Clinical Trials Directory

Trials / Sponsors / Fondazione Telethon

Fondazione Telethon

Academic / Other · 10 registered clinical trials — 1 currently recruiting.

StatusTrialPhaseStarted
UnknownMethodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy
Severe Combined Immunodeficiency Due to ADA Deficiency
—2021-04-23
WithdrawnRetroviral Insertion Site Methodology Study
Immune System Diseases
Phase 42020-06-25
Active Not RecruitingA Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldric
Wiskott-Aldrich Syndrome
Phase 32019-01-21
CompletedUse of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.
Pyruvate Dehydrogenase Complex Deficiency
Phase 22018-10-01
CompletedNatural History Study in Subjects With Usher Syndrome
Usher Syndrome, Type 1B
—2018-06-01
CompletedEvaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)
Severe Combined Immunodeficiency Due to ADA Deficiency
—2018-04-12
CompletedGene Therapy in Patients With Mucopolysaccharidosis Disease
Mucopolysaccharidosis Type VI
Phase 1 / Phase 22017-07-17
Enrolling By InvitationStrimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA
Immunologic Deficiency Syndromes
—2017-03-28
CompletedGene Therapy for Wiskott-Aldrich Syndrome
Wiskott-Aldrich Syndrome (WAS)
Phase 1 / Phase 22010-04-20
CompletedADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
Immunologic Deficiency Syndromes
Phase 22002-10-02