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Propionic Acidemia clinical trials at University of California Health

1 research study open to eligible people

Showing trials for
  • mRNA-3927 in Participants With Propionic Acidemia

    open to eligible people ages 1 year and up

    This First-in-Human (FIH) Phase 1/2 study will evaluate the safety, pharmacological activity, and efficacy of mRNA-3927 in participants 1 year of age and older with genetically confirmed propionic acidemia (PA). This study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the optimal dose of mRNA-3927 in participants with PA. After establishing a dose with acceptable safety and pharmacodynamic (PD) response in a Dose Optimization Part (Part 1), additional participants will be enrolled into the study in a Dose Expansion Part (Part 2) to allow for further characterization of the efficacy, safety, and PD of mRNA-3927.

    at UCLA

Our lead scientists for Propionic Acidemia research studies include .

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