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, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower). 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
Primary Outcome
Safety and tolerability of TS1-ASO will be evaluated by systematic collection and analysis of AEs and SAEs following intrathecal administration. Events will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 and assessed for severity, seriousness, and relationship to study drug and procedure. Safety assessments include continuous cardiac monitoring (telemetry), serial electrocardiograms, neurologic examinations, and routine la
Interventions
- DRUG TS1-ASO
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2026-09 |
| Est. Completion | 2030-12 |
| Phase | Phase 1 |
What is known before NCT07600658 opens enrollment
NCT07600658 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 5 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower).
The record links to 0 conditions, and to 1 intervention - of which TS1-ASO is the first listed.
NCT07600658 does not publish any study locations in the registry export this page uses.
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
How this trial's enrollment target compares
Where NCT07600658's enrollment target sits among peer trials
5 1989th of 2000 higher than 6 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia