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Showing 1–24 of 87 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: TSC Clear all
Terminated Phase 3 Interventional Results available

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

NCT04485104
View directory record Official record
Conditions
Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome, Seizure in Participants With Lennox-Gastaut Syndrome
Interventions
GWP42003-P
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Month to 23 Months
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
6
States / cities
Little Rock, Arkansas • Los Angeles, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Sep 3, 2026, 1:31 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety of Simvastatin in LAM and TSC

NCT02061397
View directory record Official record
Conditions
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Interventions
Simvastatin, Sirolimus Oral Product, Everolimus Oral Product
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 24, 2020 · Synced Sep 3, 2026, 1:31 PM EDT
Active, not recruiting Not applicable Interventional

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

NCT06139172
View directory record Official record
Conditions
Telomeric 22Q13 Monosomy Syndrome, Tuberous Sclerosis, Hamartoma Syndrome, Multiple, Fragile X Syndrome, Angelman Syndrome, Rett Syndrome, Chromosome 15Q, Partial Deletion, Creatine Deficiency, X-linked
Interventions
Functional Behavioral Training (FBT), Positive Parenting Strategies-Treatment as Usual
Behavioral · Other
Lead sponsor
Rush University Medical Center
Other
Eligibility
2 Years to 12 Years
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 3, 2026, 1:31 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy of Trans Sodium Crocetinate (TSC) With Radiation and Temozolomide in Newly Diagnosed Glioblastoma

NCT01465347
View directory record Official record
Conditions
Glioblastoma, GBM, Glioma
Interventions
Trans Sodium Crocetinate (TSC)
Drug
Lead sponsor
Diffusion Pharmaceuticals Inc
Industry
Eligibility
18 Years and older
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
18
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 13, 2017 · Synced Sep 3, 2026, 1:31 PM EDT
Completed Phase 1Phase 2 Interventional

Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies

NCT05364021
View directory record Official record
Conditions
Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
Interventions
LP352, Placebo
Drug
Lead sponsor
Longboard Pharmaceuticals
Industry
Eligibility
12 Years to 65 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
29
States / cities
Tucson, Arizona • Little Rock, Arkansas • Downey, California + 24 more
Source: ClinicalTrials.gov public record
Updated Nov 7, 2024 · Synced Sep 3, 2026, 1:31 PM EDT
Completed Phase 2 Interventional

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

NCT05626634
View directory record Official record
Conditions
Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox-Gastaut Syndrome
Interventions
LP352, bexicaserin
Drug
Lead sponsor
Longboard Pharmaceuticals
Industry
Eligibility
12 Years to 65 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
25
States / cities
Little Rock, Arkansas • Downey, California • San Francisco, California + 20 more
Source: ClinicalTrials.gov public record
Updated Dec 2, 2025 · Synced Sep 3, 2026, 1:31 PM EDT
Completed Not applicable Interventional

TSC Remote Assessment and Intervention

NCT04698538
View directory record Official record
Conditions
TSC
Interventions
JASPER
Behavioral
Lead sponsor
University of California, Los Angeles
Other
Eligibility
12 Months to 36 Months
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Oct 29, 2024 · Synced Sep 3, 2026, 1:31 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

NCT00790400
View directory record Official record
Conditions
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
Interventions
Everolimus (RAD001), Everolimus Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
118 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
6
States / cities
Birmingham, Alabama • Phoenix, Arizona • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2017 · Synced Sep 3, 2026, 1:31 PM EDT
Completed No phase listed Observational

Tuberous Sclerosis Complex Natural History Study: Renal Manifestations

NCT00598455
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
7 Years to 65 Years
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated May 3, 2017 · Synced Sep 3, 2026, 1:31 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Compare Treatment With the Drug Selumetinib Alone Versus Selumetinib and Vinblastine in Patients With Recurrent or Progressive Low-Grade Glioma

NCT04576117
View directory record Official record
Conditions
Recurrent Low Grade Astrocytoma, Recurrent WHO Grade 2 Glioma, Refractory Low Grade Astrocytoma, Refractory Low Grade Glioma, Refractory WHO Grade 1 Glioma
Interventions
Biospecimen Collection, Magnetic Resonance Imaging, Quality-of-Life Assessment, Questionnaire Administration, Selumetinib Sulfate, Vinblastine Sulfate
Procedure · Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 25 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
91
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Loma Linda, California + 77 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Sep 3, 2026, 1:31 PM EDT
Completed Phase 3 Interventional Results available

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

NCT01906866
View directory record Official record
Conditions
Sleep Disorders
Interventions
Circadin 2/5/10 mg, Placebo
Drug
Lead sponsor
Neurim Pharmaceuticals Ltd.
Industry
Eligibility
2 Years to 17 Years
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
18
States / cities
Phoenix, Arizona • Miami Lakes, Florida • Orange City, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 22, 2024 · Synced Sep 3, 2026, 1:31 PM EDT
Not listed Not applicable Interventional

JASPER Early Intervention for Tuberous Sclerosis

NCT03422367
View directory record Official record
Conditions
Tuberous Sclerosis
Interventions
JASPER
Behavioral
Lead sponsor
University of California, Los Angeles
Other
Eligibility
6 Months to 40 Months
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
2
States / cities
Los Angeles, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 5, 2020 · Synced Sep 3, 2026, 1:31 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)

NCT00789828
View directory record Official record
Conditions
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Interventions
Everolimus, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
117 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
12
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 2, 2016 · Synced Sep 3, 2026, 1:31 PM EDT
Completed Phase 2 Interventional Results available

COLA: A Pilot Clinical Trial of COX-2 Inhibition in LAM and TSC

NCT02484664
View directory record Official record
Conditions
Lymphangioleiomyomatosis (LAM)
Interventions
Celecoxib
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 70 Years · Female only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 19, 2022 · Synced Sep 3, 2026, 1:31 PM EDT
Recruiting Phase 2 Interventional

Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study

NCT05104983
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Epilepsy
Interventions
Sirolimus, Placebo
Drug
Lead sponsor
Darcy Krueger
Other
Eligibility
1 Day to 6 Months
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
11
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Sep 3, 2026, 1:31 PM EDT
Withdrawn Not applicable Interventional

Use of a Tonometer to Identify Epileptogenic Lesions During Pediatric Epilepsy Surgery

NCT04344626
View directory record Official record
Conditions
Epilepsy, Focal Cortical Dysplasia, Tuberous Sclerosis, Hemimegalencephaly, Polymicrogyria, Rasmussen Encephalitis, Sturge-Weber Syndrome, Gliosis, Stroke, Tumor, Brain
Interventions
Intra-operative brain tonometry
Device
Lead sponsor
University of California, Los Angeles
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Oct 9, 2023 · Synced Sep 3, 2026, 1:31 PM EDT
Terminated Phase 2 Interventional

Study of Trans Sodium Crocetinate in Patients With Interstitial Lung Disease

NCT05079126
View directory record Official record
Conditions
Interstitial Lung Disease
Interventions
Trans Sodium Crocetinate, Placebo
Drug
Lead sponsor
Diffusion Pharmaceuticals Inc
Industry
Eligibility
30 Years to 85 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Aug 9, 2022 · Synced Sep 3, 2026, 1:31 PM EDT
Active, not recruiting Phase 2 Interventional

Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)

NCT05103358
View directory record Official record
Conditions
Tumor, Tumor, Solid, Metastasis, Metastatic Cancer, Cancer, Cancer Metastatic, Tumors, Neoplasms, Neoplasm Metastasis, Solid Tumor, Advanced Solid Tumor, Advanced Cancer, Malignant Solid Tumor, Malignant Solid Neoplasm, Malignant Neoplasm, Malignant Tumor, TSC, TSC1, TSC2, Metastatic Solid Tumor, Metastatic Neoplasm
Interventions
nab-sirolimus
Drug
Lead sponsor
Aadi Bioscience, Inc.
Industry
Eligibility
12 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
159
States / cities
Birmingham, Alabama • Mobile, Alabama • Goodyear, Arizona + 137 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2024 · Synced Sep 3, 2026, 1:31 PM EDT
Completed Phase 3 Interventional Results available

A Placebo-controlled Study of Efficacy & Safety of 2 Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Partial-onset Seizures

NCT01713946
View directory record Official record
Conditions
Tuberous Sclerosis Complex-associated Refractory Seizures
Interventions
RAD001, Placebo, Antiepileptic drug (1 to 3 only), open label RAD001 (only used for post-extension phase)
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 65 Years
Enrollment
366 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
22
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 18 more
Source: ClinicalTrials.gov public record
Updated Nov 6, 2018 · Synced Sep 3, 2026, 1:31 PM EDT
Recruiting No phase listed Observational

Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)

NCT06160310
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Lymphangioleiomyomatosis
Interventions
Not listed
Lead sponsor
David M. Ritter
Other
Eligibility
Female only
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Sep 3, 2026, 1:31 PM EDT
Terminated Phase 2Phase 3 Interventional Results available

A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

NCT04378075
View directory record Official record
Conditions
Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome, Mitochondrial Encephalopathy (MELAS), Pontocerebellar Hypoplasia Type 6 (PCH6), Alpers Disease, Alpers Syndrome
Interventions
Vatiquinone, Placebo
Drug · Other
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 20 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
12
States / cities
San Diego, California • Stanford, California • New Haven, Connecticut + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2026 · Synced Sep 3, 2026, 1:31 PM EDT
Recruiting No phase listed Observational

Familial Investigations of Childhood Cancer Predisposition

NCT03050268
View directory record Official record
Conditions
Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML, BAP1 Tumor Predisposition Syndrome, Carney Complex, Choroid Plexus Carcinoma, Constitutional Mismatch Repair Deficiency Syndrome, Diamond-Blackfan Anemia, DICER1 Syndrome, Dyskeratosis Congenita, Emberger Syndrome, Familial Acute Myeloid Leukemia, Familial Adenomatous Polyposis, Fanconi Anemia, Familial Cancer, Familial Wilms Tumor, Familial Neuroblastoma, GIST, Hereditary Breast and Ovarian Cancer, Hereditary Paraganglioma-Pheochromocytoma Syndrome, Hodgkin Lymphoma, Juvenile Polyposis, Li-Fraumeni Syndrome, Lynch Syndrome, MDS, Melanoma Syndrome, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Neuroblastoma, Neurofibromatosis Type 1, Neurofibromatosis Type II, Nevoid Basal Cell Carcinoma Syndrome, Non Hodgkin Lymphoma, Noonan Syndrome and Other Rasopathy, Overgrowth Syndromes, Pancreatic Cancer, Peutz-Jeghers Syndrome, Pheochromocytoma/Paraganglioma, PTEN Hamartoma Tumor Syndrome, Retinoblastoma, Rhabdoid Tumor Predisposition Syndrome, Rhabdomyosarcoma, Rothmund-Thomson Syndrome, Tuberous Sclerosis, Von Hippel-Lindau Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Sep 3, 2026, 1:31 PM EDT
Completed Phase 2 Interventional Results available

Rapalogues for Autism Phenotype in TSC: A Feasibility Study

NCT01929642
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Self-injury, Autism
Interventions
Sirolimus, Everolimus
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
2 Years to 30 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 28, 2021 · Synced Sep 3, 2026, 1:31 PM EDT
Completed No phase listed Observational

Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

NCT01780441
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
3 Months to 12 Months
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
5
States / cities
Birmingham, Alabama • Los Angeles, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 3, 2021 · Synced Sep 3, 2026, 1:31 PM EDT