RecruitingNCT05769621
A Retrospective Study to Characterize Participants With Propionic Acidemia
A Retrospective Cohort Study to Characterize Propionic Acidemia Patients
Sponsor
ModernaTX, Inc.
Enrollment
60 participants
Start Date
Jun 16, 2023
Study Type
OBSERVATIONAL
Conditions
Summary
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
Eligibility
Min Age: 2 Years
Inclusion Criteria13
- 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 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.
- Must have a confirmed age (months) of disease onset (early or late, where early is defined as the neonatal period).
- Censoring Criteria:
- Participants medical records meeting the following censoring criterion will not be abstracted:
- Participation in a clinical study of any investigational agent.
- Received gene therapy treatment
- Confirmed organ transplantation
- Investigator is no longer able to obtain relevant clinical information of the participant.
- Death
Exclusion Criteria1
- \- Participation (prior or ongoing) in clinical study mRNA-3927-P101 (NCT04159103) or mRNA-3927-P101-EXT (NCT05130437).
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Locations(29)
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NCT05769621
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