A Retrospective Study to Characterize Participants With Propionic Acidemia
Recruiting now
Conditions studied: Propionic Acidemia
In brief
This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
Key facts
- Study ID
- NCT05769621
- Run by
- ModernaTX, Inc.
- People needed
- 60
- Starts
- 2023-06-16
- Expected to finish
- 2026-09-30
- Last updated by the study team
- 2026-02-05
Who can join
Age: 2 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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).
Where it is running
- University Hospital Birmingham — Birmingham, United Kingdom (enrolling)
- Johns Hopkins University — Baltimore, Maryland, United States (enrolling)
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
- Seattle Children's Hospital — Seattle, Washington, United States (enrolling)
- CHU Toulouse — Toulouse, Haute-Garonne, France (enrolling)
- Hôpital Necker - Enfants Malades — Paris, Paris, France (enrolling)
- CHU de Marseille - Hôpital de la Timone — Marseille, France (enrolling)
- Azienda Ospedaliera Universitaria Federico II — Naples, Campania, Italy (enrolling)
- Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN — Florence, Tuscany, Italy (enrolling)
- Hospital Sant Joan de Deu — Esplugues de Llobregat, Barcelona, Spain (enrolling)
- Hospital Universitario Marques de Valdecilla — Santander, Cantabria, Spain (enrolling)
- Hospital Universitario Cruces — Barakaldo, Spain (enrolling)
- Hospital Universitario Raymon y Cajal — Madrid, Spain (enrolling)
- Hospital Universitario Virgen del Rocio - PPDS — Seville, Spain (enrolling)
- Hospital Universitari I Politecnic La Fe de Valencia — Valencia, Spain (enrolling)
- Fujita Health University Hospital — Toyoake, Aichi-ken, Japan
- University of Michigan Hospital — Ann Arbor, Michigan, United States
- The Jikei University School of Medicine — Minato, Tokyo, Japan
- Texas Children's Hospital — Houston, Texas, United States
- Erasmus MC — Rotterdam, South Holland, Netherlands
- Royal Children's Hospital Melbourne — Parkville, Victoria, Australia
- Stollery Children's Hospital University of Alberta — Edmonton, Alberta, Canada
- Great Ormond Street Hospital — City of London, London, United Kingdom
- CHRU Nancy Hôpital des Enfants — Vandœuvre-lès-Nancy, Meurthe-et-Moselle, France
- University of Stanford Medical Center — Stanford, California, United States
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.