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ClinicalTrials.gov public records Last synced Oct 20, 2026, 2:27 PM EDT

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Showing 1–24 of 73 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Tuberous Sclerosis Clear all
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 17, 2017 · Synced Oct 20, 2026, 2:27 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)

NCT01031901
View directory record Official record
Conditions
Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma
Interventions
Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin
Drug
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
13 Years and older
Enrollment
52 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 27, 2012 · Synced Oct 20, 2026, 2:27 PM EDT
Completed Phase 1Phase 2 Interventional

Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)

NCT00792766
View directory record Official record
Conditions
Tuberous Sclerosis, Angiolipoma
Interventions
everolimus (RAD001)
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 18, 2013 · Synced Oct 20, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex

NCT02849457
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Early Vigabatrin, Delayed Vigabatrin (Placebo)
Drug
Lead sponsor
Martina Bebin
Other
Eligibility
1 Day to 6 Months
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
13
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2024 · Synced Oct 20, 2026, 2:27 PM 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 27, 2026 · Synced Oct 20, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

Topical Rapamycin to Erase Angiofibromas in TSC

NCT01526356
View directory record Official record
Conditions
Angiofibromas, Tuberous Sclerosis
Interventions
Placebo, Rapamycin
Drug
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
Not listed
Enrollment
179 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
9
States / cities
Birmingham, Alabama • Los Angeles, California • Oakland, California + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 26, 2020 · Synced Oct 20, 2026, 2:27 PM EDT
Recruiting No phase listed Observational

Study of the Disease Process of Lymphangioleiomyomatosis

NCT00001465
View directory record Official record
Conditions
Lung Disease, Pneumothorax, Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
Toshibia Aquilion One CT
Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
16 Years to 100 Years
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 20, 2026, 2:27 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Assessing Changes in Multi-parametric MRI in Patients With Acute Demyelinating Lesions Taking Clemastine Fumarate as a Myelin Repair Therapy

NCT06065670
View directory record Official record
Conditions
Demyelinating Diseases, Demyelination; Corpus Callosum, Multiple Sclerosis Brain Lesion, Multiple Sclerosis Acute and Progressive, Clinically Isolated Syndrome, CNS Demyelinating
Interventions
Clemastine Fumarate, Placebo
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 55 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced Oct 20, 2026, 2:27 PM 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 26, 2026 · Synced Oct 20, 2026, 2:27 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Role of Genetic Factors in the Development of Lung Disease

NCT00001532
View directory record Official record
Conditions
Cystic Fibrosis, Pulmonary Fibrosis, Tuberous Sclerosis, Asthma, Pulmonary Sarcoidosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 90 Years
Enrollment
3,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1996
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 20, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD

NCT02451696
View directory record Official record
Conditions
Epilepsy, Tuberous Sclerosis Complex, Focal Cortical Dysplasia
Interventions
Everolimus
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
2 Years to 40 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 1, 2021 · Synced Oct 20, 2026, 2:27 PM 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 29, 2023 · Synced Oct 20, 2026, 2:27 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 3, 2025 · Synced Oct 20, 2026, 2:27 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 31, 2026 · Synced Oct 20, 2026, 2:27 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 6, 2020 · Synced Oct 20, 2026, 2:27 PM EDT
Completed Not applicable Interventional

Early Behavioral Intervention to Improve Social Communication Function in Infants With Tuberous Sclerosis Complex

NCT02687633
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
JASPER
Behavioral
Lead sponsor
University of California, Los Angeles
Other
Eligibility
12 Months to 36 Months
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Oct 20, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)

NCT04285346
View directory record Official record
Conditions
Tuberous Sclerosis
Interventions
Ganaxolone
Drug
Lead sponsor
Marinus Pharmaceuticals
Industry
Eligibility
2 Years to 65 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
7
States / cities
Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 4, 2023 · Synced Oct 20, 2026, 2:27 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 – 2028
U.S. locations
11
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 20, 2026, 2:27 PM EDT
Completed Phase 2 Interventional

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

NCT05059327
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Basimglurant with crossover to Placebo, Placebo with crossover to Basimglurant
Drug
Lead sponsor
Noema Pharma AG
Industry
Eligibility
5 Years to 30 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
9
States / cities
Los Angeles, California • Baltimore, Maryland • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2025 · Synced Oct 20, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

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
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
159
States / cities
Birmingham, Alabama • Mobile, Alabama • Goodyear, Arizona + 137 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 20, 2026, 2:27 PM EDT
Terminated Phase 3 Interventional Results available

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

NCT05604170
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Ganaxolone
Drug
Lead sponsor
Marinus Pharmaceuticals
Industry
Eligibility
1 Year to 65 Years
Enrollment
117 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
20
States / cities
Little Rock, Arkansas • Los Angeles, California • Orange, California + 17 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2025 · Synced Oct 20, 2026, 2:27 PM 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 24, 2026 · Synced Oct 20, 2026, 2:27 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 10, 2023 · Synced Oct 20, 2026, 2:27 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 30, 2024 · Synced Oct 20, 2026, 2:27 PM EDT