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

About

Brief Summary

This First-in-Human (FIH) Phase 1/2 study will evaluate mRNA-3927 in participants 1 year of age and older with genetically confirmed propionic acidemia (PA). The study is designed to characterize baseline biomarker levels followed by assessment of safety, pharmacokinetics (PK), and pharmacodynamics (PD) of different doses of mRNA-3927 in participants affected by PA as part of the Dose Optimization phase.

Primary Purpose

Treatment

Study Type

Interventional

Phase

Phase I/II

Eligibility

Gender

All

Healthy Volunteers

Minimum Age

1 Year

Maximum Age

N/A

Inclusion Criteria:

Participant must be ≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants.
Participant must be ≥1 year of age at the time of consent/assent if enrolled after the first 2 participants.
Confirmed diagnosis of propionic acidemia based on diagnosis by molecular genetic testing (propionyl-CoA carboxylase subunit α [PCCA] and/or propionyl CoA carboxylase subunit β [PCCB] mutations)

Exclusion Criteria:

Estimated glomerular filtration rate <30 milliliters (mL)/minute/1.73 square meter (m^2) as estimated by Schwartz formula for participants < 18 years of age or the Chronic Kidney Disease Epidemiology Collaboration creatinine based formula for participants ≥ 18 years of age or for participants of all ages receiving chronic dialysis.
History of organ transplantation or planned organ transplantation during the period of study participation.
Corrected QT interval (QTc) >480 milliseconds (ms) using Bazett's correction.
Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification.
COVID-19 vaccination (generally 2 doses or a booster) within 6 weeks between their last COVID-19 vaccination dose and first study drug administration.

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