A Retrospective Cohort Study to Characterize Propionic Acidemia Patients
ModernaTX, Inc.
Summary
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
Description
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.
Eligibility
- Age range
- 2+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: Participants' medical records are eligible for abstraction only if all of the following criteria apply: * Confirmed diagnosis of PA based on diagnosis by molecular genetic testing (propionyl-CoA carboxylase subunitα\[PCCA\] and/or propionyl-CoA carboxylase subunitβ\[PCCB\] mutations). * Participant provided informed consent (and assent, as applicable). * Medical records with sufficient data to support medical encounter for CEC adjudication (MEA adjudication) must be available dating back to birth or 01 January 2015, whichever occurs later. Participants born prior to 2015…
Locations (29)
- University of Stanford Medical CenterStanford, California
- Johns Hopkins UniversityBaltimore, Maryland
- University of Michigan HospitalAnn Arbor, Michigan
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania
- Texas Children's HospitalHouston, Texas
- Seattle Children's HospitalSeattle, Washington