A research study of how a new medicine NNC0365-3769 (Mim8) works in the body of healthy people and patients with bleeding disorder

Phase 1

• Conditions: Healthy volunteers (Haemophilia A with or without inhibitors)Haemophilia AHaemophilia 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: EUCTR2019-000465-20-PL
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-08-14
• Last Update Posted: 15 January 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 94

Inclusion Criteria

Single ascending dose (SAD) part:
- Male, aged 18-45 years (both inclusive) at the time of signing informed consent
- 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

Multiple ascending dose (MAD) part:
- Male, aged 12-64 years (both inclusive) at the time of signing informed consent (Germany and Japan have local requirements)
- Diagnosis of congenital haemophilia A with FVIII activity <1% based on medical records

Exploratory biomarker cohort:
- Male, aged =12 years at the time of signing informed consent (Germany and Japan have local requirements)
- Diagnosis of congenital haemophilia A with FVIII activity <1% based on medical records
Are the trial subjects under 18? yes
Number of subjects for this age range: 6
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 87
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

SAD part:
- Factor VIII 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

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To investigate the safety and tolerability of subcutaneous Mim8 in healthy subjects and in subjects with severe haemophilia A with or without FVIII inhibitors;Secondary Objective: 1. To investigate the pharmacokinetics of subcutaneous Mim8 in healthy subjects and in subjects with severe haemophilia A with or without FVIII inhibitors<br>2. To investigate the pharmacodynamics of subcutaneous Mim8 in healthy subjects and in subjects with severe haemophilia A with or without FVIII inhibitors;Primary end point(s): 1. Single ascending dose (SAD) part: Number of treatment emergent adverse events<br>2. Multiple ascending dose (MAD) part: Number of treatment emergent adverse events<br>3. Extensíon to the MAD part: Number of treatment emergent adverse events;Timepoint(s) of evaluation of this end point: 1. From time of dosing (Day 1) to Week 16<br>2. From time of first dosing (Day 1) to Week 12<br>3. From Week 12 up to Week 176 (16 weeks after last dose)