Clinical Trial: A Phase I/II, First-In-Human Trial to Evaluate the Safety, Tolerability, and Pharmacokinetic Activity to Prevent or Treat Neuropsychiatric Symptoms in Pediatric Subjects With Timothy Syndrome

U.S., May 23 -- ClinicalTrials.gov registry received information related to the study (NCT07600658) titled 'A Phase I/II, First-In-Human Trial to Evaluate the Safety, Tolerability, and Pharmacokinetic Activity to Prevent or Treat Neuropsychiatric Symptoms in Pediatric Subjects With Timothy Syndrome' on May 04.

Brief Summary: The goal of this clinical trial is to learn if an antisense oligonucleotide (TS1-ASO) can safely treat and potentially prevent neuropsychiatric and neurodevelopmental symptoms in pediatric participants (age >2 months) with Timothy Syndrome Type 1 (TS1).

The main questions it aims to answer are:

1. Is TS1-ASO safe and well tolerated when administered intrathecally in children with TS1?

2. What are the pharmacokinetic...