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Tuberous sclerosis
MONDO:0001734Hereditary disease characterized by seizures, intellectual disability, developmental delay, and skin and ocular lesions. First signs usually occur during infancy or childhood but in rare cases may not occur until 2nd or 3rd decade.
Also known as: Bourneville disease, Bourneville syndrome, Bourneville's disease, Bourneville's syndrome, TSC, epiloia, tuberous sclerosis, tuberous sclerosis complex
75 clinical trials for this condition and its sub-types, 41 tagged with Tuberous sclerosis itself.
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Sub-types of Tuberous sclerosis
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Tuberous sclerosis 1 33 trials
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Tuberous sclerosis 2 2 trials
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Can a new drug curb seizures in children with severe epilepsy?
Disease control CompletedResearchers are testing an experimental drug called TAK-935 in children aged 2 to 17 with Dravet syndrome or Lennox-Gastaut syndrome, two severe forms of epilepsy that often resist treatment. The trial compares TAK-935 added to standard anti-seizure medications against a placebo …
Phase 2 • Sponsor: Takeda • Aim: Disease control
Last updated Sep 16, 2026 00:00 UTC
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New epilepsy drug shows promise in long-term safety trial
Disease control CompletedThis study tested the long-term safety of a drug called LP352 (bexicaserin) in 41 people aged 12 to 65 with severe epilepsy syndromes like Dravet or Lennox-Gastaut. Participants took the drug three times daily for up to 52 weeks. The main goal was to check for side effects, while…
Phase 2 • Sponsor: Longboard Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC
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Play therapy may boost social skills in infants with tuberous sclerosis — a trial puts it to the test
Symptom relief CompletedThis trial tests whether JASPER, a play-based behavioral intervention, can improve social communication and play skills in infants (12–36 months) with tuberous sclerosis complex (TSC). Caregivers learn strategies during daily and weekly sessions to support their child's developme…
Sponsor: University of California, Los Angeles • Aim: Symptom relief
Last updated Jul 25, 2026 00:00 UTC
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Epidiolex shows promise for easing seizures and behavior issues in TSC
Symptom relief CompletedThis study looked at whether adding Epidiolex (a CBD-based medicine) to usual treatment can improve seizures, behavior, sleep, and quality of life in people aged 1 to 65 with tuberous sclerosis complex (TSC). 79 participants took the drug and were monitored for changes in symptom…
Phase 4 • Sponsor: Jazz Pharmaceuticals • Aim: Symptom relief
Last updated Jun 27, 2026 12:24 UTC
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Remote therapy shows promise for TSC behavior issues in preschoolers
Symptom relief CompletedThis study looked at behavior problems in 101 children aged 3 to 6 with tuberous sclerosis complex (TSC). Families completed assessments online, and those with elevated behavior issues were offered a parent-training therapy (PCIT) over the internet. The goal was to help parents m…
Sponsor: University of California, Los Angeles • Aim: Symptom relief
Last updated Jun 27, 2026 11:02 UTC
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Walking analysis sheds light on rare genetic disorders
Knowledge-focused CompletedThis study looked at whether a special walking test (3D gait analysis) can help identify movement problems in people with rare genetic diseases like Tuberous Sclerosis and STXBP1. About 40 participants aged 6 and older who could walk without help took part. The goal was to see if…
Sponsor: Universiteit Antwerpen • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:31 UTC
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20-Year study tests precision medicine for rare Brain-Skin disorders
Knowledge-focused CompletedThis completed study looked at 1,200 people with neurocutaneous syndromes (NF1, TSC, SWS, VHL) in Western China. Researchers compared those who received genetic testing, targeted drugs, and coordinated specialist care against those who got standard care. The goal was to see if pr…
Sponsor: West China Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:09 UTC
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New drug interaction study aims to make radiprodil safer for future use
Knowledge-focused CompletedThis study tested how the experimental drug radiprodil interacts with five common medications (warfarin, midazolam, digoxin, rosuvastatin, and omeprazole) in 18 healthy adults. Participants took radiprodil alone and then with each of these drugs to measure changes in drug levels …
Phase 1 • Sponsor: GRIN Therapeutics, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:58 UTC