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 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 ≥1 year of age at the time of consent/assent (Inclusion of the first 2 participants entering the study at the Dose Escalation stage will be restricted to individuals age ≥8 years)
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 milliliter (mL)/minute/1.73 m^2 as estimated by Schwartz formula; or participants who receive chronic dialysis
History of organ transplantation or planned organ transplantation during the period of study participation
QTc >480 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

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