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NCT02962414 2026-04-02

Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

Phase 3 Active not recruiting

Seizures, Tuberous Sclerosis

NCT05252585 2026-04-02

A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML)

Phase 4 Active not recruiting

Tuberous Sclerosis, Angiomyolipoma

NCT06392009 2026-04-01

Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

Phase 1/2 Active not recruiting

Focal Cortical Dysplasia, Tuberous Sclerosis

NCT05044819 2026-03-17

Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution

Phase 4 Active not recruiting

Epilepsies, Myoclonic, Liver Cirrhosis, Tuberous Sclerosis, Lennox Gastaut Syndrome

NCT05864846 2026-03-12

EpiCom

Phase 4 Active not recruiting

Behavior, Seizures, Tuberous Sclerosis

NCT05485831 2026-02-17

EpiTALY

Epilepsies, Myoclonic, Seizures, Tuberous Sclerosis, Lennox Gastaut Syndrome

NCT07403266 2026-02-11

Spectrum

Phase 3 Recruiting

Tuberous Sclerosis

NCT07369505 2026-01-27

Sapu003 in Advanced mTOR-sensitive Solid Tumors

Phase 1 Recruiting

EndocrineGastrointestinalGenitourinaryNET

Carcinoma, Renal Cell, Carcinoma, Hepatocellular, Tuberous Sclerosis, Neuroendocrine Tumors

NCT07287202 2025-12-23

Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME)

Phase 1/2 Not yet recruiting

Focal Cortical Dysplasia, Tuberous Sclerosis, Hemimegalencephaly

NCT05626634 2025-12-03

Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy

Phase 2 Completed

CDKL5 deficiency disorder, Epilepsy, Epilepsies, Myoclonic, Tuberous Sclerosis, Lennox Gastaut Syndrome

NCT03363763 2025-12-01

Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex

Phase 2 Terminated

24 enrolled 11 charts

Erythema, Exanthema, Fibroma, Tuberous Sclerosis, Facies

NCT06965881 2025-11-21

Study to Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itraconazole With Radiprodil in Healthy Adults

Phase 1 Terminated

Focal Cortical Dysplasia, Tuberous Sclerosis

NCT06975605 2025-11-18

Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs.

Phase 1 Completed

Focal Cortical Dysplasia, Tuberous Sclerosis

NCT04485104 2025-10-31

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

Phase 3 Terminated

3 enrolled 28 charts

Tuberous Sclerosis 1, Tuberous Sclerosis 2, Epilepsy, Epilepsies, Myoclonic, Seizures, Spasms, Infantile, Tuberous Sclerosis, Lennox Gastaut Syndrome

NCT05604170 2025-10-01

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

Phase 3 Terminated

117 enrolled 11 charts

Tuberous Sclerosis

NCT05323734 2025-07-11

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy

Phase 3 Completed

129 enrolled 13 charts

Tuberous Sclerosis

NCT05059327 2025-06-04

Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC

Phase 2 Completed

Seizures, Tuberous Sclerosis

NCT06149663 2025-01-22

Intermediate-Size Expanded Access Protocol (EAP) for LP352

CDKL5 deficiency disorder, Epilepsy, Epilepsies, Myoclonic, Tuberous Sclerosis, Lennox Gastaut Syndrome

NCT05495425 2024-11-22

Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC

Phase 3 Completed

Tuberous Sclerosis

NCT05364021 2024-11-08

PACIFIC

Phase 1/2 Completed

CDKL5 deficiency disorder, Epilepsy, Epilepsies, Myoclonic, Tuberous Sclerosis, Lennox Gastaut Syndrome

NCT05286632 2024-11-06

KidneYou - Innovative Digital Therapy

Phase NA Completed

Nephronophthisis, familial juvenile, Treatment Adherence and Compliance, Albuminuria, Diabetes Mellitus, Hypertension, Polycystic Kidney Diseases, Tuberous Sclerosis, Renal Insufficiency, Chronic, Kidney Diseases, Cystic

NCT01906866 2024-04-23

Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities

Phase 3 Completed

125 enrolled 22 charts

Autism Spectrum Disorder, Sleep Wake Disorders, Tuberous Sclerosis, Angelman Syndrome, Parasomnias, Smith-Magenis Syndrome

NCT03826628 2023-09-08

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

Phase 2/3 Completed

107 enrolled 24 charts

Tuberous Sclerosis

NCT04285346 2023-04-04

TSC

Phase 2 Completed

23 enrolled 9 charts

Tuberous Sclerosis

NCT02654340 2023-02-10

TuScCom

Astrocytoma, Tuberous Sclerosis, Lymphangioleiomyomatosis, Angiomyolipoma, Malformations of Cortical Development

NCT02544763 2022-09-28

A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

Phase 3 Completed

224 enrolled 18 charts

Seizures, Tuberous Sclerosis

NCT02544750 2022-07-14

An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

Phase 3 Completed

199 enrolled 14 charts

Seizures, Tuberous Sclerosis

NCT03525834 2021-09-09

Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.

Phase 4 Completed

40 enrolled 10 charts

Tuberous Sclerosis, Lymphangioleiomyomatosis, Angiomyolipoma

NCT03650452 2021-02-18

ELEKTRA

Phase 2 Completed

141 enrolled 24 charts

CDKL5 deficiency disorder, Brain Diseases, Central Nervous System Diseases, Epilepsy, Epilepsies, Myoclonic, Nervous System Diseases, Seizures, Spasms, Infantile, Tuberous Sclerosis, Hypoxia-Ischemia, Brain, Malformations of Cortical Development, Lennox Gastaut Syndrome

NCT02634931 2019-02-20

Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex

Phase 3 Completed

Tuberous Sclerosis, Angiofibroma, Plaque, Amyloid

NCT00790400 2017-02-17

EXIST-2

Phase 3 Completed

118 enrolled 15 charts

Tuberous Sclerosis 2, Astrocytoma, Hereditary Sensory and Autonomic Neuropathies, Seizures, Tuberous Sclerosis, Lymphangioleiomyomatosis, Angiomyolipoma

NCT02635789 2017-02-02

Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)

Phase 3 Completed

Tuberous Sclerosis, Angiofibroma, Plaque, Amyloid

NCT00789828 2016-02-03

EXIST-1

Phase 3 Completed

117 enrolled 15 charts

Astrocytoma, Tuberous Sclerosis

NCT01903681 2014-04-04

Assessment of the Pharmacokinetics of Circadin® in Children With Neurodevelopmental Disorders and Sleep Disturbances

Phase 1 Completed

Autism Spectrum Disorder, Tuberous Sclerosis, Angelman Syndrome, Parasomnias, Smith-Magenis Syndrome

NCT01690871 2013-08-02

A Phase II Study of Orally Administered BEZ235 Monotherapy in Patients With Metastatic or Unresectable Malignant PEComa

Phase 2 Withdrawn

Tuberous Sclerosis, Angiomyolipoma, Perivascular Epithelioid Cell Neoplasms