Sponsor Pipeline

Fondazione Telethon Clinical Trial Pipeline

Data updated daily from ClinicalTrials.gov — last refresh 2026-08-16
4
Active Trials
2
Recruiting
1
Phase 3
1
Disease Areas
1
Changes, 30 Days
What Changed in the Last 30 Days
DetectedTrialChange
2026-07-28 NCT07680803
Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia		 Recruitment opened
Active Trials by Phase
Phase 3
1
Phase 2
1
Phase 1/2
1
Not specified
1
Active Trials
NCT IDTitleConditionStatus
Phase 3
NCT03837483 A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in… Wiskott-Aldrich Syndrome Active, Not Recruiting
Phase 2
NCT07680803 Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia Transfusion-dependent Beta-Thalassemia Recruiting
Phase 1/2
NCT07665021 Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO) Osteopetrosis Recruiting
Not specified
NCT03478670 Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined… Immunologic Deficiency Syndromes Enrolling by Invitation
Disease Areas
Thalassemia (1)
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