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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.
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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