2 completed, 1 active not recruiting, 1 terminated. 2 Phase 3, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 2 | Completed 1, Terminated 1 |
| Phase 2 | 1 | Completed 1 |
Follow Tuberous Sclerosis 2: new trials by email Literature Review of Tuberous Sclerosis 2 trials Research Report on Tuberous Sclerosis 2
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1) nab-sirolimus - Tumor, Tumor, Solid | Phase 2 | Completed | Aadi Bioscience, Inc. | 128 | 2022-03-10 | 2026-10-28 |
| Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures GWP42003-P - Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome | Phase 3 | Terminated | Jazz Pharmaceuticals | 3 | 2021-05-19 | 2025-10-31 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) Everolimus (RAD001), Everolimus Placebo - Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM) | Phase 3 | Completed | Novartis Pharmaceuticals | 118 | 2009-04-01 | 2017-02-17 |
Tuberous Sclerosis 1 (3) · Epilepsies, Myoclonic (2) · Epilepsy (2) · Hereditary Sensory and Autonomic Neuropathies (2) · Seizures (2) · Tuberous Sclerosis (2) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1) · 3-methylcrotonyl CoA carboxylase 1 deficiency (1) · 3-methylcrotonyl CoA carboxylase 2 deficiency (1) · Acidemia, isovaleric (1) · Acrodermatitis enteropathica (1) · Adenine phosphoribosyltransferase deficiency (1) · Adrenal hyperplasia, congenital, type 5 (1)
Congenital Abnormalities (4) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4) · Heredodegenerative Disorders, Nervous System (4) · Nervous System Diseases (4) · Neurodegenerative Diseases (4) · Pathological Conditions, Signs and Symptoms (4) · Neoplasms (3) · Neoplasms, Multiple Primary (3) · Neoplastic Syndromes, Hereditary (3) · Nervous System Malformations (3) · Neurologic Manifestations (3) · Neuromuscular Diseases (3) · Brain Diseases (2) · Central Nervous System Diseases (2)
Cannabidiol (1) · Everolimus (1)
Aadi Bioscience, Inc. (1) · Jazz Pharmaceuticals (1) · Novartis Pharmaceuticals (1) · RTI International (1)
United States (4) · Italy (2) · Spain (2) · Canada (1) · France (1) · Germany (1) · Japan (1) · Netherlands (1) · Poland (1) · Puerto Rico (1) · Russia (1) · South Korea (1) · United Kingdom (1)
PMID 26156073, PMID 29023494, PMID 30053159, PMID 30192751, PMID 31315600, PMID 35142618
2008 (1)