A Retrospective Study to Characterize Participants With Propionic Acidemia
Start Date
6/16/2023
Completion Date
9/30/2026
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.
Eligibility Criteria
Age Range: 2 years to No maximum
Conditions
Locations
University of Stanford Medical Center
Stanford, California 94305-2200
United States
Johns Hopkins University
Baltimore, Maryland 21287-0010
United States
University of Michigan Hospital
Ann Arbor, Michigan 48109-5000
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
Texas Children's Hospital
Houston, Texas 77030-2614
United States
Seattle Children's Hospital
Seattle, Washington 98105-3901
United States
Royal Children's Hospital Melbourne
Parkville, Victoria 3052
Australia
Stollery Children's Hospital University of Alberta
Edmonton, Alberta T6G 2B7
Canada
CHU Toulouse
Toulouse, Haute-Garonne 31300
France
CHRU Nancy Hôpital des Enfants
Vandœuvre-lès-Nancy, Meurthe-et-Moselle 54511
France
Hôpital Necker - Enfants Malades
Paris, Paris 75743
France
CHU de Marseille - Hôpital de la Timone
Marseille, 13386
France
Azienda Ospedaliera Universitaria Federico II
Naples, Campania 80131
Italy
ASST di Monza - Azienda Ospedaliera San Gerardo
Monza, Monza A Brianza 20900
Italy
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
Florence, Tuscany 50139
Italy
Fujita Health University Hospital
Toyoake, Aichi-ken 470-1192
Japan
The Jikei University School of Medicine
Minato, Tokyo 105-8471
Japan
Erasmus MC
Rotterdam, South Holland 3015 GD
Netherlands
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona 8950
Spain
Hospital Universitario Marques de Valdecilla
Santander, Cantabria 39008
Spain
Hospital Universitario Cruces
Barakaldo, 48903
Spain
Hospital Universitario Raymon y Cajal
Madrid, 28034
Spain
Hospital Universitario 12 de Octubre
Madrid, 28041
Spain
Hospital Universitario Virgen del Rocio - PPDS
Seville, 41013
Spain
Hospital Universitari I Politecnic La Fe de Valencia
Valencia, 46026
Spain
Victoria Hospital
Kirkcaldy, Fife KY2 5AH
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, B29 6JD
United Kingdom