21 completed, 7 unknown, 6 active not recruiting, 5 recruiting. 16 Phase 2, 9 Phase 3, 5 Phase 1, 4 Not applicable, 1 Phase 4.
Follow Tuberous Sclerosis Complex: new trials by email Literature Review of Tuberous Sclerosis Complex trials Research Report on Tuberous Sclerosis Complex
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 |
|---|---|---|---|---|---|---|
| Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1) AV078, Placebo - Tuberous Sclerosis Complex, Epilepsy | Phase 2 | Not yet recruiting | Aeovian Pharmaceuticals, Inc. | 42 | 2026-09-01 | 2026-09-29 |
| A Smart Phone Application to Improve Adoption of the 2024 Kidney Disease Improving Global Outcomes (KDIGO) Chronic Kidney Disease (CKD) Guidelines Use of an electronic digital Application - Chronic Kidney Disease, Proteinuria | Not applicable | Recruiting | St. James's Hospital, Ireland | 80 | 2026-01-14 | 2026-05-01 |
| Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY Epidiolex 100 mg/mL Oral Solution - Lennox Gastaut Syndrome, Dravet Syndrome | Active not recruiting | Jazz Pharmaceuticals | 111 | 2025-02-24 | 2026-06-03 | |
| Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II Radiprodil - Tuberous Sclerosis Complex, Focal Cortical Dysplasia | Phase 1 / Phase 2 | Active not recruiting | GRIN Therapeutics, Inc. | 30 | 2024-07-10 | 2026-04-01 |
| Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry) Tuberous Sclerosis Complex, Lymphangioleiomyomatosis | Recruiting | David M. Ritter | 300 | 2023-07-01 | 2026-01-23 | |
| Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex Rapamycin, Placebo - Tuberous Sclerosis Complex | Phase 3 | Recruiting | Katarzyna Kotulska | 200 | 2023-01-23 | 2026-10-22 |
| Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSC NPC-12Y gel, NPC-12Y placebo gel - Tuberous Sclerosis Complex | Phase 3 | Completed | Nobelpharma | 43 | 2022-06-01 | 2024-11-22 |
| Lymphangioleiomyomatosis, a Study on Cathepsin K 24h urine, urine sample - Lymphangioleiomyomatosis, Tuberous Sclerosis Complex | Completed | University Hospital, Tours | 19 | 2022-05-31 | 2023-03-29 | |
| Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy Ganaxolone - Tuberous Sclerosis Complex | Phase 3 | Terminated | Marinus Pharmaceuticals | 117 | 2022-05-16 | 2025-10-01 |
| Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy Ganaxolone, Placebo - Tuberous Sclerosis Complex | Phase 3 | Completed | Marinus Pharmaceuticals | 129 | 2022-04-01 | 2025-07-11 |
| Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC Basimglurant with crossover to Placebo, Placebo with crossover to Basimglurant - Tuberous Sclerosis Complex | Phase 2 | Completed | Noema Pharma AG | 61 | 2022-03-03 | 2025-06-04 |
| Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study Sirolimus, Placebo - Tuberous Sclerosis Complex, Epilepsy | Phase 2 | Recruiting | Darcy Krueger | 64 | 2021-10-13 | 2026-10-02 |
| Feasibility of [11C]Acetate-PET in LAM and TSC [11C]acetate - Lymphangioleiomyomatosis, Tuberous Sclerosis Complex | Phase 1 / Phase 2 | Active not recruiting | Brigham and Women's Hospital | 7 | 2021-08-30 | 2026-01-21 |
| Acceptance and Commitment Therapy in Tuberous Sclerosis Complex Acceptance and Commitment Therapy (ACT) - Tuberous Sclerosis Complex | Not applicable | Unknown | University Hospitals Bristol and Weston NHS Foundation Trust | 15 | 2021-08-12 | 2024-02-14 |
| Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution Cannabidiol - Lennox Gastaut Syndrome, Dravet Syndrome | Phase 4 | Active not recruiting | Jazz Pharmaceuticals | 154 | 2021-07-07 | 2026-08-26 |
| Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants Vigabatrin, Rapamycin, Placebo - Tuberous Sclerosis Complex | Phase 2 / Phase 3 | Recruiting | Katarzyna Kotulska | 60 | 2021-05-07 | 2026-10-22 |
| Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants TAVT-18 (sirolimus) - Tuberous Sclerosis Complex, Epilepsy | Phase 1 / Phase 2 | Completed | Children's Hospital Medical Center, Cincinnati | 5 | 2020-09-08 | 2024-05-16 |
| The Effectiveness and Safety of Resective Epilepsy Surgery for TRE Surgery - Tuberous Sclerosis Complex, Epilepsy | Active not recruiting | Beijing Children's Hospital | 200 | 2019-12-12 | 2025-01-17 | |
| The Effectiveness and Safety of Vagus Nerve Stimulation for TRE Tuberous Sclerosis Complex, Epilepsy | Completed | Beijing Children's Hospital | 70 | 2019-12-10 | 2024-12-19 | |
| Dermatologic Patterns of Tuberous Sclerosis Patients and Somatic Mutation Relationship Tuberous Sclerosis Complex | Unknown | University Hospital, Montpellier | 90 | 2019-03-01 | 2020-03-10 |
Showing 20 of 41. Sign in to see all of them.
Tuberous Sclerosis (41) · Epilepsy (12) · Seizures (8) · Autistic Disorder (4) · Lymphangioleiomyomatosis (4) · Drug Resistant Epilepsy (2) · Epilepsies, Myoclonic (2) · Focal Cortical Dysplasia (2) · Lennox Gastaut Syndrome (2) · Cognitive Dysfunction (1) · Hereditary Sensory and Autonomic Neuropathies (1) · Intellectual Disability (1) · Kidney Diseases (1) · Liver Cirrhosis (1) · Neoplasms (1)
Congenital Abnormalities (41) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (41) · Genetic Diseases, Inborn (41) · Hamartoma (41) · Heredodegenerative Disorders, Nervous System (41) · Malformations of Cortical Development (41) · Malformations of Cortical Development, Group I (41) · Neoplasms, Multiple Primary (41) · Neoplastic Syndromes, Hereditary (41) · Nervous System Diseases (41) · Nervous System Malformations (41) · Neurodegenerative Diseases (41) · Neoplasms (40) · Neurocutaneous Syndromes (40) · Brain Diseases (14)
Everolimus (8) · Sirolimus (6) · Cannabidiol (4) · Vigabatrin (2) · ganaxolone (2) · 2-chloro-4-(1-(4-fluorophenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl)pyridine (1) · Acceptance and Commitment Therapy (1) · Activator Appliances (1) · Anticonvulsants (1) · Aspirin (1) · Fluid Therapy (1) · Simvastatin (1) · Solutions (1) · Surgical Procedures, Operative (1) · Urination (1)
Jazz Pharmaceuticals (4) · Beijing Children's Hospital (3) · Children's Hospital Medical Center, Cincinnati (3) · Erasmus Medical Center (2) · Katarzyna Kotulska (2) · Marinus Pharmaceuticals (2) · Aeovian Pharmaceuticals, Inc. (1) · Boston Children's Hospital (1) · Brigham and Women's Hospital (1) · Dana-Farber Cancer Institute (1) · Darcy Krueger (1) · David M. Ritter (1) · GRIN Therapeutics, Inc. (1) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. (1) · Martina Bebin (1)
United States (23) · France (7) · Italy (7) · Poland (7) · United Kingdom (7) · Spain (6) · Australia (5) · Canada (5) · China (5) · Netherlands (5) · Belgium (3) · Germany (3) · Israel (3) · Japan (2) · Turkey (Türkiye) (2)
Darcy Krueger, MD, PhD (2) · Martina Bebin, MD, MPA (2) · Mustafa Sahin, MD, PhD (2) · Carmen P Priolo, MD PhD (1) · Darcy A Krueger, MD, PhD (1) · Darcy Krueger, M.D. Ph.D (1) · David Kwiatkowski, MD, PhD (1) · David M Ritter, MD, PhD (1) · Donal Sexton, MB BCh BAO BSc, MRCPI, MD, PhD (1) · Frank McCormack, MD (1) · John J Bissler, M.D. (1) · Katarzyna Kotulska-Jozwiak (1)
PMID 31912454, PMID 34957550, PMID 38996393, PMID 21482669, PMID 23798472, PMID 29572283, PMID 31217257, PMID 33346789, PMID 35175622, PMID 35587487, PMID 37638552, PMID 38441854, PMID 39331837, PMID 39625912, PMID 41100778
2015 (2), 2023 (1), 2025 (1), U01NS082320 (1), U01NS092595 (1)