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Showing 1–24 of 73 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Tuberous Sclerosis Clear all
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 5, 2026, 5:56 AM EDT
Completed Phase 1 Interventional

Tuberous Sclerosis Complex: Facial Angiofibroma Skin Cream

NCT01853423
View directory record Official record
Conditions
Facial Angiofibroma
Interventions
Rapamune
Drug
Lead sponsor
Le Bonheur Children's Hospital
Other
Eligibility
3 Years to 45 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 18, 2016 · Synced Sep 5, 2026, 5:56 AM 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 5, 2026, 5:56 AM EDT
Recruiting Phase 1Phase 2 Interventional

Assessing Changes in Multi-parametric MRI in MS Patients Taking Clemastine Fumarate as a Myelin Repair Therapy

NCT05359653
View directory record Official record
Conditions
Multiple Sclerosis (MS), Multiple Sclerosis, Relapsing-Remitting, Multiple Sclerosis, Primary Progressive, Multiple Sclerosis, Chronic Progressive, Multiple Sclerosis Relapse, Multiple Sclerosis Brain Lesion, Multiple Sclerosis Benign
Interventions
Clemastine Fumarate, Placebo
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 55 Years
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Sep 5, 2026, 5:56 AM EDT
Active, not recruiting Phase 4 Interventional

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

NCT05044819
View directory record Official record
Conditions
Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
Interventions
Cannabidiol
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year and older
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
26
States / cities
Little Rock, Arkansas • Downey, California • Long Beach, California + 22 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Sep 5, 2026, 5:56 AM EDT
Recruiting No phase listed Observational

TSC Biosample Repository and Natural History Database

NCT05676099
View directory record Official record
Conditions
Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
Phlebotomy, Buccal (cheek) swab, Genetic Testing, Tissue donation after routine clinical procedure
Procedure · Genetic · Other
Lead sponsor
National Tuberous Sclerosis Association
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2050
U.S. locations
24
States / cities
Birmingham, Alabama • Loma Linda, California • Los Angeles, California + 19 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Sep 5, 2026, 5:56 AM EDT
Completed Phase 3 Interventional Results available

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

NCT02544763
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Seizures
Interventions
GWP42003-P, Placebo
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year to 65 Years
Enrollment
224 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
23
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Los Angeles, California + 20 more
Source: ClinicalTrials.gov public record
Updated Sep 27, 2022 · Synced Sep 5, 2026, 5:56 AM EDT
Terminated Phase 4 Interventional Results available

Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients

NCT01266291
View directory record Official record
Conditions
Complex Partial Seizures
Interventions
vigabatrin
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 11, 2017 · Synced Sep 5, 2026, 5:56 AM EDT
Withdrawn Not applicable Interventional

Turmeric as Treatment in Epilepsy

NCT03254680
View directory record Official record
Conditions
Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome, Tuberous Sclerosis, Focal Seizures
Interventions
Turmeric
Dietary Supplement
Lead sponsor
NYU Langone Health
Other
Eligibility
1 Year to 70 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 29, 2018 · Synced Sep 5, 2026, 5:56 AM EDT
Terminated Phase 2 Interventional Results available

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

NCT03363763
View directory record Official record
Conditions
Angiofibroma of Face, Tuberous Sclerosis
Interventions
Sirolimus 0.2%, Sirolimus 0.4%, Placebo ointment
Drug
Lead sponsor
Aucta Pharmaceuticals, Inc
Industry
Eligibility
2 Years to 21 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
7
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 30, 2025 · Synced Sep 5, 2026, 5:56 AM 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 5, 2026, 5:56 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)

NCT02461459
View directory record Official record
Conditions
Tuberous Sclerosis, Autism Disorder, Intellectual Disability
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Months and older
Enrollment
205 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2026
U.S. locations
6
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Sep 5, 2026, 5:56 AM EDT
Completed Phase 4 Interventional

A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex

NCT05864846
View directory record Official record
Conditions
Tuberous Sclerosis Complex Associated Neuropsychiatric Disease
Interventions
Cannabidiol Oral Solution [Epidiolex]
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year to 65 Years
Enrollment
79 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
12
States / cities
Aurora, Colorado • New Haven, Connecticut • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced Sep 5, 2026, 5:56 AM 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 5, 2026, 5:56 AM EDT
Completed No phase listed Observational

Studies in Patients With Tuberous Sclerosis Complex

NCT03276195
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
Translational Genomics Research Institute
Other
Eligibility
Not listed
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 28, 2023 · Synced Sep 5, 2026, 5:56 AM 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 5, 2026, 5:56 AM EDT
Available No phase listed Expanded access

Intermediate-Size Expanded Access Protocol (EAP) for LP352

NCT06149663
View directory record Official record
Conditions
Dravet Syndrome, Lennox Gastaut Syndrome, Developmental and Epileptic Encephalopathies
Interventions
LP352
Drug
Lead sponsor
Longboard Pharmaceuticals
Industry
Eligibility
2 Years to 65 Years
U.S. locations
19
States / cities
Downey, California • Los Angeles, California • San Francisco, California + 15 more
Source: ClinicalTrials.gov public record
Updated Jan 21, 2025 · Synced Sep 5, 2026, 5:56 AM 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 5, 2026, 5:56 AM 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 5, 2026, 5:56 AM 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 5, 2026, 5:56 AM EDT
Completed Phase 2 Interventional

Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

NCT00457808
View directory record Official record
Conditions
Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
Rapamycin, sirolimus
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 65 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2006
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 8, 2007 · Synced Sep 5, 2026, 5:56 AM 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 5, 2026, 5:56 AM 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 5, 2026, 5:56 AM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Parent and Infant Inter(X)Action Intervention (PIXI)

NCT03836300
View directory record Official record
Conditions
Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions
Parent-Infant Inter(X)action Intervention (PIXI)
Behavioral
Lead sponsor
RTI International
Other
Eligibility
Up to 99 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2026
U.S. locations
1
States / cities
Research Triangle Park, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 5, 2026, 5:56 AM EDT