A Retrospective Study to Characterize Participants With Propionic Acidemia
NCT ID: NCT05769621
Last Updated: 2025-09-02
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
60 participants
OBSERVATIONAL
2023-06-16
2026-08-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
RETROSPECTIVE
Study Groups
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PA Participants
Participants with PA who meet all eligibility criteria for medical record abstraction.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* 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 Criteria
2 Years
ALL
No
Sponsors
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ModernaTX, Inc.
INDUSTRY
Responsible Party
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Locations
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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
CHU Toulouse
Toulouse, Haute-Garonne, France
Hôpital Necker - Enfants Malades
Paris, Paris, France
CHU de Marseille - Hôpital de la Timone
Marseille, , France
Azienda Ospedaliera Universitaria Federico II
Napoli, Campania, Italy
ASST di Monza - Azienda Ospedaliera San Gerardo
Monza, Monza A Brianza, Italy
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
Florence, Tuscany, Italy
Erasmus MC
Rotterdam, South Holland, Netherlands
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, Spain
Hospital Universitario Marques de Valdecilla
Santander, Cantabria, Spain
Hospital Universitario Cruces
Barakaldo, , Spain
Hospital Universitario Raymon y Cajal
Madrid, , Spain
Hospital Universitario 12 de Octubre
Madrid, , Spain
Hospital Universitario Virgen del Rocio - PPDS
Seville, , Spain
Hospital Universitari I Politecnic La Fe de Valencia
Valencia, , Spain
St Mary's Hospital - PPDS
Manchester, Lancashire, United Kingdom
Great Ormond Street Hospital
City of London, London, United Kingdom
University Hospital Birmingham
Birmingham, , United Kingdom
Countries
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Other Identifiers
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mRNA-3927-P002
Identifier Type: -
Identifier Source: org_study_id
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