Rare Disease Trial, Recruiting (NCT05805202)

Rare Disease Trial, Recruiting NCT05805202 Sponsor: Mario Negri Institute for Pharmacological Research Condition: Rare Disease

Printed from Voxsanity (voxsanity.com.au) — NCT05805202 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

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Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

Adults and children who have been diagnosed with a condition called aHUS, which means their blood shows signs of red blood cell destruction, low platelet counts, and kidney failure (confirmed through specific blood tests)
Anyone joining must be willing and able to provide written consent (or have a parent/guardian provide it for children)

Who may not be able to join:

People whose condition is caused by a different blood disorder called TTP (Thrombotic Thrombocytopenic Purpura), identified by a specific enzyme test result
People whose condition was caused by a bacterial infection involving Shiga toxin (sometimes linked to certain types of food poisoning), confirmed through stool or blood tests
People who have a separate blood clotting disorder called DIC (Disseminated Intravascular Coagulation), identified through abnormal clotting test results

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.

Last synced 28 July 2026

This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.

Contact this trial

Phone: +3903545351

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Voxsanity

Clinical trials and treatments, explained plainly · voxsanity.com.au

Date:

To: My Doctor

Re: Clinical trial enquiry

I am writing to enquire about the following clinical trial which may be relevant to my care:

Trial: NCT05805202

Sponsor: Mario Negri Institute for Pharmacological Research

Phase: Not Applicable

Status: Recruiting

Condition: Rare Disease

Trial contact: +3903545351

More information: https://clinicaltrials.gov/study/NCT05805202

Eligibility summary (AI generated)

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

Adults and children who have been diagnosed with a condition called aHUS, which means their blood shows signs of red blood cell destruction, low platelet counts, and kidney failure (confirmed through specific blood tests)
Anyone joining must be willing and able to provide written consent (or have a parent/guardian provide it for children)

Who may not be able to join:

People whose condition is caused by a different blood disorder called TTP (Thrombotic Thrombocytopenic Purpura), identified by a specific enzyme test result
People whose condition was caused by a bacterial infection involving Shiga toxin (sometimes linked to certain types of food poisoning), confirmed through stool or blood tests
People who have a separate blood clotting disorder called DIC (Disseminated Intravascular Coagulation), identified through abnormal clotting test results

Important: Always verify eligibility with the trial site directly before applying.

Access in Australia: A treatment being studied in a clinical trial is generally not yet listed on the PBS. Where a medicine is not approved or not available through a trial, the TGA Special Access Scheme may allow a doctor to access unapproved therapeutic goods for individual patients. See voxsanity.com.au/sas-navigator/ for a plain English explainer.

This information was sourced from ClinicalTrials.gov via Voxsanity (data last synced 28 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

Recruiting

Phase

Not Applicable

Sponsor

Mario Negri Institute for Pharmacological Research

Registry

ClinicalTrials.gov

Start date

3 May 2023

Est. completion

31 December 2026

Where this trial is recruiting

🇮🇹 Italy

Primary endpoints

Generation and characterization of patient-specific and healthy donor iPSC; Differentiation of iPSC into endothelial cells; Characterizationof iPSC into endothelial cells; Cell culture viability

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov