Propionic Acidemia Clinical Trial
Official title:
A Retrospective Cohort Study to Characterize Propionic Acidemia Patients
This is a non-interventional, global, multicenter, retrospective cohort study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).
Status | Recruiting |
Enrollment | 100 |
Est. completion date | March 24, 2026 |
Est. primary completion date | March 24, 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | Abstraction Criteria: Participants' medical records are eligible for abstraction only if all of the following criteria apply: - Availability of evidence supportive of the participant having received a physician's diagnosis of PA. - Participant provided informed consent or assent, as applicable. - Participant has had at least one MDE in a consecutive 24-month period within the entire abstraction timeframe. Censoring Criteria: Participants medical records meeting the following censoring criterion will not be abstracted: - Participation in a clinical study of mRNA-3927 or another investigational agent. |
Country | Name | City | State |
---|---|---|---|
Australia | Royal Children's Hospital Melbourne | Melbourne | Victoria |
Canada | Stollery Children's Hospital University of Alberta | Edmonton | Alberta |
France | CHU de Marseille - Hôpital de la Timone | Marseille | |
France | Hôpital Necker - Enfants Malades | Paris Cedex 15 | Paris |
France | CHU Toulouse | Toulouse | |
France | CHRU Nancy Hôpital des Enfants | VandÅ“uvre-lès-Nancy | Meurthe-et-Moselle |
Italy | Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN | Firenze | Toscana |
Italy | ASST di Monza - Azienda Ospedaliera San Gerardo | Monza | Monza A Brianza |
Italy | Azienda Ospedale Pediatrico Bambino Gesu - INCIPIT - PIN | Roma | Lazio |
Netherlands | Erasmus MC | Rotterdam | |
Spain | Hospital Universitario Cruces | Barakaldo | |
Spain | Hospital Sant Joan de Deu | Barcelona | |
Spain | Hospital Universitario 12 de Octubre | Madrid | |
Spain | Hospital Universitario Raymon y Cajal | Madrid | |
Spain | Hospital Universitario Marques de Valdecilla | Santander | Cantabria |
Spain | Hospital Universitario Virgen del Rocio - PPDS | Sevilla | |
Spain | Hospital Universitari I Politecnic La Fe de Valencia | Valencia | |
United Kingdom | University Hospital Birmingham | Birmingham | |
United Kingdom | Great Ormond Street Hospital | City of London | London |
United Kingdom | Victoria Hospital | Kirkcaldy | Fife |
United Kingdom | St Mary's Hospital - PPDS | Manchester | Lancashire |
United States | University of Michigan Hospital | Ann Arbor | Michigan |
United States | Boston Children's Hospital | Boston | Massachusetts |
United States | Texas Children's Hospital | Houston | Texas |
United States | Johns Hopkins University | Lutherville | Maryland |
United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | University of Utah Health Care | Salt Lake City | Utah |
United States | Seattle Children's Hospital | Seattle | Washington |
United States | University of Stanford Medical Center | Stanford | California |
Lead Sponsor | Collaborator |
---|---|
ModernaTX, Inc. |
United States, Australia, Canada, France, Italy, Netherlands, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of Annualized Metabolic Decompensation Events (MDEs) | Up to 10 years | ||
Secondary | Annualized MDE Duration | Up to 10 years | ||
Secondary | Number of Participants with MDE Symptoms | MDE symptoms may include persistent vomiting, anorexia/failure to feed, lethargy or increased seizure activity. | Up to 10 years | |
Secondary | Number of Annualized Hospitalizations Related to PA | Up to 10 years | ||
Secondary | Annualized Duration of Hospitalizations Related to PA | Up to 10 years | ||
Secondary | Number of Annualized Acute Healthcare Visits Related to PA | Up to 10 years | ||
Secondary | Number of Participants who Received Medication During and Within ± 3 Days of an MDE | Medications for standard long-term and acute management of PA, which may include, but are not limited to, anesthesia, antibiotics/antifungals, anticoagulation medications, antiepileptics, biotin supplementation, hydration treatment, PA treatment, pain medications, and transfusion related medications. | Up to 10 years | |
Secondary | Number of Participants Requiring Dietary Management | Dietary management includes the following:
Prescribed protein intake during metabolic stability and during MDEs Standard dietary management and sick-day diet |
Up to 10 years | |
Secondary | Change from Baseline in Biomarkers | Up to 10 years |
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