A Retrospective Study to Characterize Participants With Propionic Acidemia
ConditionPropionic Acidemia
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age2+
SponsorModernaTX, Inc.
About this trial
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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 will require additional information to confirm eligibility.
Experienced at least one MDE in the 24 months preceding the Index Date or experienced at least 3 MDEs within any one 12-month retrospective review period (based on Index Date) dating back to birth or 01 January 2015, whichever occurs later.
Disqualifiers
None
Trial population
Individuals of a minimum age of 2 years with confirmed diagnosis of PA.
Trial design
Design model
Cohort
Time perspective
Retrospective
Treatments tested in this trial
Not listed
Trial groups
60 Participants
are grouped into 1 trial groupGroup A: PA Participants
Trial outcomes
Primary outcomes
1
Number of Annualized Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs)
Time frame
Up to 10 yearsSecondary outcomes
1
Number of CEC-adjudicated MDEs as Assessed by a Pre-Specified Severity Rating Scale
Time frame
Up to 10 years2
Number of Annualized CEC-adjudicated PA-related Hospitalizations
Time frame
Up to 10 years3
Number of Annualized CEC-adjudicated MDE-related Hospitalizations
Time frame
Up to 10 years4
Number of Annualized CEC-adjudicated PA-related Urgent Healthcare Visits
Time frame
Up to 10 yearsSponsors and contacts
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Source ClinicalTrials.gov