FONDAZIONE TELETHON ETS
- Country
- ๐ฎ๐นItaly
- Ownership
- Subsidiary
- Established
- 1990-01-01
- Employees
- 51
- Market Cap
- -
- Website
- http://telethon.it
Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy
- Conditions
- Severe Combined Immunodeficiency Due to ADA Deficiency
- Interventions
- Genetic: Strimvelis
- First Posted Date
- 2021-07-13
- Last Posted Date
- 2024-01-29
- Lead Sponsor
- Fondazione Telethon
- Target Recruit Count
- 15
- Registration Number
- NCT04959890
- Locations
- ๐ฎ๐น
Ospedale San Raffaele, Milan, Italy
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome
- Conditions
- Wiskott-Aldrich Syndrome
- Interventions
- Genetic: OTL-103
- First Posted Date
- 2019-02-12
- Last Posted Date
- 2024-01-31
- Lead Sponsor
- Fondazione Telethon
- Target Recruit Count
- 10
- Registration Number
- NCT03837483
- Locations
- ๐บ๐ธ
Children's Healthcare of Atlanta, Inc, Atlanta, Georgia, United States
๐ฎ๐นOspedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET), Milan, Italy
Natural History Study in Subjects With Usher Syndrome
- Conditions
- Usher Syndrome, Type 1B
- First Posted Date
- 2019-01-24
- Last Posted Date
- 2024-12-09
- Lead Sponsor
- Fondazione Telethon
- Target Recruit Count
- 56
- Registration Number
- NCT03814499
- Locations
- ๐ฎ๐น
Eye Clinic of the University of Campania Luigi Vanvitelli, Naples, Italy
๐ณ๐ฑStichting Oogziekenhuis Rotterdam, Rotterdam, Netherlands
๐ช๐ธInstituto de Investigacion Sanitaria de la Fundacion Jimenez Diaz, Madrid, Spain
Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.
- Conditions
- Pyruvate Dehydrogenase Complex Deficiency
- Interventions
- First Posted Date
- 2018-11-07
- Last Posted Date
- 2021-10-13
- Lead Sponsor
- Fondazione Telethon
- Target Recruit Count
- 1
- Registration Number
- NCT03734263
- Locations
- ๐ฎ๐น
Federico II University, Napoli, Italy
Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
- Conditions
- Immunologic Deficiency Syndromes
- Interventions
- Genetic: Strimvelis
- First Posted Date
- 2018-03-27
- Last Posted Date
- 2024-01-29
- Lead Sponsor
- Fondazione Telethon
- Target Recruit Count
- 50
- Registration Number
- NCT03478670
- Locations
- ๐ฎ๐น
Ospedale San Raffaele, Milano, Lombardia, Italy
Retroviral Insertion Site Methodology Study
- Conditions
- Immune System Diseases
- Interventions
- Genetic: Strimvelis
- First Posted Date
- 2017-10-16
- Last Posted Date
- 2024-01-29
- Lead Sponsor
- Fondazione Telethon
- Registration Number
- NCT03311074
Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)
- Conditions
- Severe Combined Immunodeficiency Due to ADA Deficiency
- Interventions
- First Posted Date
- 2017-07-27
- Last Posted Date
- 2023-11-14
- Lead Sponsor
- Fondazione Telethon
- Target Recruit Count
- 16
- Registration Number
- NCT03232203
- Locations
- ๐ฎ๐น
Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET), Milan, Italy
Gene Therapy in Patients With Mucopolysaccharidosis Disease
- First Posted Date
- 2017-06-02
- Last Posted Date
- 2024-11-22
- Lead Sponsor
- Fondazione Telethon
- Target Recruit Count
- 9
- Registration Number
- NCT03173521
- Locations
- ๐ฎ๐น
Department of Translational Medicine (DISMET) of "Federico II" University, Naples, Naples, Italy
๐น๐ทChildren's Hospital Hacettepe University, Ankara, Turkey
Gene Therapy for Wiskott-Aldrich Syndrome
- Conditions
- Wiskott-Aldrich Syndrome (WAS)
- Interventions
- Genetic: OTL-103
- First Posted Date
- 2012-01-24
- Last Posted Date
- 2025-04-04
- Lead Sponsor
- Fondazione Telethon
- Target Recruit Count
- 8
- Registration Number
- NCT01515462
- Locations
- ๐ฎ๐น
Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET), Milan, Italy
ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
- Conditions
- Immunologic Deficiency Syndromes
- Interventions
- Genetic: Gene Therapy
- First Posted Date
- 2008-01-22
- Last Posted Date
- 2024-01-29
- Lead Sponsor
- Fondazione Telethon
- Target Recruit Count
- 12
- Registration Number
- NCT00598481
- Locations
- ๐ฎ๐ฑ
Investigational Site, Jerusalem, Israel
๐ฎ๐นOspedale San Raffaele, Milano, Lombardia, Italy