Phase 2
N=117
Study of PXL065 in Patients With Nonalcoholic Steatohepatitis (NASH)
NASH - Nonalcoholic Steatohepatitis
Bottom Line
View on ClinicalTrials.gov: NCT04321343 ↗Enrolled (actual)
117
Serious AEs
6.0%
Results posted
Aug 2023
Primary outcome: Primary: 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]) — -22.900; -18.590; -21.335; 2.413 percentage of change in LFC — p=0.0083
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- PXL065 (Drug); Placebo oral tablet (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Poxel SA
- Primary completion
- Jun 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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]) |
-22.900; -18.590; -21.335; 2.413 | 0.0083 sig |
| PRIMARY Relative Change From Baseline to Week 36 in the Percentage of LFC (Assessed by MRI-PDFF) (Wilcoxon Test Sensitivity Analysis) |
-26.555; -25.353; -24.782; 0.937 | 0.0105 sig |
| SECONDARY Absolute Change From Baseline to Week 36 in the Percentage of LFC (Assessed by MRI-PDFF) |
-4.934; -4.360; -4.584; -0.263 | 0.0105 sig |
| SECONDARY Percentage of Responders (Relative Reduction of at Least 30% in LFC) at Week 36 |
8; 11; 12; 5; 12; 13 | 0.1634 |
| SECONDARY Change From Baseline to Week 36 in Alanine Amino Transferase (ALT) |
-18.4; -13.6; -6.7; -11.9 | 0.4318 |
| SECONDARY Percentage of Responders (Normalization of ALT) |
3; 12; 10; 4; 14; 12 | 0.7137 |
| SECONDARY Change From Baseline to Week 36 in Aspartate Amino Transferase (AST) |
-10.1; -10.2; -4.9; -7.2 | 0.7449 |
| SECONDARY Percentage of Responders (Normalization of AST) |
9; 4; 11; 4; 8; 13 | 0.1325 |
| SECONDARY Change From Baseline to Week 36 in Gamma Glutamyltransferase (GGT) |
-12.2; -21.8; -6.9; -4.2 | 0.4413 |
| SECONDARY Change From Baseline to Week 36 in Alkaline Phosphatase (ALP) |
-2.8; -8.0; -5.8; 3.1 | 0.1166 |
| SECONDARY Change From Baseline to Week 36 in Pro-C3 |
-2.054; -1.753; -2.481; -1.041 | 0.4963 |
| SECONDARY Change From Baseline to Week 36 in Enhanced Liver Fibrosis (ELF) Score |
-0.142; -0.206; -0.358; -0.079 | 0.7672 |
| SECONDARY Change From Baseline to Week 36 in Fibrosis-4 (Fib-4) Score |
-0.040; -0.107; -0.198; -0.012 | 0.8662 |
| SECONDARY Change From Baseline to Week 36 in NAFLD Fibrosis Score |
0.170; -0.006; -0.264; 0.215 | 0.8595 |
| SECONDARY Improvement of at Least 1 Point in NASH CRN Fibrosis Score From Baseline to Week 36 |
9; 11; 9; 4; 12; 11 | 0.1084 |
| SECONDARY Improvement in NAS of at Least 2 Points With no Worsening in NASH CRN Fibrosis Score From Baseline to Week 36 |
8; 11; 13; 7; 13; 11 | 0.7677 |
| SECONDARY NASH Resolution With no Worsening in NASH CRN Fibrosis Score at Week 36 |
8; 8; 8; 7; 13; 14 | 0.5981 |
| SECONDARY NASH Resolution With Improvement of at Least 1 Point in NASH CRN Fibrosis Score at Week 36 |
7; 7; 4; 3; 14; 15 | 0.1474 |
| SECONDARY Change From Baseline to Week 36 in Glycated Hemoglobin (HbA1c) |
0.14; -0.06; -0.20; 0.21 | 0.6384 |
| SECONDARY Change From Baseline to Week 36 in Fasting Plasma Glucose (FPG) |
0.147; 0.361; 0.205; 0.198 | 0.8961 |
| SECONDARY Change From Baseline to Week 36 in Serum Insulin |
-78.08; -39.76; -56.55; 6.85 | 0.0216 sig |
| SECONDARY Change From Baseline to Week 36 in Serum C-peptide |
-0.205; -0.222; -0.250; 0.051 | 0.0958 |
| SECONDARY Change From Baseline to Week 36 in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) |
-0.086; -0.123; -0.210; 0.111 | 0.2606 |
| SECONDARY Change From Baseline to Week 36 in Quantitative Insulin Sensitivity Check Index (QUICKI) |
0.006; 0.007; 0.012; -0.005 | 0.3955 |
| SECONDARY Change From Baseline to Week 36 in Adipo-IR |
-36.18; -17.01; -41.87; 12.77 | 0.0199 sig |
| SECONDARY Change From Baseline to Week 36 in Adiponectin |
1.532; 2.688; 4.734; -0.143 | 0.0639 |
| SECONDARY Change From Baseline to Week 36 in Weight |
1.080; 2.356; 0.577; -0.103 | 0.1944 |
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
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
Data sourced from ClinicalTrials.gov (NCT04321343). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.