Safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple subcutaneous doses of NNC0365-3769 (Mim8) in healthy subjects and in subjects with haemophilia A with or without factor VIII inhibitors (NN7769-4513)

JRCT ID: jRCT2031200136

Registered date:05/10/2020

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: severe haemophilia A with or without FVIII inhibitors
• Date of first enrollment: 10/01/2020
• Target sample size: 82
• Countries of recruitment: Germany,Japan,Bulgaria,Japan,Austria,Japan,United States,Japan,United Kingdom,Japan,Turkey,Japan,Switzerland,Japan,Spain,Japan,South Africa,Japan,Poland,Japan,Italy,Japan
• Study type: Interventional

Intervention(s)

SAD part:Subjects will receive a single dose of Mim8 or placebo
MAD part: Subjects participates in the MAD part will be approximately 28 weeks for MAD cohorts with once-weekly dosing and 25 weeks for the MAD cohort with dosing every 4 weeks. The treatment period will consist of 12 once-weekly doses or 3 once-monthly doses.
Exploratory biomarker cohort: NA

Outcome(s)

• Primary Outcome: Number of treatment emergent adverse events

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: <= 64age old
• Gender: Male

Include criteria

SAD part: Considered to be generally healthy based on the medical history, physical examination, and the results of vital signs, electrocardiogram and clinical laboratory tests performed during the screening visit, as judged by the investigator

MAD part: Considered to be generally healthy based on the medical history, physical examination, and the results of vital signs, electrocardiogram and clinical laboratory tests performed during the screening visit, as judged by the investigator

Exploratory biomarker cohort: Diagnosis of congenital haemophilia A with FVIII activity <1% based on medical records

Exclude criteria

SAD part:
-Factor FVIII activity>= 150% at screening
-Increased risk of thrombosis, e.g. known history of personal or first-degree relative(s) with unprovoked deep vein thrombosis
-Any clinical signs or established diagnosis of venous or arterial thromboembolic disease

MAD part:
-Known congenital or acquired coagulation disorders other than haemophilia A
-Increased risk of thrombosis as evaluated by the investigator. E.g. known history of personal or first-degree relative(s) with unprovoked deep vein thrombosis with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing
-Any clinical signs or established diagnosis of venous or arterial thromboembolic disease with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing
-Advanced atherosclerotic disease (e.g. known history of ischemic heart disease, ischemic stroke) as evaluated by the investigator
-Any autoimmune disease that may increase the risk of thrombosis
-Receipt of emicizumab or drugs with similar modes of action within 5 half-lives before trial product administration
-Ongoing or planned immune tolerance induction therapy

Exploratory biomarker cohort:
-Known congenital or acquired coagulation disorders other than haemophilia A
-Increased risk of thrombosis as evaluated by the investigator. E.g. known history of personal or first-degree relative(s) with unprovoked deep vein thrombosis with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing
-Any clinical signs or established diagnosis of venous or arterial thromboembolic disease with exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing
-Advanced atherosclerotic disease (e.g. known history of ischemic heart disease, ischemic stroke) as evaluated by the investigator
-Any autoimmune disease that may increase the risk of thrombosis
-Ongoing or planned immune tolerance induction therapy

Related Information

• Primary Sponsor: Esaki Risa
• Secondary ID(s): NCT04204408,2019-000465-20