Recruiting

Observational Study

Sponsor:

Moderna

Code:

NCT05769621

Conditions

Propionic Acidemia

Eligibility Criteria

Sex: All

Age: 2+

Healthy Volunteers: Not accepted

Study Details

Brief summary:

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

Conditions

Propionic Acidemia

Study ID

NCT05769621

Start date

Jun 16, 2023

Status verified date

Feb, 2026

Completion date

Sep 30, 2026

Anticipated

Primary completion date

Sep 30, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2+

Healthy Volunteers: Not accepted

Inclusion Criteria:

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

Exclusion Criteria:

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).

Study Design

Enrollment

60 participants

Anticipated

Interventions and Outcome Measures

Arms

PA Participants

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

Primary outcome measure

  • Number of Annualized Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs) [ Time Frame: Up to 10 years ]

Central Contacts and Locations

Central contacts

Locations

Johns Hopkins University

Recruiting

Baltimore, Maryland, United States, 21287-0010

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Seattle Children's Hospital

Recruiting

Seattle, Washington, United States, 98105-3901

More Information

Sponsor

ModernaTX, Inc.

Last update posted

Feb 5, 2026

Last verified

Feb, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-18. This information was provided to ClinicalTrials.gov by ModernaTX, Inc. on 2026-02-05.