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A Study Observing Disease Progression in a Patient With Hutchinson-Gilford Progeria Syndrome (HGPS)

Study acronym: progeria
StatusNot Yet Recruiting
PhaseNot specified
Started
View on ClinicalTrials.gov ↗

Record History

  1. Study First Submitted
This study is a prospective, non-interventional, single-patient observational study following one patient with Hutchinson-Gilford Progeria Syndrome (HGPS), a rare disease that causes rapid, premature aging, over a 3-year period without any therapeutic intervention. The main purpose of this study is to track disease-related biomarkers over time - through physical measurements, cardiac and vascular ultrasound, bone age and bone density scans, cognitive testing, and quality-of-life assessments, along with blood tests (a total of 4 research blood draws, 3ml each) - in order to establish baseline natural history data that will support the future development and evaluation of an RNA-targeting gene therapy (based on AAV-Cas13 technology) for this condition.
Trial Details
NCT Number NCT07839351
Lead Sponsor Bundang CHA Hospital
Conditions
(from sponsor)		 Hutchinson-Gilford Progeria Syndrome
Related terms Progeria
Enrollment 1 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov