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(NN7769-4514)
- Conditions
- haemophilia A with or without FVIII inhibitors
- Registration Number
- JPRN-jRCT2031210643
- Lead Sponsor
- Esaki Risa
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 272
Male or female with diagnosis of congenital haemophilia A of any severity based on medical records
-Participants has been prescribed treatment with factor VIII concentrates or bypassing agent in the last 26 weeks prior to screening
-Age above or equal to 12 years at the time of signing informed consent. Japan, South Korea and Taiwan: Please see local requirements in Appendix9_section10.9
-Body weight >=30 kg
-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
-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
-Any disorder, except for conditions associated with haemophilia A, which in the investigators opinion might jeopardise participants safety or compliance with the protocol
-Known or suspected hypersensitivity to study product(s), any constituents of the product or to related products
-Receipt of gene therapy at any given time point
-Ongoing or planned ITI therapy
-Major surgery planned at the time of screening.
-Known congenital or acquired coagulation disorders other than haemophilia A
-Hepatic dysfunction defined as AST and/or ALT >3 times the upper limit combined with total bilirubin >1.5 times the upper limit measured at screening
-Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) <=30 ml/min/1.73 m2 for serum creatinine measured at screening
-Previous or current thromboembolic disease or eventsa (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
-Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation
-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
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method umber of treated bleeds from randomisation (week 0) to end of main (Week 26)
- Secondary Outcome Measures
Name Time Method