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

NCT05306418 | Completed Phase 3 DMC FDA Drug

• 4 other identifiers: NN7769-4516U1111-1255-15402020-003467-26jRCT2031220670
• Study Type: interventional
• Target: 70
• Locations: 19 countries
• Sites: 52

Brief 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. 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.

Conditions

Haemophilia A With or Without Inhibitors

Outcome Measures

Primary Outcomes (1)

• Number of treatment emergent adverse events

  Count of events

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

Secondary Outcomes (12)

• Number of treated bleeds

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

• Number of treated spontaneous bleeds

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

• Number of treated traumatic bleeds

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

• Number of treated joint bleeds

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

• Number of treated target joint bleeds

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

Study Arms (1)

Mim8

EXPERIMENTAL

52-week treatment period with a part 1 and part 2, where all participants receive Mim8 prophylaxis

Drug: Mim8

Interventions

Mim8 DRUG

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

Mim8

Eligibility Criteria

• Age: 1 Year - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

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 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%).
• For previously untreated participants:
• a. 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.)

You may not qualify if:

• 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.)
• 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.)

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (52)

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, 90027, United States

Location

Univ of Colorado Sch of Med

Aurora, Colorado, 80045, United States

Location

St Joseph's Hospital Foundation

Tampa, Florida, 33607, United States

Location

Children's Healthcare Atlanta

Atlanta, Georgia, 30322, United States

Location

Children's Hospital at Memorial Health

Savannah, Georgia, 31404, United States

Location

University of Iowa_Iowa City

Iowa City, Iowa, 52242, United States

Location

Univ Hosp Cleveland Med Ctr

Cleveland, Ohio, 44106, United States

Location

Penn State MS Hershey Med Ctr

Hershey, Pennsylvania, 17033-2360, United States

Location

St Christopher Hosp for Child

Philadelphia, Pennsylvania, 19134, United States

Location

McMaster Children's Hospital

Hamilton, Ontario, L8N 3Z5, Canada

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Beijing Children's Hospital, Capital Medical University

Beijing, Beijing Municipality, 100045, China

Location

Beijing Children's Hospital，Capital Medical University

Beijing, Beijing Municipality, 100045, China

Location

Nanfang Hospital, Southern Medical University-Haematology

Guangzhou, Guangdong, 510515, China

Location

Chengdu Women's and Children's Central Hospital

Chengdu, Sichuan, 610000, China

Location

Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology

Tianjin, Tianjin Municipality, 300020, China

Location

Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310052, China

Location

Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie

Bonn, 53127, Germany

Location

Nirmal Hospital Pvt. Ltd.

Surat, Gujarat, 395002, India

Location

Seth GS Medical College & KEM Hospital

Mumbai, Maharashtra, 400012, India

Location

Sahyadri Super Speciality Hospital

Pune, Maharashtra, 411004, India

Location

J K Lon Hospital

Jaipur, Rajasthan, 302004, India

Location

Post Graduate Institute of Child Health

Noida, Uttar Pradesh, 201303, India

Location

Sheba MC - The Israeli National Hemophilia Center

Tel Litwinsky, 52621, Israel

Location

Ospedale Pediatrico Bambino Ges

Rome, 00165, Italy

Location

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

Torino, 10126, Italy

Location

Ota Memorial Hospital_Pediatrics

Gunma, 373-8585, Japan

Location

Sapporo Tokushukai Hospital_Pediatrics

Hokkaido, 004-0041, Japan

Location

Saitama Children's Med Centre_Hematology-Oncology

Saitama, 330-8777, Japan

Location

Ogikubo Hospital_Pediatries & Blood

Tokyo, 167-0035, Japan

Location

Children Oncohaematology department Children's Hospital,

Vilnius, 08406, Lithuania

Location

Academisch Medisch Centrum

Amsterdam, 1105 AZ, Netherlands

Location

UMC Utrecht, Van Creveldkliniek

Utrecht, 3584 CX, Netherlands

Location

Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego

Wroclaw, Lower Silesian Voivodeship, 50-556, Poland

Location

CSK UM Uniwersyteckie Centrum Pediatrii im. M. Konopnickiej

Lodz, 91-738, Poland

Location

Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa

Lublin, 20-093, Poland

Location

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

Lisbon, 1169-045, Portugal

Location

ULS São João, E.P.E.

Porto, 4200-319, Portugal

Location

SAHI Kuzbass Hospital(former Regional clinical hospital)

Kemerovo, 650066, Russia

Location

Children Regional Clinical Hospital

Krasnodar, 350007, Russia

Location

Morozovskaya municipal children hospital

Moscow, 119049, Russia

Location

Republican Hospital n.a. V. A. Baranov

Petrozavodsk, 185019, Russia

Location

Charlotte Maxeke Johannesburg Academic Hospital

Parktown, Johannesburg, Gauteng, 2193, South Africa

Location

Daejeon Eulji Medical Center, Eulji University

Daejeon, 35233, South Korea

Location

Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea

Location

Hospital Sant Joan de Déu

Esplugues Llobregat, 08950, Spain

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Universitätsklinik für Kinderheilkunde

Bern, 3010, Switzerland

Location

Pädiatrische Onkologie-Hämatologie

Lucerne, 6000, Switzerland

Location

National Taiwan University Children's Hospital

Taipei, 100, Taiwan

Location

Arthur Bloom Haemophilia Centre

Cardiff, CF14 4XW, United Kingdom

Location

St Thomas' Hospital - Haemostasis and Thrombosis Centre

London, SE1 7EH, United Kingdom

Location

Study Officials

• Clinical Transparency (dept. 2834)

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 23, 2022
• First Posted: April 1, 2022
• Study Start: April 4, 2022
• Primary Completion: November 13, 2024
• Study Completion: November 13, 2024
• Last Updated: November 13, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share

Locations

KR (2); CN (6); IL (1); TW (1); GB (2); CH (2); ES (2); IT (2); US (9); JP (4); PL (3); RU (4); CA (2); DE (1); LI (1); PT (2); IN (5); ZA (1); NL (2)