A Retrospective Study to Characterize Participants With Propionic Acidemia
- Conditions
- Propionic Acidemia
- Registration Number
- NCT05769621
- Lead Sponsor
- ModernaTX, Inc.
- Brief Summary
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
- Detailed 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 60
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).
- Participants who meet any of the censoring criteria listed below may be enrolled into the study if they have a minimum of 2 years of data available for abstraction prior to meeting the censoring criteria and they meet all inclusion criteria based on the data available for abstraction.
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
Participant's medical records will not be eligible for abstraction if the participant meets the following exclusion criteria:
- Participation (prior or ongoing) in clinical study mRNA-3927-P101 (NCT04159103) or mRNA-3927-P101-EXT (NCT05130437).
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of Annualized Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs) Up to 10 years
- Secondary Outcome Measures
Name Time Method Number of CEC-adjudicated MDEs as Assessed by a Pre-Specified Severity Rating Scale Up to 10 years Number of Annualized CEC-adjudicated PA-related Hospitalizations Up to 10 years Number of Annualized CEC-adjudicated MDE-related Hospitalizations Up to 10 years Number of Annualized CEC-adjudicated PA-related Urgent Healthcare Visits Up to 10 years
Trial Locations
- Locations (25)
Azienda Ospedaliera Universitaria Federico II
🇮🇹Napoli, Campania, Italy
University Hospital Birmingham
🇬🇧Birmingham, United Kingdom
University of Stanford Medical Center
🇺🇸Stanford, California, United States
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
University of Michigan Hospital
🇺🇸Ann Arbor, Michigan, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Royal Children's Hospital Melbourne
🇦🇺Parkville, Victoria, Australia
Stollery Children's Hospital University of Alberta
🇨🇦Edmonton, Alberta, Canada
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