This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
Data will be abstracted through medical record review ensuring inclusion only of data from participants meeting all the inclusion criteria and none of the censoring criteria. Data will be collected retrospectively from study sites, including medical clinics, hospitals and academic medical centers.
Study Type
OBSERVATIONAL
Enrollment
60
Number of Annualized Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs)
Time frame: Up to 10 years
Number of CEC-adjudicated MDEs as Assessed by a Pre-Specified Severity Rating Scale
Time frame: Up to 10 years
Number of Annualized CEC-adjudicated PA-related Hospitalizations
Time frame: Up to 10 years
Number of Annualized CEC-adjudicated MDE-related Hospitalizations
Time frame: Up to 10 years
Number of Annualized CEC-adjudicated PA-related Urgent Healthcare Visits
Time frame: Up to 10 years
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University of Stanford Medical Center
Stanford, California, United States
NOT_YET_RECRUITINGJohns Hopkins University
Baltimore, Maryland, United States
RECRUITINGUniversity of Michigan Hospital
Ann Arbor, Michigan, United States
ACTIVE_NOT_RECRUITINGChildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
RECRUITINGTexas Children's Hospital
Houston, Texas, United States
TERMINATEDSeattle Children's Hospital
Seattle, Washington, United States
RECRUITINGRoyal Children's Hospital Melbourne
Parkville, Victoria, Australia
WITHDRAWNStollery Children's Hospital University of Alberta
Edmonton, Alberta, Canada
WITHDRAWNCHU Toulouse
Toulouse, Haute-Garonne, France
RECRUITINGCHRU Nancy Hôpital des Enfants
Vandœuvre-lès-Nancy, Meurthe-et-Moselle, France
WITHDRAWN...and 19 more locations