Alport syndrome
MONDO:0018965A rare renal disease characterized by glomerular nephropathy with hematuria progressing to end-stage renal disease (ESRD), frequently associated with sensorineural deafness, and occasionally with ocular anomalies.
Also known as: hereditary nephritis, Alport deafness-nephropathy, Alport syndrome, Alport's syndrome
20 clinical trials for this condition and its sub-types.
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Broader categories
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Kidney hope: drug combo may slow alport disease
Disease control CompletedThis completed Phase 4 trial tested two drugs—dapagliflozin and spironolactone—alone and together in 12 adults with Alport syndrome, a genetic kidney disease. The goal was to see if these drugs could lower protein levels in urine, a key sign of kidney damage. Participants took ea…
Phase: PHASE4 • Sponsor: Stefan Lujinschi • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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New drug aims to protect kidneys in rare genetic disease
Disease control CompletedThis study tested a new drug, setanaxib, in 20 people with Alport syndrome, a genetic condition that damages kidneys. The goal was to see if the drug is safe and can help preserve kidney function when added to standard care. Participants took either setanaxib or a placebo for 24 …
Phase: PHASE1, PHASE2 • Sponsor: Calliditas Therapeutics AB • Aim: Disease control
Last updated Jun 27, 2026 12:06 UTC
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Could a malaria drug help kids with a rare kidney disease?
Disease control CompletedThis study tested whether hydroxychloroquine, a drug used for malaria and autoimmune conditions, can safely reduce blood and protein in the urine of children with X-linked Alport syndrome, a genetic kidney disease. Fifty children aged 3 to 18 with stable kidney function took the …
Phase: PHASE2 • Sponsor: Shanghai Children's Hospital • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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Experimental drug targets genetic cause of alport syndrome in tiny pilot
Disease control CompletedThis phase 2 pilot study tested a drug called ELX-02 in 3 adults with Alport syndrome caused by specific 'nonsense' mutations. The goal was to see if the drug is safe and if it can reduce protein in the urine, a sign of kidney damage. Participants received daily injections for 8 …
Phase: PHASE2 • Sponsor: Eloxx Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
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New drug shows promise for slowing kidney disease in alport syndrome
Disease control CompletedThis study tested a drug called vonafexor in 26 people with Alport syndrome, a genetic condition that damages kidneys. The goal was to see if the drug is safe and if it can help protect kidney function. Participants took increasing doses over 24 weeks. The study is now complete, …
Phase: PHASE2 • Sponsor: Enyo Pharma • Aim: Disease control
Last updated Jun 27, 2026 09:03 UTC
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New hope for kidney patients: experimental drug targets protein leak
Disease control CompletedThis study tested an experimental drug called R3R01 in 43 people with two rare kidney diseases (Alport syndrome and focal segmental glomerulosclerosis) whose protein in urine remained high despite standard treatment. The main goals were to check safety and see if the drug could r…
Phase: PHASE2 • Sponsor: River 3 Renal Corp. • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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Alport syndrome registry opens doors for future therapies
Knowledge-focused CompletedThis study created a registry of 655 people and families with Alport syndrome to collect health information over time. The goal is to better understand how the disease progresses and to provide a foundation for testing new treatments. Participants simply share their medical histo…
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:59 UTC