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

NCT04159103 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 77

Last updated 2026-01-22

No results posted yet for this study

Summary

This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination 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).

Conditions

Interventions

BIOLOGICAL

mRNA-3927

mRNA-3927 dispersion for IV infusion

Sponsors & Collaborators

  • ModernaTX, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-04-15
Primary Completion
2027-08-31
Completion
2027-08-31
FDA Drug
Yes

Countries

  • United States
  • Canada
  • France
  • Japan
  • Netherlands
  • Saudi Arabia
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04159103 on ClinicalTrials.gov