Study of an experimental drug for Alport syndrome
Part of Genetic & congenital, Kidney & urinary, Skin, Women’s health & pregnancy clinical trials.
This study tests an experimental medicine for people with Alport syndrome caused by a specific type of gene change (nonsense mutation). The drug may help reduce protein in the urine and slow kidney decline. It is a blinded study, so you won't know if you get the real drug or a placebo.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have Alport syndrome due to a nonsense mutation in the COL4A5 gene (if you are male) or COL4A3/COL4A4 gene (male or female).
- Your kidney function (eGFR) must be above 40 ml/min/1.73 m².
- You must have protein in your urine (spot urine protein/creatinine ratio of 300 mg/g or higher).
- You must be on a stable dose of an ACE inhibitor or ARB (blood pressure/kidney medication) for at least 12 weeks before the study starts.
- You must not have had an organ transplant, be on dialysis, have liver disease (cirrhosis, portal hypertension, or elevated liver enzymes/bilirubin), or have had acute kidney injury in the past 4 weeks.
- You must not have active dizziness.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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