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Conditions: Leiomyomatosis
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: Not listed
Enrollment: 400 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1998 – 2003
U.S. locations: 1
States / cities: Bethesda, Maryland

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Conditions: Tuberous Sclerosis Complex
Interventions: everolimus; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years to 65 Years
Enrollment: 206 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2027
U.S. locations: 14
States / cities: Phoenix, Arizona • Los Angeles, California • Oakland, California + 11 more

Conditions: Relapsing-remitting Multiple Sclerosis
Interventions: BAF312, Placebo; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 297 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 16
States / cities: Cullman, Alabama • San Francisco, California • Centennial, Colorado + 13 more

Conditions: Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
Interventions: Confirmatory Testing; Diagnostic Test
Lead sponsor: RTI International; Other
Eligibility: 1 Day to 31 Days
Enrollment: 30,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

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

Search by objective public record fields.

Official Record of Patients Diagnosed With Lymphangioleiomyomatosis (LAM)

Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

TANDem-2: Closing the Gap to Interventions for TAND

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

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

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

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

Topical Rapamycin to Erase Angiofibromas in TSC

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)

Intermediate-Size Expanded Access Protocol (EAP) for LP352

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

Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies

TSC Biosample Repository and Natural History Database

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

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

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

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

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

Study of the Disease Process of Lymphangioleiomyomatosis

Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

Safety, Tolerability, Efficacy and Optimal Dose Finding Study of BAF312 in Patients With Relapsing-remitting Multiple Sclerosis

Early Check: Expanded Screening in Newborns

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