Vonafexor for Alport Syndrome (ALPESTRIA-1 Trial)

Phase 2

Recruiting

Research Sponsored by Enyo Pharma

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Has eGFR between ≥ 30 and < 90 ml/min/1.73m2

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

Must not have

Is taking CYP3A4/5 inhibitors or inducers

Has moderate or severe hepatic impairment (Child-Pugh score B or C)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up during on-treatment (assessed up to 24 weeks) and off-treatment periods (assessed up to 36 weeks) compared to baseline

Awards & highlights

No Placebo-Only Group

Summary

"This trial is testing how safe vonafexor is and how it affects kidney function in people with Alport syndrome who are at risk of their condition getting worse."

Who is the study for?

This trial is for people with Alport syndrome at risk of kidney function decline. Participants must have certain levels of kidney function, no hepatitis B/C or HIV, use contraception if applicable, and be on stable treatments for related conditions. Pregnant individuals or those with recent serious illnesses are excluded.

What is being tested?

The study tests the safety and impact of vonafexor on kidney function in Alport syndrome patients. It's a proof-of-concept trial to see how well this drug works and what effects it has on those who might face worsening kidney health.

What are the potential side effects?

While specific side effects aren't listed here, participants will be monitored for any adverse reactions to vonafexor which could include typical drug responses such as digestive issues, allergic reactions, changes in liver enzymes or other organ-related complications.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My kidney function, measured by eGFR, is between 30 and 90 ml/min.

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I have been diagnosed with Alport syndrome through symptoms, kidney biopsy, and genetic testing.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently taking medication that affects liver enzymes.

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My liver function is significantly impaired.

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I have not had any active cancer in the past year.

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I haven't had any major illnesses or conditions in the last 30 days that could affect my participation.

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My LDL cholesterol is high, putting me at risk for heart disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ during on-treatment (assessed up to 24 weeks) and off-treatment periods (assessed up to 36 weeks) compared to baseline

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~during on-treatment (assessed up to 24 weeks) and off-treatment periods (assessed up to 36 weeks) compared to baseline

This trial's timeline: 3 weeks for screening, Varies for treatment, and during on-treatment (assessed up to 24 weeks) and off-treatment periods (assessed up to 36 weeks) compared to baseline for reporting.