CACNA1A-related complex neurodevelopmental disorder
MONDO:0100254A progressive complex neurodevelopmental condition caused by variants in the CACNA1A gene. Phenotypic onset (usually) occurs around age 1 and most often includes intellectual disability but can also include epileptic encephalopathy, benign paroxysmal torticollis of infancy and paroxysmal tonic upgaze psychomotor delay, learning difficulties, absence epilepsy, episodic ataxia, and hemiplegic migraines.
11 clinical trials for this condition and its sub-types, 1 tagged with CACNA1A-related complex neurodevelopmental disorder itself.
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Sub-types of CACNA1A-related complex neurodevelopmental disorder
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Spinocerebellar ataxia type 6 9 trials
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Episodic ataxia type 2 1 trial
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Migraine, familial hemiplegic, 1 1 trial
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New drug could slow rare brain disease that steals balance
Disease control OngoingThis phase 3 trial tests whether the drug troriluzole can slow the progression of spinocerebellar ataxia, a rare genetic disorder that affects coordination and balance. About 300 adults with different types of SCA are randomly assigned to take either troriluzole or a placebo dail…
Phase 3 • Sponsor: Biohaven Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Could a drug slow rare brain disease? new study uses Real-World data to find out
Disease control OngoingThis study looks at whether the drug troriluzole can slow the progression of spinocerebellar ataxia (SCA), a rare genetic disease that affects movement and balance. Researchers will compare 909 patients who took troriluzole for up to three years with similar patients who did not …
Sponsor: Biohaven Therapeutics Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:04 UTC