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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Tuberous Sclerosis Clear all
Completed Phase 1Phase 2 Interventional Results available

Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants

NCT04595513
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Epilepsy
Interventions
TAVT-18 (sirolimus)
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
1 Day to 6 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated May 15, 2024 · Synced Sep 25, 2026, 12:55 AM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

TANDem-2: Closing the Gap to Interventions for TAND

NCT06879665
View directory record Official record
Conditions
Adult Caregivers of Individuals With TSC, Tuberous Sclerosis Complex (TSC)
Interventions
Well-Beans for Caregivers Program
Behavioral
Lead sponsor
Vrije Universiteit Brussel
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2027
U.S. locations
1
States / cities
Silver Spring, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 21, 2025 · Synced Sep 25, 2026, 12:55 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 25, 2026, 12:55 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 30, 2025 · Synced Sep 25, 2026, 12:55 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 24, 2013 · Synced Sep 25, 2026, 12:55 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 22, 2026 · Synced Sep 25, 2026, 12:55 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 25, 2026, 12:55 AM EDT
Completed Phase 3 Interventional Results available

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

NCT05218655
View directory record Official record
Conditions
Inherited Mitochondrial Disease
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
101 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
14
States / cities
La Jolla, California • Stanford, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2025 · Synced Sep 25, 2026, 12:55 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 25, 2020 · Synced Sep 25, 2026, 12:55 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 25, 2026, 12:55 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 25, 2026, 12:55 AM EDT
Completed Phase 2 Interventional Results available

A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies

NCT03650452
View directory record Official record
Conditions
Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
Interventions
TAK-935, Placebo
Drug
Lead sponsor
Takeda
Industry
Eligibility
2 Years to 17 Years
Enrollment
141 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
15
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2026 · Synced Sep 25, 2026, 12:55 AM 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 25, 2026, 12:55 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

NCT03826628
View directory record Official record
Conditions
Facial Angiofibroma, Tuberous Sclerosis
Interventions
rapamycin, placebo
Drug
Lead sponsor
Dermatology Specialties Limited Partnership
Industry
Eligibility
6 Years to 65 Years
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
6
States / cities
Phoenix, Arizona • La Jolla, California • St. Petersburg, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 7, 2023 · Synced Sep 25, 2026, 12:55 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 25, 2026, 12:55 AM 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 25, 2026, 12:55 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 3, 2025 · Synced Sep 25, 2026, 12:55 AM 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 10, 2026 · Synced Sep 25, 2026, 12:55 AM 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 26, 2012 · Synced Sep 25, 2026, 12:55 AM EDT
Completed No phase listed Observational

Potential EEG Biomarkers and Antiepileptogenic Strategies for Epilepsy in TSC

NCT01767779
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
1 Day to 6 Months
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
5
States / cities
Birmingham, Alabama • Los Angeles, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 10, 2019 · Synced Sep 25, 2026, 12:55 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 20, 2026 · Synced Sep 25, 2026, 12:55 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex

NCT00411619
View directory record Official record
Conditions
Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
Interventions
Everolimus
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
3 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2014
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 19, 2014 · Synced Sep 25, 2026, 12:55 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 17, 2013 · Synced Sep 25, 2026, 12:55 AM 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 25, 2026, 12:55 AM EDT