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).
Propionic Acidemia
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).
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
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Ronald Reagan UCLA Medical Center, Los Angeles, California, United States, 90095
Lucile Packard Children's Hospital Stanford, Stanford, California, United States, 94304
Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit, Baltimore, Maryland, United States, 21287
Boston Children's Hospital, Boston, Massachusetts, United States, 02115
University of Michigan Hospitals, Ann Arbor, Michigan, United States, 48109
Icahn School of Medicine at Mount Sinai - Clinical Research Unit, New York, New York, United States, 10029
Duke University Medical System (Duke Health), Durham, North Carolina, United States, 27710
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 45229
Children's Hospital of Philadelphia (CHOP), Philadelphia, Pennsylvania, United States, 19104
Texas Children's Hospital, Houston, Texas, United States, 77030
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
For general information about clinical research, read Learn About Studies.
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ModernaTX, Inc.,
2027-01-06