A Retrospective Study to Characterize Participants With Propionic Acidemia

Study Overview
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Brief Summary
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This is a non-interventional, global, multicenter, retrospective cohort study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).

Detailed Description
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This study will abstract participants data from their medical records from approximately 2015-2025. All data will be abstracted retrospectively in 6-month increments.

Official Title
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A Retrospective Cohort Study to Characterize Propionic Acidemia Patients

Conditions
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Propionic Acidemia

Participation Criteria
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Eligibility Criteria
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Abstraction Criteria: Participants' medical records are eligible for abstraction only if all of the following criteria apply: Availability of evidence supportive of the participant having received a physician's diagnosis of PA. Participant provided informed consent or assent, as applicable. Participant has had at least one MDE in a consecutive 24-month period within the entire abstraction timeframe. Censoring Criteria: Participants medical records meeting the following censoring criterion will not be abstracted: - Participation in a clinical study of mRNA-3927 or another investigational agent.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| PA Participants<br>Participants with PA who meet all eligibility criteria for medical record abstraction. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Annualized Metabolic Decompensation Events (MDEs) |  | Up to 10 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annualized MDE Duration |  | Up to 10 years | 
| Number of Participants with MDE Symptoms | MDE symptoms may include persistent vomiting, anorexia/failure to feed, lethargy or increased seizure activity. | Up to 10 years | 
| Number of Annualized Hospitalizations Related to PA |  | Up to 10 years | 
| Annualized Duration of Hospitalizations Related to PA |  | Up to 10 years | 
| Number of Annualized Acute Healthcare Visits Related to PA |  | Up to 10 years | 
| Number of Participants who Received Medication During and Within ± 3 Days of an MDE | Medications for standard long-term and acute management of PA, which may include, but are not limited to, anesthesia, antibiotics/antifungals, anticoagulation medications, antiepileptics, biotin supplementation, hydration treatment, PA treatment, pain medications, and transfusion related medications. | Up to 10 years | 
| Number of Participants Requiring Dietary Management | Dietary management includes the following: Prescribed protein intake during metabolic stability and during MDEs Standard dietary management and sick-day diet | Up to 10 years | 
| Change from Baseline in Biomarkers |  | Up to 10 years |