Brief Title
A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors
Official Title
A Multinational, Open-label, Randomised, Controlled Study to Investigate Efficacy and Safety of NNC0365-3769 (Mim8) in Adults and Adolescents With Haemophilia A With or Without Inhibitors
Brief Summary
This study is investigating how Mim8 works compared to other medicines in people with
haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine
that will be used for prevention of bleeding episodes. Mim8 works by replacing the function
of the missing clotting factor VIII (FVIII).
When and how often participants will receive Mim8 is dependent on their previous treatment -
but is otherwise decided by chance. Mim8 will be injected into a skinfold on the stomach with
a thin needle either once a week or once a month.
The study will last 54-124 weeks (12-29 months) depending on how long participants will be
followed in run-in before they start treatment and if they continue in the follow period or
transfer to an open label extension study. Participants will have 12-17 clinic visits.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Number of treated bleeds
Secondary Outcome
Number of injection site reactions
Condition
Haemophilia A
Intervention
NNC0365-3769 (Mim8)
Study Arms / Comparison Groups
no PPX- no PPX - Mim8 PPXQW/QM
Description: Participants not receiving prophylaxis will not enter the run-in period. In arm 1, participants will be randomised to continue no prophylaxis (on-demand treatment with their Standard of Care products) or Mim8 once-weekly or once-monthly prophylaxis in agreement with investigators in the main part of the study (26 weeks). After the main part, participants will continue in the extension part of the study (26 weeks) in agreement with the investigator, either weekly or monthly Mim8 prophylaxis regimen.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
267
Start Date
December 2, 2021
Completion Date
May 1, 2025
Primary Completion Date
May 24, 2024
Eligibility 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 or female participants with diagnosis of congenital haemophilia A of any severity
based on medical records.
3. Participant has been prescribed treatment with factor VIII concentrates or bypassing
agent in the last 26 weeks prior to screening.
4. Age above or equal to 12 years at the time of signing informed consent.
5. Body weight greater than or equal to 30 kg.
6. Applicable to participants treated with on-demand/no prophylaxis prior to enrolment:
≥5 bleeds in the last 26 weeks prior to screening visit, for which factor VIII
concentrates or bypassing agent has been prescribed.
7. Applicable to participants with FVIII activity ≥1% who are on prophylactic treatment:
≥1 bleed in the last 26 weeks prior to screening visit, for which factor VIII
concentrates or bypassing agent has been prescribed.
8. Willingness and ability to comply with scheduled visits and study procedures,
including the completion of diary and patient-reported outcomes questionnaires.
Exclusion Criteria:
1. Previous participation in this study. Participation is defined as signed informed
consent.
2. Participation (i.e., signed informed consent) in any interventional clinical study
with receipt of the last dose within 6 months (or 5 half-lives of the investigational
medicinal product, whichever is shorter) before planned randomisation.
3. 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.
4. Female who is pregnant, breast-feeding or intends to become pregnant or is of
child-bearing potential and not using a highly effective contraceptive method. Breast
feeding is allowed only during the run-in period.
5. Any disorder, except for conditions associated with haemophilia A, which in the
investigator's opinion might jeopardise participant's safety or compliance with the
protocol.
6. Known or suspected hypersensitivity to trial product(s), any constituents of the
product or to related products.
7. Receipt of gene therapy at any given time point.
8. Ongoing or planned immune tolerance induction (ITI) therapy.
9. Major surgery planned to take place after screening.
10. Known congenital or acquired coagulation disorders other than haemophilia A.
11. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine
aminotransferase (ALT) above 3 times the upper limit combined with total bilirubin
above1.5 times the upper limit measured at screening.
12. Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) below or equal
to 30 ml/min/1.73 m^2 for serum creatinine measured at screening.
13. Previous or current thromboembolic disease or events (with the exception of previous
catheter-associated thrombosis for which anti-thrombotic treatment is not currently
ongoing) or risk of thromboembolic disease, as evaluated by investigator.
14. Mental incapacity, unwillingness to cooperate, or a language barrier precluding
adequate understanding and cooperation.
15. Other conditions (e.g., autoimmune disease) or laboratory abnormality that may
increase risk of bleeding or thrombosis as evaluated by the investigator.
Gender
All
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Clinical Transparency (dept. 2834), (+1) 866-867-7178, [email protected]
Location Countries
Austria
Location Countries
Austria
Administrative Informations
NCT ID
NCT05053139
Organization ID
NN7769-4514
Secondary IDs
U1111-1249-4378
Responsible Party
Sponsor
Study Sponsor
Novo Nordisk A/S
Study Sponsor
Clinical Transparency (dept. 2834), Study Director, Novo Nordisk A/S
Verification Date
March 2023