A Retrospective Study to Characterize Participants With Propionic Acidemia

Propionic Acidemia Clinical Trial

Official title:

A Retrospective Cohort Study to Characterize Propionic Acidemia Patients

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05769621
• Other study ID #: mRNA-3927-P002
• Status: Recruiting
• Start date: June 16, 2023
• Est. completion date: March 24, 2026

Study information

• Verified date: February 2024
• Source: ModernaTX, Inc.
• Contact: Moderna Clinical Trials Support Center
• Phone: 1-877-777-7187
• Email: clinicaltrials[@]modernatx.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This is a non-interventional, global, multicenter, retrospective cohort study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).

Description:

This study will abstract participants data from their medical records from approximately 2015-2025. All data will be abstracted retrospectively in 6-month increments.

Recruitment information / eligibility

• 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.

Related Conditions & MeSH terms

• Propionic Acidemia

Locations

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	Vandœuvre-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

Sponsors (1)

Lead Sponsor	Collaborator
ModernaTX, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Canada,  France,  Italy,  Netherlands,  Spain,  United Kingdom, 

Outcome

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