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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05306418
Recruitment Status : Recruiting
First Posted : April 1, 2022
Last Update Posted : September 22, 2022
Sponsor:
Information provided by (Responsible Party):
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.


Condition or disease Intervention/treatment Phase
Haemophilia A With or Without Inhibitors Drug: Mim8 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety, Efficacy and Exposure of Subcutaneously Administered NNC0365-3769 (Mim8) Prophylaxis in Children With Haemophilia A With or Without FVIII Inhibitors
Actual Study Start Date : April 4, 2022
Estimated Primary Completion Date : March 3, 2025
Estimated Study Completion Date : March 3, 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arm Intervention/treatment
Experimental: Mim8
52-week treatment period with a part 1 and part 2, where all participants receive Mim8 prophylaxis
Drug: Mim8

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





Primary Outcome Measures :
  1. Number of treatment emergent adverse events [ Time Frame: From treatment initiation to follow up visit (week 0 to week 72) ]
    Count of events


Secondary Outcome Measures :
  1. Number of treated bleeds [ Time Frame: From treatment initiation to end of treatment (week 0 to week 52) ]
    Count of bleeds

  2. Number of treated spontaneous bleeds [ Time Frame: From treatment initiation to end of treatment (week 0 to week 52) ]
    Count of bleeds

  3. Number of treated traumatic bleeds [ Time Frame: From treatment initiation to end of treatment (week 0 to week 52) ]
    Count of bleeds

  4. Number of treated joint bleeds [ Time Frame: From treatment initiation to end of treatment (week 0 to week 52) ]
    Count of bleeds

  5. Number of treated target joint bleeds [ Time Frame: From treatment initiation to end of treatment (week 0 to week 52) ]
    Count of bleeds

  6. Number of injection site reactions [ Time Frame: From treatment initiation to end of treatment (week 0 to week 52) ]
    Count of reactions

  7. Consumption of factor product per bleed treatment (number of injections) [ Time Frame: From run-in initiation to end of treatment (week -26 to week 52) ]
    Count of injections

  8. Occurrence of anti-Mim8 antibodies [ Time Frame: From treatment initiation to end of treatment (week 0 to week 52) ]
    Count of participants

  9. Mim8 plasma concentration [ Time Frame: From treatment initiation to end of treatment (week 0 to week 52) ]
    µg/mL

  10. Change in physical function domain of PEDS QL (Paediatric Quality of Life inventory) Generic Core Scales [ Time Frame: 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

  11. Treatment preference for Mim8 versus previous treatment using Caregiver H PPQ (Caregiver Haemophilia Patient Preference ) [ Time Frame: Once during treatment (week 26) ]
    Percentage of participants

  12. Change in participants' treatment burden using the Hemo TEM (Haemophilia treatment experience measure) [ Time Frame: 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   1 Year to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05306418


Contacts
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Contact: Novo Nordisk (+1) 866-867-7178 clinicaltrials@novonordisk.com

Locations
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China, Beijing
Novo Nordisk Investigational Site Not yet recruiting
Beijing, Beijing, China, 100045
Israel
Novo Nordisk Investigational Site Not yet recruiting
Tel-Hashomer, Israel, 52621
Poland
Novo Nordisk Investigational Site Not yet recruiting
Lodz, Poland, 91-738
Novo Nordisk Investigational Site Recruiting
Wroclaw, Poland, 50-556
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
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Study Director: Clinical Transparency (dept. 2834) Novo Nordisk A/S
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Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT05306418    
Other Study ID Numbers: NN7769-4516
U1111-1255-1540 ( Other Identifier: World Health Organization (WHO )
2020-003467-26 ( EudraCT Number )
First Posted: April 1, 2022    Key Record Dates
Last Update Posted: September 22, 2022
Last Verified: September 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
URL: http://novonordisk-trials.com

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn