Clinical trial NCT04321343: Study of PXL065 in Patients With Nonalcoholic Steatohepatitis (NASH)
Official title: A 36-week, Randomized, Double-blind, Placebo-controlled, Parallel Group Trial to Assess the Efficacy and Safety of PXL065 Versus Placebo in Noncirrhotic Biopsy-proven NonAlcoholic SteatoHepatitis (NASH) Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: NASH - Nonalcoholic Steatohepatitis
Lead sponsor: Poxel SA
Sponsor class: INDUSTRY
First posted: 2020-03-25
Enrollment: 117
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will assess the effect of 3 doses of PXL065 versus placebo on liver fat content in NASH patients after 36 weeks of treatment

Detailed description: The study will be performed in patients with NASH. The primary endpoint will be the assessment of the change in the percentage of liver fat content (assessed by MRI-PDFF).

Eligibility criteria: Inclusion Criteria:

* Patients have given written informed consent
* Body mass index (BMI) ≤ 50 kg/m²
* For patients with type 2 diabetes mellitus: either naive of glucose lowering drug or under stable oral glucose lowering drug
* Estimated glomerular filtration rate (eGFR) ≥ 45 mL/min/1.73m²
* Liver fat content ≥ 8% on MRI-PDFF
* Qualifying liver biopsy (NAS) ≥ 4 and fibrosis score F1, F2 or F3
* Effective contraception for women of child bearing potential

Exclusion Criteria:

* Evidence of another form of liver disease
* Evidence of liver cirrhosis
* Evidence of hepatic impairment
* Positive serologic evidence of current infectious liver disease
* History of excessive alcohol intake
* Acute cardiovascular disease within 6 months prior to Randomization
* Any disease which in the Investigator's opinion which in the Investigator's opinion would exclude the patient from the study
* Use of non-permitted concomitant medication
* Pregnancy or lactation

Primary outcomes:
measure: Relative Change From Baseline to Week 36 in the Percentage of Liver Fat Content (LFC) (Assessed by Magnetic Resonance Imaging - Proton Density Fat Fraction [MRI-PDFF])
timeFrame: Baseline and Week 36
measure: Relative Change From Baseline to Week 36 in the Percentage of LFC (Assessed by MRI-PDFF) (Wilcoxon Test Sensitivity Analysis)
timeFrame: Baseline and Week 36
Secondary outcomes:
measure: Absolute Change From Baseline to Week 36 in the Percentage of LFC (Assessed by MRI-PDFF)
timeFrame: Baseline and Week 36
measure: Percentage of Responders (Relative Reduction of at Least 30% in LFC) at Week 36
timeFrame: Baseline and Week 36
measure: Change From Baseline to Week 36 in Alanine Amino Transferase (ALT)
timeFrame: Baseline to Week 36
measure: Percentage of Responders (Normalization of ALT)
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Aspartate Amino Transferase (AST)
timeFrame: Baseline to Week 36
measure: Percentage of Responders (Normalization of AST)
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Gamma Glutamyltransferase (GGT)
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Alkaline Phosphatase (ALP)
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Pro-C3
timeFrame: Baseline and Week 36
measure: Change From Baseline to Week 36 in Enhanced Liver Fibrosis (ELF) Score
timeFrame: Baseline and Week 36
measure: Change From Baseline to Week 36 in Fibrosis-4 (Fib-4) Score
timeFrame: Baseline and Week 36
measure: Change From Baseline to Week 36 in NAFLD Fibrosis Score
timeFrame: Baseline and Week 36
measure: Improvement of at Least 1 Point in NASH CRN Fibrosis Score From Baseline to Week 36
timeFrame: Baseline and Week 36
measure: Improvement in NAS of at Least 2 Points With no Worsening in NASH CRN Fibrosis Score From Baseline to Week 36
timeFrame: Baseline and Week 36
measure: NASH Resolution With no Worsening in NASH CRN Fibrosis Score at Week 36
timeFrame: Baseline and Week 36
measure: NASH Resolution With Improvement of at Least 1 Point in NASH CRN Fibrosis Score at Week 36
timeFrame: Baseline and Week 36
measure: Change From Baseline to Week 36 in Glycated Hemoglobin (HbA1c)
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Fasting Plasma Glucose (FPG)
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Serum Insulin
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Serum C-peptide
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR)
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Quantitative Insulin Sensitivity Check Index (QUICKI)
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Adipo-IR
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Adiponectin
timeFrame: Baseline to Week 36
measure: Change From Baseline to Week 36 in Weight
timeFrame: Baseline to Week 36
Other outcomes:
Locations:
city: Chandler
country: United States
facility: Study site 11
state: Arizona
city: Glendale
country: United States
facility: Study site 12
state: Arizona
city: Tucson
country: United States
facility: Study site 13
state: Arizona
city: Tucson
country: United States
facility: Study site 21
state: Arizona
city: Chula Vista
country: United States
facility: Study site 17
state: California
city: Fresno
country: United States
facility: Study site 16
state: California
city: Huntington Park
country: United States
facility: Study site 04
state: California
city: Los Angeles
country: United States
facility: Study site 05
state: California
city: Orange
country: United States
facility: Study site 22
state: California
city: Panorama City
country: United States
facility: Study site 06
state: California
city: Santa Ana
country: United States
facility: Study site 07
state: California
city: Boca Raton
country: United States
facility: Study site 15
state: Florida
city: Fort Myers
country: United States
facility: Study site 31
state: Florida
city: Port Orange
country: United States
facility: Study site 08
state: Florida
city: Sarasota
country: United States
facility: Study site 01
state: Florida
city: West Des Moines
country: United States
facility: Study site 28
state: Iowa
city: Kansas City
country: United States
facility: Study site 18
state: Kansas
city: Flowood
country: United States
facility: Study site 24
state: Mississippi
city: Jackson
country: United States
facility: Study site 10
state: Mississippi
city: East Syracuse
country: United States
facility: Study site 27
state: New York
city: Fayetteville
country: United States
facility: Study site 14
state: North Carolina
city: Summerville
country: United States
facility: Study site 29
state: South Carolina
city: Chattanooga
country: United States
facility: Study site 25
state: Tennessee
city: Clarksville
country: United States
facility: Study site 30
state: Tennessee
city: Germantown
country: United States
facility: Study Site 02
state: Tennessee
city: Austin
country: United States
facility: Study site 19
state: Texas
city: Edinburg
country: United States
facility: Study site 09
state: Texas
city: Edinburg
country: United States
facility: Study site 23
state: Texas
city: San Antonio
country: United States
facility: Pinnacle Clinical Research (Study site 20)
state: Texas
Study officials:
References: