Vonafexor in Impaired Renal Function and Suspected MASH

Overview

Sponsor-declared trial summary

Key facts

Sponsor

ENYO Pharma

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trial duration

1 Jul 2025 → 13 Mar 2026

Decision date (initial)

2024-03-14

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Safety, Efficacy, Pharmacokinetic

To determine the effect of vonafexor on renal function in a population with suspected MASH and mild to moderately reduced GFR.

Secondary objectives 3

1. To determine the PK profile of vonafexor 25 mg and 100 mg QD
2. To compare the on-treatment with the off-treatment effect of two dose levels of vonafexor on renal function and proteinuria
3. To determine the safety and tolerability profile of two dose levels of vonafexor

Conditions and MedDRA coding

Impaired renal function and suspected MASH

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Male or female subject.
2. Age between 18 and 75 years, both inclusive.
3. Overweight or obesity (body mass index BMI ≥ 25.0 kg/m2 and ≤ 45.0 kg/m2) with or without type 2 diabetes mellitus (T2DM with an HbA1c ≤ 9.5%).
4. eGFR ≥ 30 and < 90 (mL/min/1.73 m²).
5. Presumed mild to higher liver fibrosis as shown by a FIBROTEST score ≥ 0.28 and/or FIB-4 score ≥ 1.3.

Exclusion criteria 5

1. Known or suspected hypersensitivity to IMP or any of the excipients or to any component of the IMP formulation.
2. History of multiple and/or severe allergies to drugs including contrast media or foods or a history of severe anaphylactic reaction.
3. Known non-MASH liver disease.
4. History or presence of cirrhosis.
5. Proteinuria in the nephrotic range with a protein-to-creatinine ratio > 3.5 g protein/g creatinine (350 mg/mmol).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline of mGFRiohexol and eGFRcreat at W16

Secondary endpoints 7

1. Plasma concentrations pre-dose (0h) and post-dose (2h, 5h, and 7h) on Day 1 (W0) and D113 (W16), which will be modelled against the MASH PopPK expected values.
2. Change from baseline mGFRiohexol off treatment at W24.
3. Change from baseline of eGFRcreat on treatment at W4, W8, W12 and off treatment at W20, W24 and W28
4. Correlation of mGFRiohexol with eGFRcreat at baseline, on treatment at W16 and off treatment at W24
5. Levels and change in albuminuria (Urinary Albumin to Creatinine Ratio (UACR) and proteinuria (Urinary Protein to Creatinine ratio (UPCR)) in morning urine samples at baseline, on treatment at W4, W8, W12, W16 with off treatment at W20, W24 and W28.
6. AEs, SAEs
7. Changes from baseline at W16 in laboratory safety parameters, physical examination, vital signs and ECGs

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Auxiliary 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

ENYO Pharma

Sponsor organisation

ENYO Pharma

Address

Wellio Silex 2, 9 Rue Des Cuirassiers 9 Rue Des Cuirassiers

City

Lyon

Postcode

69003

Country

France

Scientific contact point

Organisation

ENYO Pharma

Contact name

Clinical Operation Department

Public contact point

Organisation

ENYO Pharma

Contact name

Clinical Operation Department

Third parties 3

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications