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Scientists hunt for genetic clues to personalize treatment for rare kidney disease

NCT ID NCT05805202

Knowledge-focused Sponsor: Mario Negri Institute for Pharmacological Research Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

About 112 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2023

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Adults and children with aHUS defined by history of microangiopathic hemolytic anemia and thrombocytopenia (hematocrit (Ht) \<30%, hemoglobin (Hb) \<10 g/dL, LDH \>500 IU/L, undetectable haptoglobin, fragmented erythrocytes in the peripheral blood smear with negative Coomb's test, and platelet count \<150,000/microL), associated with acute renal failure. * Written informed consent Exclusion Criteria: * TTP (ADAMTS13 activity \<10%) * STEC-HUS (presence of stx and eae genes or Shiga-toxin in the stools and/or serum antibodies against Shiga-toxin and/or STEC LPS). * Disseminated intravascular coagulation (prolonged thromboplastin time and lower than normal fibrinogen levels).

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Conditions

The condition(s) this trial relates to.

atypical hemolytic-uremic syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Atypical hemolytic uremic syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Centro di Ricerche Cliniche per le Malattie Rare "Aldo e Cele Daccò"

    RECRUITING

    Ranica, BG, 24020, Italy

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