A research study investigating Mim8 in adults and adolescents with haemophilia A with or without inhibitors

Phase 1

• Conditions: Haemophilia A Haemophilia A with inhibitors; MedDRA version: 20.0Level: LLTClassification code 10018938Term: Haemophilia A (Factor VIII)System Organ Class: 100000004850; MedDRA version: 20.0Level: LLTClassification code 10053751Term: Hemophilia A with anti factor VIIISystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2020-001048-24-DK
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-01-20
• Last Update Posted: 15 November 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 244

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 with diagnosis of congenital haemophilia A of any severity based on medical records
3. Participants 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 above or equal to 30 kg
6. Applicable to participants treated with on-demand/no prophylaxis prior to enrolment: more than or equal to 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 above or equal to 1% who are on prophylactic treatment: more than or equal to 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
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 204
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

1. Previous participation in this study. Participation is defined as signed informed consent
2. Participation in any clinical study of an approved or non-approved investigational medicinal product, within 30 days (or 5 half-lives of the investigational medicinal product, whichever is greater) before screening
3. Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months (or 5 half-lives of the medicinal product, whichever is shorter) prior to planned first dose, 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 study 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 ITI therapy
9. Major surgery planned at the time of screening.
10. Known congenital or acquired coagulation disorders other than haemophilia A
11. Hepatic dysfunction defined as AST and/or ALT more than 3 times the upper limit combined with total bilirubin more than 1.5 times the upper limit measured at screening
12. Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) less than 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified