Tuberous sclerosis 1
MONDO:0008612An autosomal dominant syndrome caused by pathogenic variants in the TSC1 gene, characterized by the growth of hamartomas in multiple organs, including the brain, skin, kidneys, heart, and lungs. Other clinical features include seizures, intellectual disability, and skin lesions.
Also known as: TSC1 tuberous sclerosis, TSC1-related tuberous sclerosis, tuberous sclerosis 1, tuberous sclerosis caused by mutation in TSC1, tuberous sclerosis type 1, tuberous sclerosis-1, TSC1, tuberose sclerosis
65 clinical trials for this condition and its sub-types, 33 tagged with Tuberous sclerosis 1 itself.
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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