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A Retrospective Study to Characterize Participants With Propionic Acidemia
Sponsor: ModernaTX, Inc.
Summary
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
Official title: A Retrospective Cohort Study to Characterize Propionic Acidemia Patients
Key Details
Gender
All
Age Range
2 Years - Any
Study Type
OBSERVATIONAL
Enrollment
60
Start Date
2023-06-16
Completion Date
2026-09-30
Last Updated
2026-02-05
Healthy Volunteers
No
Conditions
Locations (29)
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
CHRU Nancy Hôpital des Enfants
Vandœuvre-lès-Nancy, Meurthe-et-Moselle, France
Hôpital Necker - Enfants Malades
Paris, Paris, France
CHU de Marseille - Hôpital de la Timone
Marseille, France
Azienda Ospedaliera Universitaria Federico II
Naples, Campania, Italy
ASST di Monza - Azienda Ospedaliera San Gerardo
Monza, Monza A Brianza, Italy
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
Florence, Tuscany, Italy
Fujita Health University Hospital
Toyoake, Aichi-ken, Japan
The Jikei University School of Medicine
Minato, Tokyo, Japan
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
Victoria Hospital
Kirkcaldy, Fife, United Kingdom
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