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ClinicalTrials.gov public records Last synced Oct 21, 2026, 8:32 AM EDT

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Showing 1–24 of 66 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Tuberous Sclerosis Complex Clear all
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 28, 2022 · Synced Oct 21, 2026, 8:32 AM 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 21, 2026, 8:32 AM EDT
Active, not recruiting No phase listed Observational

ESIS in Pediatric DRE

NCT05469373
View directory record Official record
Conditions
Epilepsy
Interventions
Not listed
Lead sponsor
Sarah Katie Ihnen
Other
Eligibility
1 Year to 30 Years
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 21, 2026, 8:32 AM EDT
Completed Phase 2 Interventional Results available

Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)

NCT01289912
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
RAD001, Placebo
Drug
Lead sponsor
Mustafa Sahin
Other
Eligibility
6 Years to 21 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
Boston, Massachusetts • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 25, 2018 · Synced Oct 21, 2026, 8:32 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 22, 2025 · Synced Oct 21, 2026, 8:32 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 24, 2026 · Synced Oct 21, 2026, 8:32 AM EDT
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 31, 2025 · Synced Oct 21, 2026, 8:32 AM EDT
Completed Phase 2 Interventional Results available

Everolimus for Cancer With TSC1 or TSC2 Mutation

NCT02201212
View directory record Official record
Conditions
TSC1, TSC2, Tuberous Sclerosis Complex, MTOR
Interventions
Everolimus
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 16, 2020 · Synced Oct 21, 2026, 8:32 AM EDT
Completed Phase 2 Interventional Results available

RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS

NCT00126672
View directory record Official record
Conditions
Nonmalignant Neoplasm, Tuberous Sclerosis, Lymphangioleimyomatosis, Kidney Angiomyolipoma
Interventions
Rapamycin
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
3 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
7
States / cities
Loma Linda, California • Hartford, Connecticut • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Oct 21, 2026, 8:32 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 17, 2017 · Synced Oct 21, 2026, 8:32 AM EDT
Completed Phase 1Phase 2 Interventional

RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM

NCT00457964
View directory record Official record
Conditions
Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
RAD001
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 25, 2013 · Synced Oct 21, 2026, 8:32 AM 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 21, 2026, 8:32 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 Dec 1, 2025 · Synced Oct 21, 2026, 8:32 AM 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 21, 2026, 8:32 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 6, 2026 · Synced Oct 21, 2026, 8:32 AM 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 21, 2026, 8:32 AM 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 21, 2026, 8:32 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 4, 2017 · Synced Oct 21, 2026, 8:32 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 10, 2023 · Synced Oct 21, 2026, 8:32 AM EDT
Completed Phase 3 Interventional Results available

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

NCT02544750
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Seizures
Interventions
GWP42003-P
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year to 65 Years
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 14, 2022 · Synced Oct 21, 2026, 8:32 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Feasibility of [11C]Acetate-PET in LAM and TSC

NCT05467397
View directory record Official record
Conditions
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Interventions
[11C]acetate
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older · Female only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 21, 2026, 8:32 AM EDT
Recruiting No phase listed Observational

Study of Skin Tumors in Tuberous Sclerosis

NCT00001975
View directory record Official record
Conditions
Tuberous Sclerosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 90 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 21, 2026, 8:32 AM 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 21, 2026, 8:32 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)

NCT01070316
View directory record Official record
Conditions
Epilepsy, Tuberous Sclerosis Complex
Interventions
Everolimus
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
2 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
2
States / cities
Cincinnati, Ohio • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 13, 2017 · Synced Oct 21, 2026, 8:32 AM EDT