First human trial of TS1-ASO aims to tame rare genetic brain disorder
NCT ID NCT07600658
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This first-in-human trial tests a genetic drug called TS1-ASO in 5 children with Timothy Syndrome, a rare condition causing severe heart and brain problems. The drug is injected into the spine to target the genetic root of the disease. The main goal is to check safety and how the drug moves in the body, with early signs of whether it might help development and seizures.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- TS1-ASO (a genetic drug called an antisense oligonucleotide)
- What this could lead to
- If it works, this could point toward a treatment that prevents or eases severe developmental and seizure symptoms in children with Timothy Syndrome.
- What could go wrong
- This is a very early, tiny trial (only 5 participants) with no comparison group. The drug is injected into the spine, which carries risks like infection or nerve damage, and it may not improve symptoms at all.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Stanford University
Stanford, California, 94305, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.