A Retrospective Study to Characterize Participants With Propionic Acidemia

Sponsor

ModernaTX, Inc. (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05769621

Collaborator

(none)

100

Enrollment

35.8

Anticipated Duration (Months)

Study Details

Study Description

Brief 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).

Condition or Disease	Intervention/Treatment	Phase
Propionic Acidemia

Detailed 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.

Study Design

Study Type:

Observational

Anticipated Enrollment :

100 participants

Observational Model:

Cohort

Time Perspective:

Retrospective

Official Title:

A Retrospective Cohort Study to Characterize Propionic Acidemia Patients

Anticipated Study Start Date :

Mar 30, 2023

Anticipated Primary Completion Date :

Mar 24, 2026

Anticipated Study Completion Date :

Mar 24, 2026

Arms and Interventions

Arm	Intervention/Treatment
PA Participants Participants with PA who meet all eligibility criteria for medical record abstraction.

Outcome Measures

Primary Outcome Measures

Number of Annualized Metabolic Decompensation Events (MDEs) [Up to 10 years]

Secondary Outcome Measures

Annualized MDE Duration [Up to 10 years]
Number of Participants with MDE Symptoms [Up to 10 years]
MDE symptoms may include persistent vomiting, anorexia/failure to feed, lethargy or increased seizure activity.
Number of Annualized Hospitalizations Related to PA [Up to 10 years]
Annualized Duration of Hospitalizations Related to PA [Up to 10 years]
Number of Annualized Acute Healthcare Visits Related to PA [Up to 10 years]
Number of Participants who Received Medication During and Within ± 3 Days of an MDE [Up to 10 years]
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.
Number of Participants Requiring Dietary Management [Up to 10 years]
Dietary management includes the following: Prescribed protein intake during metabolic stability and during MDEs Standard dietary management and sick-day diet
Change from Baseline in Biomarkers [Up to 10 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

ModernaTX, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

ModernaTX, Inc.

ClinicalTrials.gov Identifier:

NCT05769621

Other Study ID Numbers:

mRNA-3927-P002

First Posted:

Mar 15, 2023

Last Update Posted:

Mar 15, 2023

Last Verified:

Feb 1, 2023

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Propionic Acidemia

Study Results

No Results Posted as of Mar 15, 2023