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RECRUITINGNANCT05805202

Functional Implications of Rare Gene Mutations in aHUS Open the Door to Personalized Therapy

Hemolytic Uremic Syndrome (HUS) is a rare disease characterized by rupture of red blood cells (hemolytic anemia), low platelet count (thrombocytopenia), and thrombotic occlusion of small vessels (thro...

Lead sponsor: Mario Negri Institute for Pharmacological Research•1 locations•View source on ClinicalTrials.gov

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Contacts

Marina Noris, Dr.

CONTACT

+3903545351 marina.noris@marionegri.it

Sponsors

Lead Sponsor

Mario Negri Institute for Pharmacological Research

Age

All ages

Sex

ALL

Healthy Volunteers

Yes

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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Data from ClinicalTrials.govLast updated on ClinicalTrials.gov: September 29, 2025Terms

Trial snapshot

StatusRECRUITING

PhaseNA

Enrollment112

Start dateMay 2023

Last updatedSep 29, 2025

Condition

Atypical Hemolytic Uremic Syndrome

Locations shown

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

Ranica, BG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 29, 2025Data synced to Clareo: May 17, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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