Език: EN EN
← Назад към резултатите
Набира участници NCT05769621

A Retrospective Study to Characterize Participants With Propionic Acidemia

Заболявания: Propionic Acidemia

Спонсор: ModernaTX, Inc.

Налично на: БГ
Обобщение
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
Описание
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.
Кой може да участва
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).
Места на провеждане 9
Австралия (1)
Royal Children's Hospital Melbourne
Parkville , Victoria
WITHDRAWN
Канада (1)
Stollery Children's Hospital University of Alberta
Edmonton , Alberta
WITHDRAWN
Франция (1)
CHU Toulouse
Toulouse , Haute-Garonne
Италия (1)
Azienda Ospedaliera Universitaria Federico II
Naples , Campania
Япония (1)
Fujita Health University Hospital
Toyoake , Aichi-ken
NOT_YET_RECRUITING
Нидерландия (1)
Erasmus MC
Rotterdam , South Holland
NOT_YET_RECRUITING
Испания (1)
Hospital Sant Joan de Deu
Esplugues de Llobregat , Barcelona
Великобритания (1)
Victoria Hospital
Kirkcaldy , Fife
WITHDRAWN
САЩ (1)
University of Stanford Medical Center
Stanford , California
NOT_YET_RECRUITING
Технически детайли
Статус
Набира участници
Вид изследване
OBSERVATIONAL
Пол
Мъже и жени
Минимална възраст
2 Years
Здрави доброволци
Не
Начална дата
16.06.2023
Крайна дата
30.09.2026
Регистрационен номер
NCT05769621
Източник
anzctr
Запитване за медицински туризъм

Информацията е извлечена автоматично от ClinicalTrials.gov. Консултирайте се с вашия лекар преди да предприемете действия.