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Recruiting NCT05769621

A Retrospective Study to Characterize Participants With Propionic Acidemia

Conditions: Propionic Acidemia

Sponsor: ModernaTX, Inc.

trial.available_in: БГ
Overview
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
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.
Who can participate
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).
Locations 9
Australia (1)
Royal Children's Hospital Melbourne
Parkville , Victoria
WITHDRAWN
Canada (1)
Stollery Children's Hospital University of Alberta
Edmonton , Alberta
WITHDRAWN
France (1)
CHU Toulouse
Toulouse , Haute-Garonne
Italy (1)
Azienda Ospedaliera Universitaria Federico II
Naples , Campania
Japan (1)
Fujita Health University Hospital
Toyoake , Aichi-ken
NOT_YET_RECRUITING
Netherlands (1)
Erasmus MC
Rotterdam , South Holland
NOT_YET_RECRUITING
Spain (1)
Hospital Sant Joan de Deu
Esplugues de Llobregat , Barcelona
United Kingdom (1)
Victoria Hospital
Kirkcaldy , Fife
WITHDRAWN
United States (1)
University of Stanford Medical Center
Stanford , California
NOT_YET_RECRUITING
Technical details
Status
Recruiting
Study type
OBSERVATIONAL
Sex
Male and female
Minimum age
2 Years
Healthy volunteers
No
Start date
16.06.2023
Completion date
30.09.2026
Registry ID
NCT05769621
Source
anzctr
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