Sponsor pipeline

Fondazione Telethon clinical trial pipeline

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Data updated daily from ClinicalTrials.gov, last refresh 2026-10-16

Active trials by phase

Phase 1/2
1
Phase 2
1
Phase 3
1
Not charted: 1 active trial with no phase listed on ClinicalTrials.gov (typically behavioural, device, procedure and observational studies).

Active trials

NCT IDTitleConditionStatusUpdated
Phase 3
NCT03837483 A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in… Wiskott-Aldrich Syndrome Active, Not Recruiting 2025-09-29
Phase 2
NCT07680803 Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia Transfusion-dependent Beta-Thalassemia Recruiting 2026-07-27
Phase 1/2
NCT07665021 Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO) Osteopetrosis Recruiting 2026-06-24
Not specified
NCT03478670 Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined… Immunologic Deficiency Syndromes Enrolling by Invitation 2026-05-22

Disease areas by active trials

Thalassemia (1)

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