Tuberous Sclerosis clinical trials
The registry lists 108 studies for Tuberous Sclerosis across more than 10 countries. 27 are recruiting or about to start. Per ClinicalTrials.gov, last updated 2026-09-18.
Status
- Completed
- 51
- Recruiting
- 23
- Active, not recruiting
- 10
- Status unknown
- 9
- Terminated
- 8
- Withdrawn
- 3
Phase
- Phase 2
- 33
- Phase 3
- 19
- Phase 1
- 14
- Phase 4
- 7
Where
- United States
- 58
- Australia
- 16
- United Kingdom
- 15
- Italy
- 13
- Spain
- 12
- Canada
- 12
Who runs them
Children's Hospital Medical Center, Cincinnati (7) · Jazz Pharmaceuticals (6) · Novartis Pharmaceuticals (6) · University of California, Los Angeles (4) · National Heart, Lung, and Blood Institute (NHLBI) (4) · GRIN Therapeutics, Inc. (3) · Beijing Children's Hospital (3) · Nobelpharma (3)
Studies
Recruiting first, then most recently updated. Showing 50 of 108 — search for more.
- Study of the Disease Process of LymphangioleiomyomatosisRecruitingNCT000014651 country
- Study of Skin Tumors in Tuberous SclerosisRecruitingNCT000019751 country
- Role of Genetic Factors in the Development of Lung DiseaseRecruitingNCT000015321 country
- Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME)RecruitingNCT07287202Phase 1/21 country
- Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental DisordersRecruitingNCT06081348Phase 21 country
- Parent and Infant Inter(X)Action Intervention (PIXI)Enrolling by invitationNCT03836300Not applicable1 country
- A Phase 2 Study of the Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory EpilepsyNot yet recruitingNCT07680322Phase 2
- Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)RecruitingNCT075753471 country
- TSC Biosample Repository and Natural History DatabaseRecruitingNCT056760992 countries
- Familial Investigations of Childhood Cancer PredispositionRecruitingNCT030502681 country
- A Smart Phone Application to Improve Adoption of the 2024 Kidney Disease Improving Global Outcomes (KDIGO) Chronic Kidney Disease (CKD) GuidelinesRecruitingNCT07561957Not applicable1 country
- Genetic and Electrophysiologic Study in Focal Drug-resistant EpilepsiesRecruitingNCT028906411 country
- Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC)RecruitingNCT07403266Phase 31 country
- Sapu003 in Advanced mTOR-sensitive Solid TumorsRecruitingNCT07369505Phase 11 country
- Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)RecruitingNCT061603101 country
- Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention StudyRecruitingNCT05104983Phase 21 country
- ESIS in Pediatric DRERecruitingNCT054693731 country
- TANDem-2: Closing the Gap to Interventions for TANDEnrolling by invitationNCT06879665Not applicable2 countries
- Qualitative Study in Patients With Genodermatoses and Healthcare Professionals on Reproductive CounsellingRecruitingNCT063303501 country
- Reproductive Options in Inherited Skin DiseasesEnrolling by invitationNCT063303241 country
- Observational, Retrospective, Multicenter, Nonprofit Study on the Prevalence of Renal Involvement in Pediatric Patients With Tuberous SclerosisRecruitingNCT067646021 country
- Genetic Bases of Neuroendocrine Neoplasms in Mexican PatientsRecruitingNCT065235821 country
- Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in InfantsRecruitingNCT04987463Phase 2/31 country
- Clinical Presentation and Renal Outcome of Patients With Tuberous Sclerosis Complex and/or Renal Angiomyolipoma in the Great West Region of FranceRecruitingNCT028877811 country
- National Registry of Rare Kidney DiseasesRecruitingNCT060658521 country
- GROWing Up With Rare GENEtic SyndromesRecruitingNCT044633161 country
- Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis ComplexRecruitingNCT05534672Phase 31 country
- Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)Active, not recruitingNCT06139172Not applicable1 country
- A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML)Active, not recruitingNCT05252585Phase 41 country
- 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 28 countries
- Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral SolutionActive, not recruitingNCT05044819Phase 41 country
- 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 318 countries
- Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)Active, not recruitingNCT024614591 country
- Early Behavioral Intervention to Improve Social Communication Function in Infants With Tuberous Sclerosis ComplexCompletedNCT02687633Not applicable1 country
- Treatment of Renal Angiomyolipomas in Tuberous Sclerosis by Beta-blockersTerminatedNCT02104011Phase 21 country
- Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALYActive, not recruitingNCT054858311 country
- Gait in Rare DiseasesCompletedNCT051614941 country
- 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 44 countries
- 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 1/28 countries
- Remote Assessment and Intervention for Behavior Problems in Kids With TSCCompletedNCT06311474Not applicable1 country
- Feasibility of [11C]Acetate-PET in LAM and TSCActive, not recruitingNCT05467397Phase 1/21 country
- Precision Medicine for Neurocutaneous Syndromes in Western ChinaCompletedNCT073271641 country
- Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic EncephalopathyCompletedNCT05626634Phase 22 countries
- Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis ComplexTerminatedNCT03363763Phase 22 countries
- Study to Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itraconazole With Radiprodil in Healthy AdultsTerminatedNCT06965881Phase 11 country
- Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs.CompletedNCT06975605Phase 11 country
- 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 33 countries
- Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related EpilepsyTerminatedNCT05604170Phase 310 countries
- Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related EpilepsyCompletedNCT05323734Phase 310 countries
- Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSCCompletedNCT05059327Phase 28 countries