Fondazione Telethon
Fondazione Telethon
Explore linked clinical trials.8 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia | Transfusion-dependent Beta-Thalassemia | Recruiting | Phase 2 | 2026-07-27 | NCT07680803 | |
| Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis I, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis | Active, not recruiting | Phase 1 / Phase 2 | 2026-07-16 | NCT03488394 | |
| Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO) | Bone Diseases, Developmental, bone disorder, musculoskeletal system disorder, osteochondrodysplasia, osteopetrosis, osteosclerosis | Recruiting | Phase 1 / Phase 2 | 2026-06-24 | NCT07665021 | |
| Developing a Tailored Neuropsychological Rehabilitation for Sturge-Weber Syndrome | Infarction, Necrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, brain disorder, brain infarction, brain ischemia, brain stem infarction, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, ischemic disease, nervous system disorder, stroke disorder, vascular disorder | Recruiting | Not stated | 2026-06-17 | NCT07655492 | |
| Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) | immune system disorder, inborn error of immunity, severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency | Enrolling by invitation | Not stated | 2026-05-22 | NCT03478670 | |
| STOP-HSP.Net: a Registry for Hereditary Spastic Paraplegia as an Integration Tool for Future Therapeutic Strategies | Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, central nervous system malformation, hereditary disease, hereditary motor and sensory neuropathy, hereditary spastic paraplegia, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy | Recruiting | Not stated | 2026-03-27 | NCT06572046 | |
| A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Hemic and Lymphatic Diseases, Primary Immunodeficiency Diseases, Wiskott-Aldrich syndrome, X-linked disease, blood coagulation disease, hematologic disorder, hemorrhagic disease, hereditary disease, immune system disorder, inborn error of immunity, inherited blood coagulation disorder, leukocyte disorder, leukopenia, lymphopenia | Active, not recruiting | Phase 3 | 2025-09-29 | NCT03837483 | |
| Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD | Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Hemic and Lymphatic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, chronic granulomatous disease, hematologic disorder, hereditary disease, immune system disorder, inborn error of immunity, leukocyte disorder, phagocyte bactericidal dysfunction | Recruiting | Phase 2 | 2025-09-18 | NCT03055247 |