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)

JRCT ID: jRCT2031210643

Registered date:03/03/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: haemophilia A with or without FVIII inhibitors
• Date of first enrollment: 17/02/2022
• Target sample size: 272
• Countries of recruitment: Austria,Japan,Belgium,Japan,Canada,Japan,China,Japan,Denmark,Japan,France,Japan,Germany,Japan,India,Japan,Ireland,Japan,Israel,Japan,Italy,Japan,Korea,Japan,Latvia,Japan,Lithuania,Japan,Malaysia,Japan,Mexico,Japan,Netherlands,Japan,Poland,Japan,Portugal,Japan,Romania,Japan,Russian Fed,Japan,Saudi Arabia,Japan,Serbia,Japan,Slovakia,,Japan,South Africa,Japan,Spain,Japan,Switzerland,Japan,Taiwan,Japan,Turkey,Japan,Ukraine,Japan
• Study type: Interventional

Intervention(s)

Arm 1:Study product is administered from extension period. Study product is administered weekly. The treatment period is 26 weeks.
Arm 2&3:Study product is administered weekly. The treatment period is 52 weeks.
Arm 4:Study product is administered monthly. The treatment period is 52 weeks.

Outcome(s)

• Primary Outcome: Number of treated bleeds from randomisation (week 0) to end of main (Week 26)

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

-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

Exclude criteria

-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

Related Information

• Primary Sponsor: Esaki Risa
• Secondary ID(s): NCT05053139