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Home › Conditions › tuberous sclerosis complex

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 258 citations.

11Trials tracked
3Phase 3 & 4
0Recruiting
2With published results
Phase distribution
Phase 3 3 Phase 2 8
  1. Phase 3 A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6) Completed · 258 cited
  2. Phase 3 An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6) Completed · 103 cited
  3. Phase 3 Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy Completed
  4. Phase 2 Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC) Completed
  5. Phase 2 Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC) Completed
  6. Phase 2 Rapalogues for Autism Phenotype in TSC: A Feasibility Study Completed
Show 5 more trials
  1. Phase 2 Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants Completed
  2. Phase 2 Safety of Simvastatin in LAM and TSC Completed
  3. Phase 2 A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD Completed
  4. Phase 2 Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex Completed
  5. 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.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Bumetanide shows individual-level improvement in heterogeneous neurodevelopmental cohort
Psychiatry RCT
Bumetanide shows individual-level improvement in heterogeneous neurodevelopmental cohort Bumetanide helps mood and sleep in kids with sensory issues
In a post-trial access study using a multiple-baseline single-case experimental design, 113 youth (7–19 years) with ASD, ADHD, epilepsy, or …
Kids with autism, ADHD, or epilepsy who feel sensory overload saw better mood and sleep after taking bumetanide.
medRxiv Apr 24, 2026
In pediatric TSC, whole-exome sequencing identified mutations in 93% of patients and revealed novel variants
Pediatrics Cohort
In pediatric TSC, whole-exome sequencing identified mutations in 93% of patients and revealed novel variants A New Genetic Map Could Change How Doctors Treat This Childhood Disorder
This retrospective cohort study evaluated 73 pediatric patients with tuberous sclerosis complex (TSC) at Nanjing Medical University Children…
For families navigating a complex genetic disorder, new research is making the path forward clearer and more personalized.
Frontiers Apr 13, 2026