NCT05769621 A Retrospective Study to Characterize Participants With Propionic Acidemia
| NCT ID | NCT05769621 |
| Status | Recruiting |
| Phase | — |
| Sponsor | ModernaTX, Inc. |
| Condition | Propionic Acidemia |
| Study Type | OBSERVATIONAL |
| Enrollment | 60 participants |
| Start Date | 2023-06-16 |
| Primary Completion | 2026-09-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 60 participants in total. It began in 2023-06-16 with a primary completion date of 2026-09-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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).
Eligibility Criteria
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).
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05769621 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, studying Propionic Acidemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05769621 currently recruiting?
Yes, NCT05769621 is actively recruiting participants. Contact the research team at WeCareClinicalTrials@modernatx.com for enrollment information.
Where is the NCT05769621 trial being conducted?
This trial is being conducted at Stanford, United States, Baltimore, United States, Ann Arbor, United States, Philadelphia, United States and 11 additional locations.
Who is sponsoring the NCT05769621 clinical trial?
NCT05769621 is sponsored by ModernaTX, Inc.. The trial plans to enroll 60 participants.