Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT07600658 · ClinicalTrials.gov registry record · Phase 1
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
A Phase 1 study, sponsored by Stanford University.
- Not yet
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
NCT07600658: Clinical Trial Phase 1 study, sponsored by Stanford University.
NCT07600658 is a Phase 1 study that has not yet begun recruiting, run by Stanford University. The registered enrollment target is 5 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07600658, a Phase 1 study, has not yet begun recruiting, sponsored by Stanford University.
- NOT YET RECRUITING
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
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
Interventions
- DRUG TS1-ASO
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2026-09 |
| Est. Completion | 2030-12 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07600658
The ClinicalTrials.gov registry entry for NCT07600658 describes a study currently listed as not yet recruiting, categorized as Phase 1. The registered enrollment target is 5 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Stanford University, which has 1,744 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which TS1-ASO is the first listed.
NCT07600658 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT07600658 about?
NCT07600658 is a clinical study 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". 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...
What is the current status of trial NCT07600658?
This trial is currently not yet recruiting. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2026-09. Estimated completion is 2030-12.
What interventions are being tested in trial NCT07600658?
The interventions under investigation include: TS1-ASO (DRUG).
Who is sponsoring clinical trial NCT07600658?
This trial is sponsored by Stanford University, which has 1,744 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
Explore more on PlainTrial
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.