Clinical Trials in Japan / NCT05769621
Recruiting
Observational
A Retrospective Study to Characterize Participants With Propionic Acidemia
NCT05769621 · tracked via the Priya Life Science Japan tracker
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
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)
- Number of Annualized Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs) — Up to 10 years
Trial sites (29)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
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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 Japan