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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
74 clinical trials for this condition and its sub-types.
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Could a common blood pressure drug shrink kidney tumors?
Disease control TerminatedThis pilot study tested whether propranolol, a beta-blocker, could shrink or stabilize kidney angiomyolipomas in people with tuberous sclerosis. Only 2 participants were enrolled before the trial was terminated, so the results are very limited. The goal was to find a less invasiv…
Phase: PHASE2 • Sponsor: University Hospital, Bordeaux • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Promising cream for rare skin condition falls short in early trial
Disease control TerminatedThis study tested a sirolimus cream (0.2% and 0.4%) applied daily for 12 weeks to treat facial angiofibromas in children aged 2-21 with tuberous sclerosis complex. The goal was to see if the cream could safely reduce the size and redness of these skin growths. The trial was termi…
Phase: PHASE2 • Sponsor: Aucta Pharmaceuticals, Inc • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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Experimental seizure drug tested in rare genetic disorder
Disease control TerminatedThis phase 3 study tested the drug ganaxolone as an add-on treatment for seizures in children and adults with tuberous sclerosis complex (TSC). The trial was open-label, meaning everyone received the drug, and it included 117 people who had previously been in related studies. The…
Phase: PHASE3 • Sponsor: Marinus Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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CBD study for baby seizures halted early
Symptom relief TerminatedThis study tested a cannabidiol (CBD) oral solution in children under 2 years old with tuberous sclerosis complex, Dravet syndrome, or Lennox-Gastaut syndrome who had uncontrolled seizures. The goal was to see if CBD is safe and can reduce seizures. However, the study was termina…
Phase: PHASE3 • Sponsor: Jazz Pharmaceuticals • Aim: Symptom relief
Last updated Jun 27, 2026 07:56 UTC
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Drug interaction study for radiprodil halted early
Knowledge-focused TerminatedThis early-stage study looked at how two common drugs (carbamazepine and itraconazole) change the levels of radiprodil in the blood of 30 healthy adults. The goal was to understand safety and dosing for future use in neurological conditions like tuberous sclerosis. However, the s…
Phase: PHASE1 • Sponsor: GRIN Therapeutics, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:59 UTC