Plain-English translation of NCT07680803 on ClinicalTrials.gov ↗ · Source last updated · How we translate trials
Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.
Italy
Testing effectiveness
Sponsor
Fondazione Telethon
Collaborators
Ospedale San Raffaele
Enrollment target
~9 participants
Started
July 2026
Primary completion
October 2028
Age range
3 Years – 35 Years
Last updated on clinicaltrials.gov in July 2026.
Reach out to the team running this trial. Response times vary — some teams are faster than others.
Central contact
Alessandro Aiuti, Professor
Fondazione Telethon
Tell us you're interested and we'll help connect you with the research team. We'll walk you through what to expect first — no email needed to get started.