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NCT06555965 · ClinicalTrials.gov registry record
STXBP1 and SYNGAP1 Related Disorders Natural History Study
A clinical trial of Genetic Disease and STXBP1 Encephalopathy With Epilepsy, sponsored by Children's Hospital of Philadelphia.
- Recruiting
- Registry status
- 600
- Enrollment target
- 5
- Study locations
NCT06555965: Recruiting study of Genetic Disease and STXBP1 Encephalopathy With Epilepsy, sponsored by Children's Hospital of Philadelphia.
NCT06555965 is a study of Genetic Disease and STXBP1 Encephalopathy With Epilepsy that is actively recruiting participants, run by Children's Hospital of Philadelphia. The registered enrollment target is 600 participants, below the 2,541-participant average among 30 other Genetic Disease trials with a reported enrollment target (76% lower). The trial reports 5 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06555965, a study of Genetic Disease and STXBP1 Encephalopathy With Epilepsy, is actively recruiting participants, sponsored by Children's Hospital of Philadelphia.
- RECRUITING
- Registry status
- 600 participants
- Enrollment target
- 5
- Study locations
Study Summary
The purpose of this study is to find out more about STXBP1 and SYNGAP1 related disorders. The information gathered by this study will be used to prepare for clinical treatment trials. The primary objective of the study is to better define and outline the clinical spectrum of STXBP1 and SYNGAP1 through detailed developmental, seizure, and quality of life assessments as an extension of routine clinical care.
Primary Outcome
The primary analysis will include all subjects meeting all inclusion and exclusion criteria and completing Visit 1. For each subject, the percentage of items performed correctly on the clinical assessments will be recorded. Changes in percentiles over time will be analyzed using a linear mixed effects model, to account for repeated measures for each patient.
Conditions Studied
Interventions
- OTHER Non-interventional study
Study Locations (5)
California
- Stanford Medicine Children's Health - Palo Alto
Colorado
- Children's Hospital Colorado - Aurora
New York
- Weill Cornell Medicine - New York
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
Texas
- Texas Children's Hospital - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 600 participants |
| Start Date | 2023-08-30 |
| Est. Completion | 2028-12-30 |
What NCT06555965 shows while recruiting
NCT06555965 is an observational study that tracks outcomes without assigning an intervention. The registered 600 participants enrollment target is mid-sized for trials with a published cap, below the 2,541-participant average among 30 other Genetic Disease trials with a reported enrollment target (76% lower).
The record links to 3 conditions, with Genetic Disease appearing as the primary indexed condition, and to 1 intervention - of which Non-interventional study is the first listed.
NCT06555965 lists 5 locations in 5 states (California, Colorado, New York).
Frequently Asked Questions
What is clinical trial NCT06555965 about?
NCT06555965 is a clinical study titled "STXBP1 and SYNGAP1 Related Disorders Natural History Study". The purpose of this study is to find out more about STXBP1 and SYNGAP1 related disorders. The information gathered by this study will be used to prepare for clinical treatment trials. The primary objective of the study is to better define and outline the clinical spectrum of STXBP1 and SYNGAP1 throu...
What is the current status of trial NCT06555965?
This trial is currently recruiting. The enrollment target is 600 participants. The study started on 2023-08-30. Estimated completion is 2028-12-30.
What conditions does trial NCT06555965 study?
This clinical trial studies the following conditions: Genetic Disease, STXBP1 Encephalopathy With Epilepsy, SYNGAP1-Related Intellectual Disability.
What interventions are being tested in trial NCT06555965?
The interventions under investigation include: Non-interventional study (OTHER).
Who is sponsoring clinical trial NCT06555965?
This trial is sponsored by Children's Hospital of Philadelphia, which has 431 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06555965 being conducted?
This trial has 5 study locations across California, Colorado, New York, Pennsylvania, Texas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT06555965's enrollment target sits among peer trials
600 9th of 30 higher than 22 of 30 other Genetic Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Genetic Disease 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.
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