Actively Recruiting
A Retrospective Study to Understand Patients With Propionic Acidemia Collecting Medical Records From Multiple Centers Globally
Led by ModernaTX, Inc. · Updated on 2026-02-05
60
Participants Needed
29
Research Sites
N/A
Total Duration
AI-Summary
What this Trial Is About
This research aims to describe the characteristics, clinical outcomes, and event rates in participants with propionic acidemia PA, a rare metabolic disorder. It is a non-interventional, observational, global, multicenter study that collects data retrospectively from medical records to better understand PA over time. Data will be gathered by reviewing medical records from various study sites, including hospitals, clinics, and academic centers. Participants included will be those diagnosed with PA confirmed by genetic testing, with records documenting specific metabolic events. The study will cover up to 10 years of medical history and events. Participants involvement consists of allowing researchers to review their medical records to collect information about metabolic decompensation events, hospitalizations, and urgent healthcare visits related to PA. Researchers will analyze the number and severity of these events, along with hospitalization data, to understand PAs clinical impact. The study does not involve direct treatment or interventions and focuses on data collection and analysis.
CONDITIONS
Brief Title
A Retrospective Study to Characterize Participants With Propionic Acidemia
Research Team
M
Moderna WeCare Team
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