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NCT05769621Recruiting

A Retrospective Study to Characterize Participants With Propionic Acidemia

ModernaTX, Inc.

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

Inclusion Criteria: 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 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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Conditions

Propionic Acidemia

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