RecruitingPhase 2NCT07211685
Connective Tissue
Alport Syndrome
Also known as hereditary nephritis, COL4A3/COL4A4/COL4A5 nephropathy
Alport syndrome is a progressive nephropathy caused by pathogenic variants in genes encoding type IV collagen alpha chains, which are essential structural components of the glomerular basement membrane, cochlea, and ocular lens. The hallmar
ORPHA:63 ↗Gene COL4A3Gene COL4A4Gene COL4A5Prevalence 1 in 5,000–10,000Onset Early childhood (symptoms), adolescence–adulthood (ESKD)Genetic — X-linked (most common), autosomal recessive, autosomal dominant
11
studies recruiting now
as of 7 Sept 2026
39
studies registered in total
as of 7 Sept 2026
18
countries with a recruiting site
as of 7 Sept 2026
8 May 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 2NCT07523581
EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety
RecruitingNCT07575347
Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)
RecruitingNot applicableNCT04571658
NEPTUNE Match Study
RecruitingPhase 2NCT05003986
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 11 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Alport Syndrome
Alport syndrome is a progressive nephropathy caused by pathogenic variants in genes encoding type IV collagen alpha chains, which are essential structural components of the glomerular basement membrane, cochlea, and ocular lens. The hallmark is persistent microscopic haematuria progressing to proteinuria, declining GFR, and end-stage kidney disease, typically earlier in males with X-linked disease. Sensorineural hearing loss and characteristic ocular findings (anterior lenticonus) complete the classic triad.
Common clinical features
Persistent microscopic haematuria from infancyProgressive proteinuriaDeclining renal function progressing to end-stage kidney diseaseSensorineural hearing loss (bilateral, high-frequency)Anterior lenticonus of the ocular lensMacular flecks on retinal examinationRecurrent macroscopic haematuria in children following upper respiratory infections
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
10 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 3Bardoxolone Methyl
Phase 2/3Solizmestrocel
Phase 2Elx-02
Phase 2Vonafexor
Phase 2Lademirsen
Phase 2Valsartan (Diovan)
Phase 2Benazepril
Phase 2Fluvastatin
+ 2 more in development
Before you apply
Things trial teams commonly ask about for Alport Syndrome. Not eligibility rules; those are set by each study.
- Current eGFR, urine protein-to-creatinine ratio, and kidney biopsy showing thinning or lamellation of the GBM on electron microscopy are standard eligibility documents — have these ready.
- X-linked vs. autosomal inheritance affects trial eligibility; confirm molecular diagnosis specifying which COL4A gene is affected and the inheritance pattern.
- Patients already on ACE inhibitors or ARBs should note dosing, as trials may require stable background therapy or specific washout before enrolment.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).