Central nervous system malformation
MONDO:002002250 clinical trials for this condition and its sub-types.
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Broader categories
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New drug aims to tame hard-to-control seizures in rare mitochondrial disorders
Disease control TerminatedThis study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …
Phase: PHASE2, PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:03 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