Tuberous Sclerosis Complex
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Study of Skin Tumors in Tuberous Sclerosis
This clinical trial update indicates a study investigating skin tumors in people with tuberous sclerosis is currently recruiting.
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CLINICAL_TRIALS safety_alert · 2026-04-22Clinical Trial Update: Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution [Phase 4]
This Phase 4 clinical trial update describes ongoing assessment of the potential for chronic liver injury in participants treated with Epidi…
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: Study of the Disease Process of Lymphangioleiomyomatosis
NHLBI-sponsored clinical trial update focusing on the disease process of lymphangioleiomyomatosis, currently recruiting.
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CLINICAL_TRIALS research · 2026-04-02Clinical Trial Update: 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 [Phase 3]
A roll-over Phase 3 study is being conducted to collect long-term safety data for everolimus in patients with tuberous sclerosis complex and…
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CLINICAL_TRIALS research · 2026-04-02Clinical Trial Update: A Phase IV Study of Safety and Efficacy of Everolimus in Taiwanese Patients With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma (TSC-AML) [Phase 4]
Phase IV study assessing the safety and efficacy of Everolimus in Taiwanese patients with tuberous sclerosis complex-associated renal angiom…
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: Astroscape: A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II [Phase 1, Phase 2]
This clinical trial update describes Radiprodil in Phase 1/2 studies evaluating safety, tolerability, pharmacokinetics, and its effect on se…
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CLINICAL_TRIALS trial_result · 2026-03-18Clinical Trial Update: Remote Assessment and Intervention for Behavior Problems in Kids With TSC [NA]
A UCLA-led clinical trial examining remote assessment and intervention for behavior problems in children with tuberous sclerosis complex has…
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CLINICAL_TRIALS research · 2026-03-12Clinical Trial Update: 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 [Phase 4]
Phase 4 study evaluating Epidiolex as add-on therapy for tuberous sclerosis complex–associated neuropsychiatric disease in ages 1 to 65, cur…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY
An observational study of Epidyolex (cannabidiol) in Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex is currently r…
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: Treatment With Full-spectrum Cannabis Extract of Refractory Epilepsy Associated With Tuberous Sclerosis Complex (TSC) [Phase 3]
A Phase 3 clinical trial is recruiting to assess a full-spectrum cannabis extract for refractory epilepsy in patients with tuberous sclerosi…
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CLINICAL_TRIALS research · 2026-01-27Clinical Trial Update: Sapu003 in Advanced mTOR-sensitive Solid Tumors [Phase 1]
Phase 1 trial update for Sapu003 in advanced mTOR-sensitive solid tumors, currently recruiting across multiple indications including metasta…
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CLINICAL_TRIALS research · 2026-01-23Clinical Trial Update: Web Intervention for Parents of Youth With Genetic Syndromes (WINGS) [NA]
Rush University Medical Center is conducting a web-based intervention (WINGS) for parents of youths with several genetic syndromes, and the …
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CLINICAL_TRIALS research · 2026-01-23Clinical Trial Update: Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)
A pregnancy registry for Tuberous Sclerosis Complex and Lymphangioleiomyomatosis is currently recruiting, sponsored by David M. Ritter.
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CLINICAL_TRIALS research · 2026-01-22Clinical Trial Update: Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study [Phase 2]
Phase 2 trial investigating sirolimus for preventing onset and progression of epilepsy in tuberous sclerosis complex; currently recruiting.
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CLINICAL_TRIALS trial_result · 2026-01-21Clinical Trial Update: Feasibility of [11C]Acetate-PET in LAM and TSC [Phase 1, Phase 2]
Phase 1/2 feasibility study evaluating [11C]Acetate-PET imaging in lymphangioleiomyomatosis and tuberous sclerosis complex. Results have bee…
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CLINICAL_TRIALS trial_result · 2026-01-09Clinical Trial Update: Precision Medicine for Neurocutaneous Syndromes in Western China
A completed clinical trial from West China Hospital exploring precision medicine approaches for neurocutaneous syndromes, including NF1, TSC…
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CLINICAL_TRIALS research · 2025-12-26Clinical Trial Update: Role of Extracellular Vesicles as Biomarkers of Pulmonary Involvement in Patients With Lymphangioleiomyomatosis and Tuberous Sclerosis Complex
A University of Milan trial is recruiting to evaluate extracellular vesicles as biomarkers of pulmonary involvement in lymphangioleiomyomato…
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CLINICAL_TRIALS research · 2025-12-24Clinical Trial Update: Regulating Together in Tuberous Sclerosis Complex [NA]
The clinical trial 'Regulating Together in Tuberous Sclerosis Complex' is currently recruiting. Sponsored by Children's Hospital Medical Cen…
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CLINICAL_TRIALS research · 2025-12-23Clinical Trial Update: Safety, Tolerability, and Pharmacokinetics of SVG103 (Paxalisib) in Focal Cortical Dysplasia Type II (FCD-II), Tuberous Sclerosis Complex (TSC) or Hemimegalencephaly (HME) [Phase 1, Phase 2]
Clinical trial update on SVG103 (paxalisib) evaluating safety, tolerability, and pharmacokinetics in focal cortical dysplasia type II, tuber…
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CLINICAL_TRIALS trial_result · 2025-12-01Clinical Trial Update: Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex [Phase 2]
Phase 2 trial evaluating topical sirolimus ointment for cutaneous angiofibromas in tuberous sclerosis complex subjects was terminated; resul…
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CLINICAL_TRIALS trial_result · 2025-11-21Clinical Trial Update: Study to Evaluate the Pharmacokinetics and Safety Effects Following Coadministration of Carbamazepine or Itraconazole With Radiprodil in Healthy Adults [Phase 1]
Phase 1 study evaluating pharmacokinetics and safety of coadministering Radiprodil with Carbamazepine or Itraconazole in healthy adults; the…
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CLINICAL_TRIALS research · 2025-11-18Clinical Trial Update: Phase 1, Open-label, Drug-drug Interaction Study to Assess Effect Between Radiprodil and the Co-administered Drugs. [Phase 1]
Phase 1 open-label drug-drug interaction study of Radiprodil in the treatment of Tuberous Sclerosis Complex and Focal Cortical Dysplasia has…
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CLINICAL_TRIALS trial_result · 2025-10-31Clinical Trial Update: 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 [Phase 3]
A Phase 3 study of adjunctive cannabidiol oral solution GWP42003-P in children with tuberous sclerosis complex, Dravet syndrome, or Lennox-G…
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FDA approval · 2025-10-16FDA Approved: ZTALMY — Supplemental Application (Immedica Pharma US Inc)
FDA approves a supplemental application for ZTALMY (ganaxolone) submitted by Immedica Pharma US Inc.
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CLINICAL_TRIALS trial_result · 2025-10-01Clinical Trial Update: Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy [Phase 3]
Open-label Phase 3 trial of adjunctive ganaxolone (GNX) for Tuberous Sclerosis Complex–related epilepsy in children and adults was terminate…