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

ModernaTX, Inc. · 2021
ClinicalTrials.gov · Datasets · License: Public Domain · 2021
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genetic-diseasesmetabolic-diseasesmetabolism
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).
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