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Clinical Trials in the UK / NCT05769621
Recruiting Observational

A Retrospective Study to Characterize Participants With Propionic Acidemia

NCT05769621 · tracked via the Priya Life Science UK tracker
Sponsor
ModernaTX, Inc.
Phase
Observational
Started
2023-06-16
Last updated
2026-02-05

Condition(s) studied

Propionic Acidemia

Study summary

This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).

Eligibility

Sex
ALL
Min age
2 Years
Max age
Healthy volunteers
No
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).

Primary outcome measure(s)

Trial sites (29)

FacilityCityRegionStatus
University of Stanford Medical Center Stanford California Not Yet Recruiting
Johns Hopkins University Baltimore Maryland Recruiting
University of Michigan Hospital Ann Arbor Michigan Active Not Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
Texas Children's Hospital Houston Texas Terminated
Seattle Children's Hospital Seattle Washington Recruiting
Royal Children's Hospital Melbourne Parkville Victoria Withdrawn
Stollery Children's Hospital University of Alberta Edmonton Alberta Withdrawn
CHU Toulouse Toulouse Haute-Garonne Recruiting
CHRU Nancy Hôpital des Enfants Vandœuvre-lès-Nancy Meurthe-et-Moselle Withdrawn
Hôpital Necker - Enfants Malades Paris Paris Recruiting
CHU de Marseille - Hôpital de la Timone Marseille France Recruiting
Azienda Ospedaliera Universitaria Federico II Naples Campania Recruiting
ASST di Monza - Azienda Ospedaliera San Gerardo Monza Monza A Brianza Active Not Recruiting
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN Florence Tuscany Recruiting
Fujita Health University Hospital Toyoake Aichi-ken Not Yet Recruiting
The Jikei University School of Medicine Minato Tokyo Not Yet Recruiting
Erasmus MC Rotterdam South Holland Not Yet Recruiting
Hospital Sant Joan de Deu Esplugues de Llobregat Barcelona Recruiting
Hospital Universitario Marques de Valdecilla Santander Cantabria Recruiting
Hospital Universitario Cruces Barakaldo Spain Recruiting
Hospital Universitario Raymon y Cajal Madrid Spain Recruiting
Hospital Universitario 12 de Octubre Madrid Spain Active Not Recruiting
Hospital Universitario Virgen del Rocio - PPDS Seville Spain Recruiting
Hospital Universitari I Politecnic La Fe de Valencia Valencia Spain Recruiting
Victoria Hospital Kirkcaldy Fife Withdrawn
St Mary's Hospital - PPDS Manchester Lancashire Active Not Recruiting
Great Ormond Street Hospital City of London London Active Not Recruiting
University Hospital Birmingham Birmingham United Kingdom Recruiting

More ModernaTX, Inc. trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05769621 on ClinicalTrials.gov ↗ ← All trials in the UK