Tuberous Sclerosis 1: Clinical Trials

3 trials for Tuberous Sclerosis 1, newest first

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1 active not recruiting, 1 completed, 1 terminated. 1 Phase 2, 1 Phase 3.

By phase and status

PhaseTrialsStatus
Phase 21Completed 1
Phase 31Terminated 1

Follow Tuberous Sclerosis 1: new trials by email Literature Review of Tuberous Sclerosis 1 trials Research Report on Tuberous Sclerosis 1

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
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 2CompletedAadi Bioscience, Inc.1282022-03-102026-09-28
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 3TerminatedJazz Pharmaceuticals32021-05-192025-10-31
Early Check: Expanded Screening in Newborns
Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome
Active not recruitingRTI International300002018-10-152026-07-01

Narrower conditions

Tuberous Sclerosis 2 (3)  ·  Epilepsies, Myoclonic (2)  ·  Epilepsy (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)  ·  Adrenocorticotropic hormone deficiency (1)  ·  Amaurosis congenita of Leber, type 2 (1)  ·  Anemia, Sickle Cell (1)

Broader conditions

Congenital Abnormalities (3)  ·  Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3)  ·  Genetic Diseases, Inborn (3)  ·  Heredodegenerative Disorders, Nervous System (3)  ·  Nervous System Diseases (3)  ·  Neurodegenerative Diseases (3)  ·  Pathological Conditions, Signs and Symptoms (3)  ·  Brain Diseases (2)  ·  Central Nervous System Diseases (2)  ·  Epilepsy, Generalized (2)  ·  Epileptic Syndromes (2)  ·  Neoplasms (2)  ·  Neoplasms, Multiple Primary (2)  ·  Neoplastic Syndromes, Hereditary (2)  ·  Nervous System Malformations (2)

Interventions

Cannabidiol (1)

Sponsors

Aadi Bioscience, Inc. (1)  ·  Jazz Pharmaceuticals (1)  ·  RTI International (1)

Countries

United States (3)  ·  Italy (1)  ·  Puerto Rico (1)  ·  South Korea (1)  ·  Spain (1)

Top investigators

Curt Scharfe, MD, PhD (1)

Results publications

PMID 31315600, PMID 35142618