Sponsor or institution

Clinical trials and research at Fondazione Telethon

• 2Clinical trials
• 1Country

All recruiting trials (2)

• Gene-modified stem cell therapy for transfusion-dependent beta-thalassemia

  A small, early-phase study will test a one-time infusion of a patient’s own genetically edited stem cells to see if it reduces transfusion needs in adults and children with transfusion-dependent beta-thalassemia.

  Recruiting at 2 sites Phase 2 Fondazione Telethon · Rome, Lazio and 1 other locations · NCT07680803

• Gene-modified stem cell therapy for children with autosomal recessive osteopetrosis

  This study tests a gene-modified stem cell therapy (FT024) in eight young children with TCIRG1-related autosomal recessive osteopetrosis to see if it is safe and feasible and to observe potential benefits.

  Recruiting Phase 1/2 Fondazione Telethon · Milan, Italy · NCT07665021

Quick answers

How many clinical trials run by Fondazione Telethon are recruiting right now?

2, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

All of them have sites in Italy. Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 1, Phase 1/2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

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