- 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
Search Results
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Showing 1–24
of 87
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Telomeric 22Q13 Monosomy Syndrome, Tuberous Sclerosis, Hamartoma Syndrome, Multiple, Fragile X Syndrome, Angelman Syndrome, Rett Syndrome, Chromosome 15Q, Partial Deletion, Creatine Deficiency, X-linked
- Interventions
- Functional Behavioral Training (FBT), Positive Parenting Strategies-Treatment as Usual
- Behavioral · Other
- Lead sponsor
- Rush University Medical Center
- Other
- Eligibility
- 2 Years to 12 Years
- Enrollment
- 92 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- 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
- 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
- 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
- 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
- Tuberous Sclerosis, Subependymal Giant Cell Astrocytoma
- Interventions
- Everolimus, Placebo
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- Not listed
- Enrollment
- 117 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2014
- U.S. locations
- 12
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 8 more
- 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
- 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
- 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
- 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
- Central Nervous System (CNS) Lesions, Brain Metastases, Brain Neoplasms, Brain Neoplasms, Benign, Brain Tumor, Primary, Brain Tumor, Recurrent, Brain Tumors, Brain Cancer, Brain Tumor, Brain Neoplasm, Primary, Multiple Sclerosis, Multiple Sclerosis Brain Lesion, Neurofibroma, Acoustic Neuroma, CNS Tumor, CNS Lesion, CNS Metastases, CNS Cancer, CNS Lymphoma, Von Hippel Lindau, Meningioma, Glioma, Schwannomas, Neuroinflammation, Neoplasia
- Interventions
- RVP-001
- Drug
- Lead sponsor
- Reveal Pharmaceuticals Inc.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 5
- States / cities
- New Haven, Connecticut • Boston, Massachusetts • Columbia, Missouri + 1 more
- 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
- Conditions
- Recurrent Low Grade Astrocytoma, Recurrent WHO Grade 2 Glioma, Refractory Low Grade Astrocytoma, Refractory Low Grade Glioma, Refractory WHO Grade 1 Glioma
- Interventions
- Biospecimen Collection, Magnetic Resonance Imaging, Quality-of-Life Assessment, Questionnaire Administration, Selumetinib Sulfate, Vinblastine Sulfate
- Procedure · Other · Drug
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 2 Years to 25 Years
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 91
- States / cities
- Birmingham, Alabama • Little Rock, Arkansas • Loma Linda, California + 77 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
- 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
- Conditions
- Facial Angiofibroma
- Interventions
- Rapamune
- Drug
- Lead sponsor
- Le Bonheur Children's Hospital
- Other
- Eligibility
- 3 Years to 45 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Tuberous Sclerosis
- Interventions
- Parent-Child Interaction Therapy (PCIT)
- Behavioral
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 3 Years to 6 Years
- Enrollment
- 101 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- 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
- 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
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 159
- States / cities
- Birmingham, Alabama • Mobile, Alabama • Goodyear, Arizona + 137 more
- 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
- 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 – 2025
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- 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 – 2027
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio