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Tuberous sclerosis complex

2 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for tuberous sclerosis complex

11 trials tracked for tuberous sclerosis complex: 3 in phase 3 or 4 and 2 with published results. The most-cited published study has 265 citations.

11Trials tracked

3Phase 3 & 4

0Recruiting

2With published results

Phase distribution

Phase 3 3 Phase 2 8

Phase 3 A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6) Completed · 265 cited
Phase 3 An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6) Completed · 104 cited
Phase 3 Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy Completed
Phase 2 Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC) Completed
Phase 2 Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC) Completed
Phase 2 Rapalogues for Autism Phenotype in TSC: A Feasibility Study Completed

Show 5 more trials

Phase 2 Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants Completed
Phase 2 Safety of Simvastatin in LAM and TSC Completed
Phase 2 A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD Completed
Phase 2 Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex Completed
Phase 2 Everolimus for Cancer With TSC1 or TSC2 Mutation Completed

Showing the 11 most-cited and recently-updated of 11 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Tuberous sclerosis complex: what the trials found

GWP42003-P demonstrated a statistically significant reduction in the number of TSC-associated seizures during maintenance and titration periods, with percent changes from baseline ranging from -20.08% to -43.36% (p=0.0009) ¹. Additionally, GWP42003-P was associated with significant improvements in Global Impression of Change scores as reported by both caregivers and participants (p=0.0074) ¹. During the Open-Label Extension (OLE) period, GWP42003-P showed a reduction in TSC-associated seizures ranging from -46.76% to -55.66% and a corresponding reduction in total seizure frequency of -46.76% to -55.18% [1, 2].

Everolimus has been evaluated in multiple trials for this condition [6, 8, 11].

Recent results — preliminary, needs further review

Early Vigabatrin showed no statistically significant difference in the number of subjects developing seizures (p=0.7375) or time to first clinical seizure (p=0.1174) ⁴. ["TAVT-18 (sirolimus)" - Not yet corroborated ⁵.] ["Simvastatin" - Not yet corroborated ⁹.] ["RAD001" - Not yet corroborated ¹⁰.]

For the clinician treating this condition

GWP42003-P is associated with significant reductions in TSC-associated seizure frequency and improved global impression of change scores [1, 2].
Everolimus has been established in clinical trials for this population [6, 8, 11].

AI synthesis of 5 cited trials, updated Jun 24, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.