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

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Brief Summary

This First-in-Human (FIH) Phase 1/2 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 genetically confirmed propionic acidemia (PA). After establishing a dose with acceptable safety and pharmacodynamic (PD) response in a Dose Optimization Group (Part 1) in participants ≥1 year of age, additional participants will be enrolled into the study in a Dose Expansion Group (Part 2) to allow for further characterization 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).

Primary Purpose
Treatment
Study Type
Interventional
Phase
Phase 1/Phase 2

Eligibility

Gender
All
Healthy Volunteers
No
Minimum Age
N/A
Maximum Age
N/A

Inclusion Criteria:

Exclusion Criteria:

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Study Stats
Protocol No.
19-002009
Category
Endocrine and Metabolic Disorders
Pediatric and Prenatal Disorders
Contact
Monserrath Campos
Location
  • UCLA Westwood
For Providers
NCT No.
NCT04159103
For detailed technical eligibility, visit ClinicalTrials.gov.