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ClinicalTrials.gov public records Last synced Jun 26, 2026, 5:48 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: TSC Clear all
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 Jun 26, 2026, 5:48 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 Jun 26, 2026, 5:48 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Safety and Efficacy of Trans Sodium Crocetinate (TSC) in Enhancing Tissue Oxygen Levels in Healthy Volunteers

NCT04808622
View directory record Official record
Conditions
Healthy Subjects
Interventions
Trans-Sodium Crocetinate, Placebo
Drug
Lead sponsor
Diffusion Pharmaceuticals Inc
Industry
Eligibility
18 Years to 50 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Jul 10, 2023 · Synced Jun 26, 2026, 5:48 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 Jun 26, 2026, 5:48 AM EDT
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 Jun 26, 2026, 5:48 AM 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 3, 2023 · Synced Jun 26, 2026, 5:48 AM EDT
Completed Phase 1Phase 2 Interventional

Safety, Efficacy, and Pharmacokinetics (PK) Study of Trans Sodium Crocetinate (TSC) in Patients With Intermittent Claudication

NCT00725881
View directory record Official record
Conditions
Intermittent Claudication
Interventions
Trans sodium crocetinate (TSC), 0.9% normal saline
Drug
Lead sponsor
Diffusion Pharmaceuticals Inc
Industry
Eligibility
40 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
7
States / cities
Birmingham, Alabama • Santa Rosa, California • Stanford, California + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2011 · Synced Jun 26, 2026, 5:48 AM 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 Jun 26, 2026, 5:48 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 Jun 26, 2026, 5:48 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 Jun 26, 2026, 5:48 AM EDT
Completed Phase 1 Interventional Results available

Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis

NCT01687179
View directory record Official record
Conditions
Lymphangioleiomyomatosis
Interventions
"Sirolimus" and "Hydroxychloroquine" 200 mg, "Sirolimus" and "Hydroxychloroquine" 400 mg
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 85 Years · Female only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 10, 2018 · Synced Jun 26, 2026, 5:48 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 May 1, 2018 · Synced Jun 26, 2026, 5:48 AM 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 18, 2024 · Synced Jun 26, 2026, 5:48 AM 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 Jun 26, 2026, 5:48 AM 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 Jun 26, 2026, 5:48 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 Jun 26, 2026, 5:48 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 Jun 26, 2026, 5:48 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 Jun 26, 2026, 5:48 AM 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 Jun 26, 2026, 5:48 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 15, 2020 · Synced Jun 26, 2026, 5:48 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 Jun 26, 2026, 5:48 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 Jun 23, 2026 · Synced Jun 26, 2026, 5:48 AM 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 Jun 26, 2026, 5:48 AM 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 Jun 26, 2026, 5:48 AM EDT