Clinical Trial

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

Recruiting Phase 1/2
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Summary
This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\<1 year of age).
Protocol Amendment History 27 amendments
This ClinicalTrials.gov record has been amended 27 times since 2019-11-07; most recent amendment 2026-01-20.
Status change: Not Yet Recruiting → Recruiting 2021-03-11
Trial Details
NCT Number NCT04159103
Lead Sponsor ModernaTX, Inc.
Conditions Propionic Acidemia
Enrollment 77 participants
Start Date 2021-04-15
Primary Completion 2027-08-31 (estimated)
Study Completion 2027-08-31 (estimated)
Updated on ClinicalTrials.gov 2026-01-22