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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New study tests everolimus for kidney tumors in TSC patients
Disease control OngoingThis study looks at how safe and effective the drug everolimus is for Taiwanese adults with tuberous sclerosis complex (TSC) who have kidney tumors called angiomyolipomas. Four participants will take the drug and be monitored for side effects and tumor shrinkage. The goal is to c…
Phase 4 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Sep 19, 2026 00:00 UTC
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Drug shows promise for Long-Term seizure control in rare disease
Disease control OngoingThis study looks at the long-term safety of everolimus in people with tuberous sclerosis complex (TSC) who have seizures that are hard to control. Participants are already taking everolimus and benefiting from it in an earlier study. The goal is to see if the drug remains safe an…
Phase 3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Aug 23, 2026 00:00 UTC
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Can early parent coaching help infants with rare genetic disorders thrive?
Disease control By invitation onlyThis study tests a program called PIXI that coaches parents of infants diagnosed with rare neurogenetic disorders (such as Fragile X, Angelman, or Down syndrome) during the first year of life. The program combines education about the disorder, guided parent-child interaction acti…
Sponsor: RTI International • Aim: Disease control
Last updated Jul 15, 2026 00:00 UTC
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CBD epilepsy drug under Real-World watch for rare seizure disorders
Disease control OngoingThis study follows about 111 people with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex who are taking Epidyolex (a CBD oral solution) for seizures. Researchers track how many stay on the drug for a year and how many seizure-free days they have. It's an o…
Sponsor: Jazz Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 13:01 UTC
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New drug radiprodil tested for tough seizures in two brain conditions
Disease control OngoingThis early-phase trial tests an experimental drug called radiprodil in 30 people with tuberous sclerosis complex or focal cortical dysplasia type II whose seizures have not responded to at least two other medications. The study is open-label, meaning everyone gets the drug, and w…
Phase 1/2 • Sponsor: GRIN Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:02 UTC
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New PET scan could reveal early drug response in rare lung disease
Diagnosis OngoingThis study tests whether a special PET scan using [11C]acetate can detect early signs that the drug rapamycin is working in people with lymphangioleiomyomatosis (LAM), a rare lung disease. Seven adults with LAM and kidney tumors will get one or two scans over 3–6 months. The goal…
Phase 1/2 • Sponsor: Brigham and Women's Hospital • Aim: Diagnosis
Last updated Jun 27, 2026 08:12 UTC
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Can a video-based therapy tame tough behaviors in kids with rare genetic disorders?
Symptom relief OngoingThis study tests a virtual behavioral therapy (Functional Behavioral Training) for children aged 2-12 with genetic syndromes like Fragile X, Angelman, or Rett syndrome who have challenging behaviors. The therapy teaches parents how to identify what triggers problem behaviors and …
Sponsor: Rush University Medical Center • Aim: Symptom relief
Last updated Sep 20, 2026 00:00 UTC
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New online program aims to ease caregiver stress in rare disease
Symptom relief By invitation onlyThis pilot study tests a short online program called 'Well-Beans for Caregivers' for adults caring for someone with tuberous sclerosis complex (TSC). The program consists of three weekly 2-hour sessions based on Acceptance and Commitment Therapy to help caregivers cope with diffi…
Sponsor: Vrije Universiteit Brussel • Aim: Symptom relief
Last updated Jun 27, 2026 07:51 UTC
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Epidiolex liver check: is Long-Term use safe?
Knowledge-focused OngoingThis study follows 154 people taking Epidiolex (cannabidiol) for seizures caused by Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex. Researchers will monitor for signs of chronic liver injury and fibrosis over time using blood tests and liver scans. The go…
Phase 4 • Sponsor: Jazz Pharmaceuticals • Aim: Knowledge-focused
Last updated Aug 28, 2026 00:00 UTC
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Brain imaging may unlock early clues to autism in tuberous sclerosis
Knowledge-focused OngoingThis study investigates whether advanced brain imaging and electrical recordings can predict the presence and severity of autism spectrum disorder (ASD) and intellectual disability (ID) in people with tuberous sclerosis complex (TSC). Researchers will follow participants over tim…
Sponsor: Boston Children's Hospital • Aim: Knowledge-focused
Last updated Aug 08, 2026 00:03 UTC