Scientists hunt for genetic clues to personalize treatment for rare kidney disease
NCT ID NCT05805202
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looks at people with atypical hemolytic uremic syndrome (aHUS), a rare disease that damages small blood vessels and kidneys. Researchers want to understand how certain rare gene mutations cause the disease by growing cells from blood samples in the lab. The goal is to find drugs that could correct these genetic problems, leading to more personalized treatments in the future.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- What this could lead to
- If successful, this research could point toward personalized therapies for atypical HUS based on a patient's specific genetic mutation.
- What could go wrong
- This is an observational study focused on understanding the disease, not testing a treatment. It is early-stage research, and any potential therapies would need many more years of testing.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Centro di Ricerche Cliniche per le Malattie Rare "Aldo e Cele Daccò"
RECRUITINGRanica, BG, 24020, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Skin biopsies could unlock hidden genetic diagnoses
- Real-World study tracks Ravulizumab's impact on rare kidney disease
- New pill shows promise for rare blood disorder aHUS
- New drug ravulizumab aims to control rare blood disorder aHUS
- Promising aHUS drug trial stalls after only 6 patients enrolled