar Pradesh, 201303

  • Sahyadri Super Speciality Hospital

    Pune, Maharashtra, 411004

  • Nirmal Hospital Pvt. Ltd.

    Surat, Gujarat, 395002

Other trials in India

Japan4 sites

  • Ota Memorial Hospital_Pediatrics

    Gunma, 373-8585

  • Sapporo Tokushukai Hospital_Pediatrics

    Hokkaido, 004-0041

  • Saitama Children's Med Centre_Hematology-Oncology

    Saitama, 330-8777

  • Ogikubo Hospital_Pediatries & Blood

    Tokyo, 167-0035

Other trials in Japan

Russia4 sites

  • SAHI Kuzbass Hospital(former Regional clinical hospital)

    Kemerovo, 650066

  • Children Regional Clinical Hospital

    Krasnodar, 350007

  • Morozovskaya municipal children hospital

    Moscow, 119049

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

    Petrozavodsk, 185019

Other trials in Russia

Poland3 sites

  • CSK UM Uniwersyteckie Centrum Pediatrii im. M. Konopnickiej

    Lodz, 91-738

  • Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa

    Lublin, 20-093

  • Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego

    Wroclaw, Lower Silesian Voivodeship, 50-556

Other trials in Poland

Canada2 sites

  • McMaster Children's Hospital

    Hamilton, Ontario, L8N 3Z5

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

Other trials in Canada

Italy2 sites

  • Ospedale Pediatrico Bambino Ges

    Rome, 00165

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

    Torino, 10126

Other trials in Italy

Netherlands2 sites

  • Academisch Medisch Centrum

    Amsterdam, 1105 AZ

  • UMC Utrecht, Van Creveldkliniek

    Utrecht, 3584 CX

Other trials in Netherlands

Portugal2 sites

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

    Lisbon, 1169-045

  • ULS São João, E.P.E.

    Porto, 4200-319

Other trials in Portugal

South Korea2 sites

  • Daejeon Eulji Medical Center, Eulji University

    Daejeon, 35233

  • Severance Hospital, Yonsei University Health System

    Seoul, 03722

Other trials in South Korea

Spain2 sites

  • Hospital Sant Joan de Déu

    Esplugues Llobregat, 08950

  • Hospital Universitario La Paz

    Madrid, 28046

Other trials in Spain

Switzerland2 sites

  • Universitätsklinik für Kinderheilkunde

    Bern, 3010

  • Pädiatrische Onkologie-Hämatologie

    Lucerne, 6000

Other trials in Switzerland

United Kingdom2 sites

  • Arthur Bloom Haemophilia Centre

    Cardiff, CF14 4XW

  • St Thomas' Hospital - Haemostasis and Thrombosis Centre

    London, SE1 7EH

Other trials in United Kingdom

Germany1 site

  • Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie

    Bonn, 53127

Other trials in Germany

Israel1 site

  • Sheba MC - The Israeli National Hemophilia Center

    Tel HaShomer, 52621

Other trials in Israel

Lithuania1 site

  • Children Oncohaematology department Children's Hospital,

    Vilnius, 08406

Other trials in Lithuania

South Africa1 site

  • Charlotte Maxeke Johannesburg Academic Hospital

    Parktown, Johannesburg, Gauteng, 2193

Other trials in South Africa

Taiwan1 site

  • National Taiwan University Children's Hospital

    Taipei, 100

Other trials in Taiwan

About this study

Official title: 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.

Being tested
Mim8

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Mim8Drug

    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

Study groups in NCT05306418
Study groupRoleWhat they receive
Mim8Experimental52-week treatment period with a part 1 and part 2, where all participants receive Mim8 prophylaxis
Receives: Drug: Mim8

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of treatment emergent adverse events

    Count of events

    Measured: From treatment initiation to follow up visit (week 0 to week 72)

Secondary outcomes (12)

  1. Number of treated bleeds

    Count of bleeds

    Measured: From treatment initiation to end of treatment (week 0 to week 52)

  2. Number of treated spontaneous bleeds

    Count of bleeds

    Measured: From treatment initiation to end of treatment (week 0 to week 52)

  3. Number of treated traumatic bleeds

    Count of bleeds

    Measured: From treatment initiation to end of treatment (week 0 to week 52)

  4. Number of treated joint bleeds

    Count of bleeds

    Measured: From treatment initiation to end of treatment (week 0 to week 52)

  5. Number of treated target joint bleeds

    Count of bleeds

    Measured: From treatment initiation to end of treatment (week 0 to week 52)

  6. Number of injection site reactions

    Count of reactions

    Measured: From treatment initiation to end of treatment (week 0 to week 52)

  7. Consumption of factor product per bleed treatment (number of injections)

    Count of injections

    Measured: From run-in initiation to end of treatment (week -26 to week 52)

  8. Occurrence of anti-Mim8 antibodies

    Count of participants

    Measured: From treatment initiation to end of treatment (week 0 to week 52)

  9. Mim8 plasma concentration

    µg/mL

    Measured: From treatment initiation to end of treatment (week 0 to week 52)

  10. 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

    Measured: From treatment initiation to end of treatment (week 0 to week 52)

  11. Treatment preference for Mim8 versus previous treatment using Caregiver H PPQ (Caregiver Haemophilia Patient Preference )

    Percentage of participants

    Measured: Once during treatment (week 26)

  12. 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.

    Measured: From treatment initiation to end of treatment (week 0 to week 52)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Apr 2022
  2. Study start4 Apr 2022
  3. Primary completion13 Nov 2024
  4. Study completion13 Nov 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
70 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05306418
Sponsor's protocol ID
NN7769-4516
Other IDs
U1111-1255-1540, 2020-003467-26, jRCT2031220670
Status last verified
Nov 2025
First submitted
23 Mar 2022
Last updated
13 Nov 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Publications

  • Kenet G, Berrueco R, Fijnvandraat K, Kjollerstrom P, Luciani M, Mahlangu J, Radhakrishnan N, Wu R, Paramo Florencio A, Thaung Zaw JJ, Carcao M. Denecimig (Mim8) prophylaxis in children with hemophilia A with or without factor VIII inhibitors. Res Pract Thromb Haemost. 2026 Aug 20;10(6):106914. doi: 10.1016/j.rpth.2026.106914. eCollection 2026 Aug.

    PubMed 42835127

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Mim8 in people with Haemophilia A With or Without Inhibitors, run by Novo Nordisk A/S at 52 sites in 19 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 13 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 52 sites in United States, China, India, Japan, Russia and 14 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of treatment emergent adverse events (time frame: From treatment initiation to follow up visit (week 0 to week 72)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05306418, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Nov 2025.

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