Alport SyndromePhase 2ALPESTRIA-1NCT06425055
Vonafexor ALPort Syndrome Efficacy & Safety TRIAl-1 (ALPESTRIA-1)
A Phase 2 study testing Vonafexor in people with Alport Syndrome, run by Enyo Pharma at 13 sites in 3 countries.
Trial summary
This study is a proof-of-concept trial of vonafexor safety, its effects on kidney function in subjects with at risk of progression Alport syndrome. Read more about this study
- Who is it for?
- Ages 16 to 55 years
- Conditions the study is for
- Alport Syndrome
- What is being tested?
- Vonafexor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 13 sitesin 3 countries
- Study dates
- 1 Aug 2024to 20 Nov 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Signed informed consent (also for legal representatives, as applicable in the US for under eighteen patients).
- Has confirmed diagnosis of Alport syndrome: clinical diagnosis (haematuria, family history, hearing loss, ocular change) OR a kidney biopsy showing glomerular basement membrane abnormalities consistent with AS, AND Genetic confirmation of AS.
- Has eGFR between ≥ 30 and < 90 ml/min/1.73m2.
- Has increased albuminuria criteria i.e. UACR ≥ 300 mg/g.
- If on an angiotensin converting enzyme inhibitor (ACEi) and/or angiotensin receptor blocker (ARB), should be on a stable well tolerated treatment during at least the 60 days prior D1.
- If on Sodium-Glucose Transport Protein 2 (SGLT2), should be on stable well tolerated treatment with SGLT2 during at least 60 days prior D1.
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- If patient has a history of arterial hypertension, should be on stable anti-hypertensive therapy for at least 60 days prior to D1 and deemed controlled by the investigator at screening and D1.
- Sexually active female subjects of childbearing potential and sexually mature male subjects must use two acceptable effective methods of contraception for the entire duration of the study and for at least 6 weeks after last dose.
- Has negative results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, and human immunodeficiency virus (HIV).
- Is able to understand all study procedures in the informed consent form (ICF) and willing to comply with all aspects of the protocol.
Who cannot join
- Is an employee of a site, clinical research organization, vendor, or sponsor involved with this study.
- Is pregnant or breastfeeding.
- Has participated in any investigational drug study within 60 days prior to D1.
- Any clinically significant illness within 30 days before D1 or surgical or medical condition (other than Alport syndrome) that could interfere with the subject's study compliance; confound the study results; impact subject safety.
- Any history of active malignancy within the last 1 year before D1.
- Any other condition or circumstance that, in the opinion of the investigator, may make the subject unlikely to complete the study or comply with study procedures and requirements, or may pose a risk to the subject's safety and well-being.
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- Has a history of an allergic condition that required the prescription of an emergency epinephrine injection (such as the EpiPen® Auto-Injector).
- Any prohibited co-medications within 30 days prior D1.
- Has ALT or AST above near normal (>1.5×ULN) at baseline.
- Are at high risk for atherosclerotic cardiovascular disease (ASCVD) risk, with an LDL-C level > 160 mg/dL (4.15 mmol/L) and subjects at intermediate risk for ASCVD risk, with a LDL-C level > 190 mg/dL (4.91 mmol/L).
- Has moderate or severe hepatic impairment (Child-Pugh score B or C).
- Is taking CYP3A4/5 inhibitors or inducers.
Trial locations and contacts
13 sites in 3 countries.
13 sites in 3 countries
United States7 sites
Dr Eric Wallace - University of Alabama
Birmingham, Alabama, 35294
Dr Arnold Silva - Boise Kidney & Hypertension
Boise, Idaho, 83703
Dr James Simon - Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Dr Ankit Mehta - Renal Disease Research Institute
Dallas, Texas, 75126
Dr Suneel Udani - NANI Research
Hinsdale, Illinois, 60521
Dr Anjay Rastogi - UCLA Health, David Geffen School of Medicine
Los Angeles, California, 90095
Dr Tingting Li - Washington University
St Louis, Missouri, 63110
France3 sites
Pr Claire Rigothier - CHU De Bordeaux
Bordeaux, 33076
Dr Moglie Le Quintrec - Hopital Lapeyronie
Montpellier, 34090
Pr. Bertrand Knebelmann - Necker Enfants Malades
Paris, 75015
Spain3 sites
Fundacio Puigvert
Barcelona, 08025
Hospital Virgen de la Arrixaca
El Palmar, 30120
Fundacion Jimenez Diaz
Madrid, 28040
About this study
Official title: Vonafexor Fixed Dose-escalation Safety and Proof-of-concept Study in Patients With at Risk of Progression Alport Syndrome
This study is a proof-of-concept trial of vonafexor safety, its effects on kidney function in subjects with at risk of progression Alport syndrome.
Read the detailed description
This is a multicenter study and several clinical sites and countries will be involved.
This single arm, fixed dose escalation, open-label, non-randomized study will evaluate three dose levels of vonafexor on safety, tolerability and their effect on kidney function and renal biomarkers in 20 patients with AS at risk of progression.
The total duration of study for a participant will be up to 40 weeks and include a screening period, a treatment period of 24 weeks and a follow-up period of 12 weeks.
- Conditions
- Alport Syndrome
- Being tested
- Vonafexor
- Medical subject headings
- Nephritis, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VonafexorDrug
Also called EYP001a
- One tablet of a low dose of vonafexor QD from Day 1 to Week 4
- One tablet of a medium dose of vonafexor QD from Week 5 to Week 8
- One tablet of a high dose of vonafexor QD from Week 9 to Week 24
| Study group | Role | What they receive |
|---|---|---|
| Single arm fixed dose escalation | Experimental | This is a single arm fixed dose escalation with three dose levels of vonafexor, all QD. Receives: Drug: Vonafexor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Treatment-Emergent Adverse Event (TEAE)
To assess the safety and tolerability of vonafexor
Measured: From first dose of treatment until 2 weeks after last dose of treatment, i.e assessed up to 26 weeks
Secondary outcomes (2)
Change in eGFR
To determine the on with the off-treatment effect of three dose levels of vonafexor on renal function
Measured: During on-treatment (assessed up to 24 weeks) and off-treatment periods (assessed up to 36 weeks) compared to baseline
Vonafexor plasma concentrations
To determine vonafexor plasma concentrations levels
Measured: During on-treatment period, assessed up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2024
- Study start1 Aug 2024
- Primary completion4 Sep 2025
- Study completion20 Nov 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Alport SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single arm fixed dose escalation
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Enyo Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06425055
- Sponsor's protocol ID
- EYP001-208
- Acronym
- ALPESTRIA-1
- Status last verified
- Feb 2026
- First submitted
- 14 May 2024
- Last updated
- 9 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual participant data that underline published results might be shared upon request with researchers who provide a methodologically sound proposal and to achieve objectives as set in the approved proposal. IPD will be shared deidentified and from 3 months and ending 5 years following article publication.
Publications
Rheault MN. Treatment Approaches for Alport Syndrome. J Am Soc Nephrol. 2026 Jan 1;37(1):172-179. doi: 10.1681/ASN.0000000897. Epub 2025 Sep 12.
PubMed 40938675
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Vonafexor Fixed Dose-escalation Safety and Proof-of-concept Study in Patients With at Risk of Progression Alport Syndrome
- ClinicalTrials.gov ID
- NCT06425055
- Acronym / sponsor ID
- ALPESTRIA-1 / EYP001-208
- Conditions
- Alport Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Enyo PharmaIndustry (pharma, biotech, devices)
- Study start
- 1 Aug 2024 (actual)
- Primary completion
- 4 Sep 2025 (actual)
- Study completion
- 20 Nov 2025 (actual)
- First posted
- 22 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a proof-of-concept trial of vonafexor safety, its effects on kidney function in subjects with at risk of progression Alport syndrome.
Detailed description
This is a multicenter study and several clinical sites and countries will be involved.
This single arm, fixed dose escalation, open-label, non-randomized study will evaluate three dose levels of vonafexor on safety, tolerability and their effect on kidney function and renal biomarkers in 20 patients with AS at risk of progression.
The total duration of study for a participant will be up to 40 weeks and include a screening period, a treatment period of 24 weeks and a follow-up period of 12 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single arm fixed dose escalation
| Arm | Type | Receives |
|---|---|---|
| Single arm fixed dose escalation | Experimental | Drug: VonafexorThis is a single arm fixed dose escalation with three dose levels of vonafexor, all QD. |
Who may qualify
- Ages
- 16 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Signed informed consent (also for legal representatives, as applicable in the US for under eighteen patients).
- Has confirmed diagnosis of Alport syndrome: clinical diagnosis (haematuria, family history, hearing loss, ocular change) OR a kidney biopsy showing glomerular basement membrane abnormalities consistent with AS, AND Genetic confirmation of AS.
- Has eGFR between ≥ 30 and < 90 ml/min/1.73m2.
- Has increased albuminuria criteria i.e. UACR ≥ 300 mg/g.
- If on an angiotensin converting enzyme inhibitor (ACEi) and/or angiotensin receptor blocker (ARB), should be on a stable well tolerated treatment during at least the 60 days prior D1.
Key exclusion criteria (12 listed)
- Is an employee of a site, clinical research organization, vendor, or sponsor involved with this study.
- Is pregnant or breastfeeding.
- Has participated in any investigational drug study within 60 days prior to D1.
- Any clinically significant illness within 30 days before D1 or surgical or medical condition (other than Alport syndrome) that could interfere with the subject's study compliance; confound the study results; impact subject safety.
- Any history of active malignancy within the last 1 year before D1.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VonafexorAlso: EYP001a* One tablet of a low dose of vonafexor QD from Day 1 to Week 4 * One tablet of a medium dose of vonafexor QD from Week 5 to Week 8 * One tablet of a high dose of vonafexor QD from Week 9 to Week 24 | Drug | Single arm fixed dose escalation |
Primary outcome
Number of Treatment-Emergent Adverse Event (TEAE)
Time frame: From first dose of treatment until 2 weeks after last dose of treatment, i.e assessed up to 26 weeks
To assess the safety and tolerability of vonafexor
2 secondary outcomes
Change in eGFR
Time frame: During on-treatment (assessed up to 24 weeks) and off-treatment periods (assessed up to 36 weeks) compared to baseline
Vonafexor plasma concentrations
Time frame: During on-treatment period, assessed up to 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2026.
- Derived from this study: PubMed 40938675Rheault MN. Treatment Approaches for Alport Syndrome. J Am Soc Nephrol. 2026 Jan 1;37(1):172-179. doi: 10.1681/ASN.0000000897. Epub 2025 Sep 12.
- Individual participant dataSharing planned: Yes. Individual participant data that underline published results might be shared upon request with researchers who provide a methodologically sound proposal and to achieve objectives as set in the approved proposal. IPD will be shared deidentified and from 3 months and ending 5 years following article publication.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
13 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vonafexor in people with Alport Syndrome, run by Enyo Pharma at 13 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, France, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Treatment-Emergent Adverse Event (TEAE) (time frame: From first dose of treatment until 2 weeks after last dose of treatment, i.e assessed up to 26 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06425055, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Feb 2026.