A Retrospective Study to Characterize Participants With Propionic 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

Qualifiers

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 design

Treatments tested in this trial

  • Not listed

Trial groups

60 Participants
are grouped into 1 trial group

Locations

29
Australia
Royal Children's Hospital Melbourne3052, ParkvilleVictoria, Australia
Canada
Stollery Children's Hospital University of AlbertaT6G 2B7, EdmontonAlberta, Canada
France
CHU Toulouse31300, ToulouseHaute-Garonne, France
CHRU Nancy Hôpital des Enfants54511, Vandœuvre-lès-NancyMeurthe-et-Moselle, France

Sponsors and collaborators

ModernaTX, Inc.

Lead sponsor