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 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 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 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 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 research · 2025-09-22Clinical Trial Update: TANDem-2: Closing the Gap to Interventions for TAND [NA]
Clinical trial update for TANDem-2, a study addressing interventions for TSC-associated neuropsychiatric disorders (TAND), is currently enro…
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CLINICAL_TRIALS research · 2025-08-29Clinical Trial Update: Gait in Rare Diseases
This clinical trial update indicates a study examining gait in rare diseases involving tuberous sclerosis complex and STXBP1 encephalopathy …
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CLINICAL_TRIALS research · 2025-07-16Clinical Trial Update: Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders [Phase 2]
A Phase 2 trial is evaluating sertraline versus placebo for anxiety in children and adolescents with neurodevelopmental disorders, including…
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CLINICAL_TRIALS research · 2025-07-08Clinical Trial Update: TSC Biosample Repository and Natural History Database
Recruiting study to create a biosample repository and natural history database for tuberous sclerosis complex and lymphangioleiomyomatosis.
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CLINICAL_TRIALS research · 2025-01-15Clinical Trial Update: Clinical Trial Data Set Re-use With Statistical Methodologies Tailored for Clinical Trials in Rare Diseases
This update describes a study on re-using clinical trial data with tailored statistical methods for rare diseases. The focus includes tubero…
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CLINICAL_TRIALS research · 2025-01-08Clinical Trial Update: Observational, Retrospective, Multicenter, Nonprofit Study on the Prevalence of Renal Involvement in Pediatric Patients With Tuberous Sclerosis
An observational, retrospective multicenter study to determine the prevalence of renal involvement in pediatric patients with tuberous scler…
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CLINICAL_TRIALS research · 2024-10-26Clinical Trial Update: Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)
This clinical study investigates determinants of autism spectrum disorder and intellectual disability in tuberous sclerosis complex, and is …
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CLINICAL_TRIALS research · 2024-06-03Clinical Trial Update: Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1) [Phase 2]
This update describes the Phase 2 PRECISION 1 basket trial of nab-sirolimus for malignant solid tumors with pathogenic TSC1/TSC2 alterations…
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CLINICAL_TRIALS research · 2017-05-04Clinical Trial Update: Tuberous Sclerosis Complex Natural History Study: Renal Manifestations
A completed clinical trial update for the Tuberous Sclerosis Complex Natural History Study focusing on renal manifestations.