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This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PA Participants | Participants with PA who meet all eligibility criteria for medical record abstraction. |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Annualized Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs) | Up to 10 years |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
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Inclusion Criteria:
Participants' medical records are eligible for abstraction only if all of the following criteria apply:
Censoring Criteria:
Participants medical records meeting the following censoring criterion will not be abstracted:
Exclusion Criteria:
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).
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Individuals of a minimum age of 2 years with confirmed diagnosis of PA.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Moderna WeCare Team | Contact | +1-866-663-3762 | WeCareClinicalTrials@modernatx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Stanford Medical Center | Not yet recruiting | Stanford | California | 94305-2200 | United States | |
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| 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 |
| Johns Hopkins University |
| Recruiting |
| Baltimore |
| Maryland |
| 21287-0010 |
| United States |
| University of Michigan Hospital | Active, not recruiting | Ann Arbor | Michigan | 48109-5000 | United States |
| Children's Hospital of Philadelphia | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
| Texas Children's Hospital | Terminated | Houston | Texas | 77030-2614 | United States |
| Seattle Children's Hospital | Recruiting | Seattle | Washington | 98105-3901 | United States |
| Royal Children's Hospital Melbourne | Withdrawn | Parkville | Victoria | 3052 | Australia |
| Stollery Children's Hospital University of Alberta | Withdrawn | Edmonton | Alberta | T6G 2B7 | Canada |
| CHU Toulouse | Recruiting | Toulouse | Haute-Garonne | 31300 | France |
| CHRU Nancy Hôpital des Enfants | Withdrawn | Vandœuvre-lès-Nancy | Meurthe-et-Moselle | 54511 | France |
| Hôpital Necker - Enfants Malades | Recruiting | Paris | Paris | 75743 | France |
| CHU de Marseille - Hôpital de la Timone | Recruiting | Marseille | 13386 | France |
| Azienda Ospedaliera Universitaria Federico II | Recruiting | Naples | Campania | 80131 | Italy |
| ASST di Monza - Azienda Ospedaliera San Gerardo | Active, not recruiting | Monza | Monza A Brianza | 20900 | Italy |
| Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN | Recruiting | Florence | Tuscany | 50139 | Italy |
| Fujita Health University Hospital | Not yet recruiting | Toyoake | Aichi-ken | 470-1192 | Japan |
| The Jikei University School of Medicine | Not yet recruiting | Minato | Tokyo | 105-8471 | Japan |
| Erasmus MC | Not yet recruiting | Rotterdam | South Holland | 3015 GD | Netherlands |
| Hospital Sant Joan de Deu | Recruiting | Esplugues de Llobregat | Barcelona | 8950 | Spain |
| Hospital Universitario Marques de Valdecilla | Recruiting | Santander | Cantabria | 39008 | Spain |
| Hospital Universitario Cruces | Recruiting | Barakaldo | 48903 | Spain |
| Hospital Universitario Raymon y Cajal | Recruiting | Madrid | 28034 | Spain |
| Hospital Universitario 12 de Octubre | Active, not recruiting | Madrid | 28041 | Spain |
| Hospital Universitario Virgen del Rocio - PPDS | Recruiting | Seville | 41013 | Spain |
| Hospital Universitari I Politecnic La Fe de Valencia | Recruiting | Valencia | 46026 | Spain |
| Victoria Hospital | Withdrawn | Kirkcaldy | Fife | KY2 5AH | United Kingdom |
| St Mary's Hospital - PPDS | Active, not recruiting | Manchester | Lancashire | United Kingdom |
| Great Ormond Street Hospital | Active, not recruiting | City of London | London | United Kingdom |
| University Hospital Birmingham | Recruiting | Birmingham | B29 6JD | United Kingdom |
| ID | Term |
|---|---|
| D056693 | Propionic Acidemia |
| ID | Term |
|---|---|
| D000592 | Amino Acid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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