decompensated liver cirrhosis MedDRA version: 20.1 Level: LLT Classification code 10064704 Term: Decompensated cirrhosis System Organ Class: 100000004871
Conditions
Interventions
Product Name: N-003 (ambrisentan) 5 mg/ml solution for oral or subcutaneous administration
Product Code: N-003
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: AMBRISENTAN
CAS
Sponsors
Noorik Biopharmaceuticals AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Signed informed consent including data protection declaration prior to study participation 2.Subjects with confirmed cirrhosis (by biopsy, ultrasound, and/or laboratory examinations) 3.Ascites Grade II or Grade III at screening 4.Currently treated with at least one diuretic or the subject is considered intolerant to diuretics in the investigator’s opinion Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 24 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 24
Exclusion criteria
Exclusion criteria: 1.Age 1.5mg/dL (>132 µmol/L) b.Serum Na+ 3.0 3.Women of childbearing potential with no effective contraceptive method (women of childbearing potential [pre-menopausal, not surgically sterile for at least 3 months prior to the time of screening] must have a confirmed negative serum ß-hCG pregnancy test prior to enrolment and at Baseline Visit. They must use an effective contraceptive method throughout the study, and agree to repeat serum ß-hCG pregnancy tests at designated visits) 4.Pregnancy or lactation 5.Systolic blood pressure 90 beats per minute at rest, or Long QT syndrome or QTc > 450 ms 2.Significant left ventricular outflow tract obstructions (e.g., severe valvular aortic stenosis, obstructive cardiomyopathy), severe mitral stenosis, restrictive amyloid myocardiopathy, acute myocarditis 3.Severe aortic insufficiency or severe mitral r
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to determine whether N-003 reduces portal pressure in patients with decompensated cirrhosis and a history of recurrent ascites.;Secondary Objective: • the effect of N-003 on renal function, as determined by 24-hour Urinary Sodium Volume (UNaV) •the effect of N-003 on weight •the effect of N-003 on abdominal girth •the effect of N-003 on liver disease severity scores (MELD and Child-Pugh scores) •the effect of N-003 on cardiac and pulmonary haemodynamics •the pharmacokinetic profile of N-003 in patients with liver impairment •the effect of N-003 on plasma endothelin •to assess the safety and tolerability of N-003 in this population;Primary end point(s): The primary efficacy endpoint is defined as the mean change in Hepatic Vein Pressure Gradient (HVPG) from baseline to Day 14.;Timepoint(s) of evaluation of this end point: Visit 1 - day 1 & visit 2 - day 14 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary efficacy endpoints include: •change in 24-hour Urinary Sodium Volume (UNaV) from baseline to Visit 1 – Day 1 •change in weight from baseline to each study visit •change in abdominal girth from baseline to each study visit •change in MELD score from baseline to each study visit •change in Child-Pugh score from baseline to each study visit •change in cardiac and pulmonary haemodynamic variables from baseline to 90 minutes post-drug administration, Day 14 (trough) and 90 minutes after last dose (Day 14);Timepoint(s) of evaluation of this end point: at all visits starting from baseline | — |
Countries
Austria, Spain
Contacts
Public ContactCEO
Noorik Biopharmaceuticals AG
Outcome results
None listed