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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

NCT07600658 · Status: NOT_YET_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2026-05-22

No results posted yet for this study

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 pharmacokinetics and preliminary efficacy of TS1-ASO on neurodevelopmental and neurologic outcomes?

This is a single-arm study (no comparison group).

Participants will:

1. Receive intrathecal injections of TS1-ASO via lumbar puncture using a stepwise dose-escalation approach
2. Undergo safety monitoring including neurologic exams, cardiac monitoring, laboratory testing, and adverse event assessments
3. Provide cerebrospinal fluid (CSF) and blood samples for pharmacokinetic and biomarker analyses
4. Complete neurodevelopmental, behavioral, and functional assessments (e.g., adaptive behavior, motor function, communication, seizure tracking) over time

Conditions

  • CACNA1C

Interventions

DRUG

TS1-ASO

Antisense oligonucleotide targeting CACNA1C exon 8A/8 splicing

Sponsors & Collaborators

Principal Investigators

  • Sergiu Pasca, MD · Stanford University

Study Design

Allocation
NA
Purpose
PREVENTION
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-09-30
Primary Completion
2029-12-31
Completion
2030-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07600658 on ClinicalTrials.gov