Open-label safety study in adults and adolescents with haemophilia A with and without FVIII inhibitors switching directly from emicizumab prophylaxis to NNC0365-3769 (Mim8) prophylaxis(NN7769-4728)

JRCT ID: jRCT2051230112

Registered date:12/10/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: The participants will be male and female adults and adolescents with haemophilia A who have received
• Date of first enrollment: 01/11/2023
• Target sample size: 60
• Countries of recruitment: Austria,Japan,Belgium,Japan,Canada,Japan,France,Japan,Germany,Japan,Italy,Japan,South Africa,Japan,South Korea,Japan,Spain,Japan,UK,Japan,USA,Japan
• Study type: Interventional

Intervention(s)

Mim8 will be administered s.c. using a prefilled fixed dose DV3407-C1 pen-injector. Mim8 dosing
frequencies are QW, Q2W, or QM and will be chosen by the participant in consultation with the
investigator irrespective of prior emicizumab regimen. For monthly Mim8 dosing (QM),
participants should receive one prophylactic dose of Mim8 each month.

Outcome(s)

• Primary Outcome: The safety of Mim8 prophylaxis during emicizumab washout in adults and adolescents with haemophilia A with or without FVIII inhibitors who have switched directly from prophylaxis with emicizumab

Secondary Outcome

-The device handling experience for administration of Mim8 using the DV3407-C1 pen-injector and to assess participants' preference of injection system
-The treatment burden with Mim8 prophylaxis in adults and adolescents with haemophilia A with or without FVIII inhibitors who have switched directly from prophylaxis with emicizumab

Key inclusion & exclusion criteria

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

Include criteria

-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.
-Male or female with diagnosis of congenital haemophilia A of any severity based on medical records.
-Age 12 years or above at the time of signing the informed consent.
-Patients treated with emicizumab QW, Q2W, or Q4W according to the label for at least 8 weeks prior to screening.
-Patients choosing to discontinue emicizumab treatment and switch to Mim8 QW, Q2W, or QM treatment for 26 weeks from start of treatment (Visit 2).
-Participant and/or caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of an electronic diary and patient-reported outcomes (PRO) questionnaires.

Exclude criteria

-Participation (i.e., signed informed consent) in any interventional, clinical study, with the exception of emicizumab, with receipt of the last dose within 8 weeks (or 5 half-lives of the investigational medicinal product (IMP), whichever is longer) before screening.
-Any disorder, which in the investigators opinion might jeopardise the participants compliance with the protocol or safety, including ongoing AEs associated with emicizumab.
-Previous participation in this study. Participation is defined as signed informed consent.
-Known congenital or acquired coagulation disorders other than haemophilia A.
-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.

-Neutralising antibodies towards emicizumab have been detected or, for patients adherent to emicizumab therapy, are suspected based on clinical and laboratory assessments.
-Receipt of FVIII gene therapy at any time.
-Ongoing or planned immune tolerance induction therapy.
-Minor or major surgery planned to take place after screening and during the 26-week treatment period.
-Known or suspected hypersensitivity to study intervention, related products, any constituents of the product or to other monoclonal antibodies.
-Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (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.
-Female who is pregnant, breast-feeding or intends to become pregnant or is of childbearing potential and not using highly effective contraceptive method.
-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: Shimizu Yu
• Secondary ID(s): NCT05878938