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

Phase 3

Completed

• Conditions: Haemophilia A With or Without Inhibitors
• Interventions: Drug: Mim8

• Registration Number: NCT05306418
• Lead Sponsor: Novo Nordisk A/S

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-03-23
• Study First Submitted QC: 2022-03-23
• Study First Posted: 2022-04-01
• Study Start: 2022-04-04
• Primary Completion: 2024-11-13
• Study Completion: 2024-11-13
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-09
• Last Update Posted: 2025-04-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 70

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.)

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Number of treatment emergent adverse events	From treatment initiation to follow up visit (week 0 to week 72)	Count of events

Secondary Outcome Measures

Name	Time	Method
Change in physical function domain of PEDS QL (Paediatric Quality of Life inventory) Generic Core Scales	From treatment initiation to end of treatment (week 0 to week 52)	Score on a scale 0-100 (applies for scale scores and total score). A higher score indicates a better health-related quality of life
Number of treated bleeds	From treatment initiation to end of treatment (week 0 to week 52)	Count of bleeds
Number of treated spontaneous bleeds	From treatment initiation to end of treatment (week 0 to week 52)	Count of bleeds
Number of treated joint bleeds	From treatment initiation to end of treatment (week 0 to week 52)	Count of bleeds
Number of treated traumatic bleeds	From treatment initiation to end of treatment (week 0 to week 52)	Count of bleeds
Number of treated target joint bleeds	From treatment initiation to end of treatment (week 0 to week 52)	Count of bleeds
Consumption of factor product per bleed treatment (number of injections)	From run-in initiation to end of treatment (week -26 to week 52)	Count of injections
Mim8 plasma concentration	From treatment initiation to end of treatment (week 0 to week 52)	µg/mL
Number of injection site reactions	From treatment initiation to end of treatment (week 0 to week 52)	Count of reactions
Occurrence of anti-Mim8 antibodies	From treatment initiation to end of treatment (week 0 to week 52)	Count of participants
Change in participants' treatment burden using the Hemo TEM (Haemophilia treatment experience measure)	From treatment initiation to end of treatment (week 0 to week 52)	Score on a scale 0-100 (applies for scale scores and total score). A lower score indicates a lower treatment burden.
Treatment preference for Mim8 versus previous treatment using Caregiver H PPQ (Caregiver Haemophilia Patient Preference )	Once during treatment (week 26)	Percentage of participants

Trial Locations

Locations (29)

Centro Hospitalar Lisboa Central - Hospital Dona Estefânia; 🇵🇹 Lisboa, Portugal

Univ Hosp Cleveland Med Ctr; 🇺🇸 Cleveland, Ohio, United States

Seth GS Medical College & KEM Hospital; 🇮🇳 Mumbai, Maharashtra, India

Sahyadri Super Speciality Hospital; 🇮🇳 Pune, Maharashtra, India

J K Lon Hospital; 🇮🇳 Jaipur, Rajasthan, India

University of Iowa_Iowa City; 🇺🇸 Iowa City, Iowa, United States

Post Graduate Institute of Child Health; 🇮🇳 Noida, Uttar Pradesh, India

Sheba MC The Israeli National Hemophilia Center; 🇮🇱 Tel-Hashomer, Israel

Ospedale Pediatrico Bambino Ges; 🇮🇹 Rome, Italy

Ota Memorial Hospital_Pediatrics; 🇯🇵 Gunma, Japan

Daejeon Eulji Medical Center, Eulji University; 🇰🇷 Daejeon, Korea, Republic of

Severance Hospital, Yonsei University Health System; 🇰🇷 Seoul, Korea, Republic of

Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa; 🇵🇱 Lublin, Poland

Penn State MS Hershey Med Ctr; 🇺🇸 Hershey, Pennsylvania, United States

St Christopher Hosp for Child; 🇺🇸 Philadelphia, Pennsylvania, United States

The Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada

Beijing Children's Hospital，Capital Medical University; 🇨🇳 Beijing, Beijing, China

Haemotology, Nanfang Hospital, Southern Medical University; 🇨🇳 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, China

Children's Hospital, Zhejiang University school of medicine; 🇨🇳 Hangzhou, Zhejiang, China

Academisch Medisch Centrum; 🇳🇱 Amsterdam, Netherlands

Uniwersytecki Szpital Kliniczny im. J.Mikulicza-Radeckiego; 🇵🇱 Wroclaw, Dolnoslaskie, Poland

Charlotte Maxeke Johannesburg Academic Hospital; 🇿🇦 Parktown, Johannesburg, Gauteng, South Africa

Hospital Sant Joan de Déu; 🇪🇸 Esplugues Llobregat, Spain

Pädiatrische Onkologie-Hämatologie; 🇨🇭 Luzern 16, Switzerland

NTU Hospital - Children and Women Hospital; 🇨🇳 Taipei, Taiwan

Arthur Bloom Haemophilia Centre; 🇬🇧 Cardiff, United Kingdom

St Thomas' Hospital; 🇬🇧 London, United Kingdom