A Retrospective Study to Characterize Participants With Propionic Acidemia and Methylmalonic Acidemia

Moderna

Status: Active, not recruiting

Conditions: Methylmalonic Acidemia; Propionic Acidemia

Study type: Observational

Funder types: Industry

Identifiers

NCT05769621

mRNA-3927-P002

Details and patient eligibility

About

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

Full 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.

Enrollment: 200 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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 or MMA due to MUT deficiency as indicated by documented evidence.
• Participant provided informed consent (and assent, as applicable).
• Participant has had at least one MDE in 2015 or later before eligibility assessment.

Censoring Criteria:

Participants medical records meeting the following censoring criterion will not be abstracted:

• Participation in a clinical study of mRNA-3927, mRNA-3705, or another investigational agent.
• Other reasons of censorship of data include:
• Withdrawal of consent
• Death
• Lost to follow-up
• End of study period

Trial design

200 participants in 2 patient groups

PA Participants

Description:

Participants with PA who meet all eligibility criteria for medical record abstraction.

MMA Participants

Description:

Participants with MMA who meet all eligibility criteria for medical record abstraction.