Tuberous Sclerosis: all 115 studies

Page 1 of 2. Open to participants first (27), then studies with results posted, then the rest; newest registration first within each.

• Neurocutaneous Syndromes in Children: Prevalence and Clinical CharacteristicsNot yet recruitingNCT07843823
• Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1)Not yet recruitingNCT07680322Phase 2
• Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)RecruitingNCT07575347
• A Smart Phone Application to Improve Adoption of the 2024 Kidney Disease Improving Global Outcomes (KDIGO) Chronic Kidney Disease (CKD) GuidelinesRecruitingNCT07561957Not applicable
• Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC)RecruitingNCT07403266Phase 3
• Sapu003 in Advanced mTOR-sensitive Solid TumorsRecruitingNCT07369505Phase 1
• Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME)RecruitingNCT07287202Phase 1/2
• TANDem-2: Closing the Gap to Interventions for TANDEnrolling by invitationNCT06879665Not applicable
• Observational, Retrospective, Multicenter, Nonprofit Study on the Prevalence of Renal Involvement in Pediatric Patients With Tuberous SclerosisRecruitingNCT06764602
• Genetic Bases of Neuroendocrine Neoplasms in Mexican PatientsRecruitingNCT06523582
• Qualitative Study in Patients With Genodermatoses and Healthcare Professionals on Reproductive CounsellingRecruitingNCT06330350
• Reproductive Options in Inherited Skin DiseasesEnrolling by invitationNCT06330324
• Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)RecruitingNCT06160310
• Intermediate-Size Expanded Access Protocol (EAP) for LP352AvailableNCT06149663
• Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental DisordersRecruitingNCT06081348Phase 2
• National Registry of Rare Kidney DiseasesRecruitingNCT06065852
• TSC Biosample Repository and Natural History DatabaseRecruitingNCT05676099
• Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis ComplexRecruitingNCT05534672Phase 3
• Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in InfantsRecruitingNCT04987463Phase 2/3
• GROWing Up With Rare GENEtic SyndromesRecruitingNCT04463316
• Parent and Infant Inter(X)Action Intervention (PIXI)Enrolling by invitationNCT03836300Not applicable
• Familial Investigations of Childhood Cancer PredispositionRecruitingNCT03050268
• Genetic and Electrophysiologic Study in Focal Drug-resistant EpilepsiesRecruitingNCT02890641
• Clinical Presentation and Renal Outcome of Patients With Tuberous Sclerosis Complex and/or Renal Angiomyolipoma in the Great West Region of FranceRecruitingNCT02887781
• Study of Skin Tumors in Tuberous SclerosisRecruitingNCT00001975
• Role of Genetic Factors in the Development of Lung DiseaseRecruitingNCT00001532
• Study of the Disease Process of LymphangioleiomyomatosisRecruitingNCT00001465
• Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related EpilepsyTerminatedNCT05604170Phase 3Results posted
• Feasibility of [11C]Acetate-PET in LAM and TSCActive, not recruitingNCT05467397Phase 1/2Results posted
• Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related EpilepsyCompletedNCT05323734Phase 3Results posted
• Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC InfantsCompletedNCT04595513Phase 1/2Results posted
• 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 SeizuresTerminatedNCT04485104Phase 3Results posted
• Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)CompletedNCT04285346Phase 2Results posted
• Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis ComplexCompletedNCT03826628Phase 2/3Results posted
• 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 EncephalopathiesCompletedNCT03650452Phase 2Results posted
• Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.CompletedNCT03525834Phase 4Results posted
• Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis ComplexTerminatedNCT03363763Phase 2Results posted
• Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis ComplexCompletedNCT02849457Phase 2Results posted
• A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)CompletedNCT02544763Phase 3Results posted
• An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)CompletedNCT02544750Phase 3Results posted
• A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCDCompletedNCT02451696Phase 2Results posted
• Everolimus for Cancer With TSC1 or TSC2 MutationCompletedNCT02201212Phase 2Results posted
• Safety of Simvastatin in LAM and TSCCompletedNCT02061397Phase 1/2Results posted
• Rapalogues for Autism Phenotype in TSC: A Feasibility StudyCompletedNCT01929642Phase 2Results posted
• Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment DisabilitiesCompletedNCT01906866Phase 3Results posted
• 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 SeizuresCompletedNCT01713946Phase 3Results posted
• Topical Rapamycin to Erase Angiofibromas in TSCCompletedNCT01526356Phase 2Results posted
• Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)CompletedNCT01289912Phase 2Results posted
• Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) PatientsTerminatedNCT01266291Phase 4Results posted
• Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)CompletedNCT01070316Phase 1/2Results posted
• Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)CompletedNCT00790400Phase 3Results posted
• Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)CompletedNCT00789828Phase 3Results posted
• Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis ComplexCompletedNCT00411619Phase 1/2Results posted
• RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMASCompletedNCT00126672Phase 2Results posted
• Precision Medicine for Neurocutaneous Syndromes in Western ChinaCompletedNCT07327164
• Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs.CompletedNCT06975605Phase 1
• Study to Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itraconazole With Radiprodil in Healthy AdultsTerminatedNCT06965881Phase 1
• Predicting Epileptogenic Tubers in Patients with Tuberous Sclerosis ComplexCompletedNCT06789419
• Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type IIActive, not recruitingNCT06392009Phase 1/2
• Remote Assessment and Intervention for Behavior Problems in Kids With TSCCompletedNCT06311474Not applicable
• Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)Active, not recruitingNCT06139172Not applicable
• Acceptance and Commitment Therapy in Tuberous Sclerosis ComplexActive, not recruitingNCT05867576Not applicable
• 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 ComplexCompletedNCT05864846Phase 4
• Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic EncephalopathyCompletedNCT05626634Phase 2
• Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSCCompletedNCT05495425Phase 3
• Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALYActive, not recruitingNCT05485831
• ESIS in Pediatric DREActive, not recruitingNCT05469373
• Study to Investigate LP352 in Subjects With Developmental and Epileptic EncephalopathiesCompletedNCT05364021Phase 1/2
• Lymphangioleiomyomatosis, a Study on Cathepsin KCompletedNCT05323370
• KidneYou - Innovative Digital TherapyCompletedNCT05286632Not applicable
• A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML)CompletedNCT05252585Phase 4
• Clinical Trial Data Set Re-use With Statistical Methodologies Tailored for Clinical Trials in Rare DiseasesCompletedNCT05199402
• Gait in Rare DiseasesCompletedNCT05161494
• Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention StudyActive, not recruitingNCT05104983Phase 2
• Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSCCompletedNCT05059327Phase 2
• Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral SolutionActive, not recruitingNCT05044819Phase 4
• TSC Remote Assessment and InterventionCompletedNCT04698538Not applicable
• Use of a Tonometer to Identify Epileptogenic Lesions During Pediatric Epilepsy SurgeryWithdrawnNCT04344626Not applicable
• The Effectiveness and Safety of Vagus Nerve Stimulation for TRECompletedNCT04198207
• The Effectiveness and Safety of Resective Epilepsy Surgery for TREActive, not recruitingNCT04198181
• Dermatologic Patterns of Tuberous Sclerosis Patients and Somatic Mutation RelationshipStatus unknownNCT04112537
• JASPER Early Intervention for Tuberous SclerosisStatus unknownNCT03422367Not applicable
• Aspirin as an add-on Treatment of Refractory Epilepsy in Tuberous Sclerosis ComplexStatus unknownNCT03356769Phase 2
• Characterizing Sleep Disorders in Children and Adults With Tuberous Sclerosis Complex (TSC)Status unknownNCT03326765Not applicable
• Studies in Patients With Tuberous Sclerosis ComplexCompletedNCT03276195
• Turmeric as Treatment in EpilepsyWithdrawnNCT03254680Not applicable
• Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous SclerosisCompletedNCT03140449Phase 3
• 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 TreatmentActive, not recruitingNCT02962414Phase 3
• Topical Everolimus in Patients With Tuberous Sclerosis ComplexWithdrawnNCT02860494Phase 2/3
• Early Behavioral Intervention to Improve Social Communication Function in Infants With Tuberous Sclerosis ComplexCompletedNCT02687633Not applicable
• Biomarkers for Tuberous Sclerosis Complex (BioTuScCom)TerminatedNCT02654340
• Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)CompletedNCT02635789Phase 3
• Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis ComplexCompletedNCT02634931Phase 3
• Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)Active, not recruitingNCT02461459
• The Cognitive Variability in NF1 and TSC Monozygotic TwinsStatus unknownNCT02436746
• Characterization of Patients With Tuberous Sclerosis Complex, Lymphangioleiomyomatosis and AngiomyolipomaCompletedNCT02325505
• Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockersTerminatedNCT02104011Phase 2
• Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Model of Epilepsy - Tuberous Sclerosis ComplexStatus unknownNCT02098759Not applicable
• A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous SclerosisStatus unknownNCT01954693Phase 2
• Assessment of the Pharmacokinetics of Circadin® in Children With Neurodevelopmental Disorders and Sleep DisturbancesCompletedNCT01903681Phase 1

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