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Long-term safety assessment of mRNA-3927 for propionic acidemia patients

A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

Phase1; Phase2 Interventional ModernaTX, Inc. · NCT05130437

This study is testing the long-term safety and effectiveness of the mRNA-3927 treatment for people with propionic acidemia who have already participated in a previous study.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment50 (estimated)
Ages1 Year and up
SexAll
SponsorModernaTX, Inc. Industry-sponsored
Locations22 sites (Los Angeles, California and 21 other locations)
Trial IDNCT05130437 on ClinicalTrials.gov

What this trial studies

This study evaluates the long-term safety and clinical activity of mRNA-3927 in participants with propionic acidemia who have previously completed the mRNA-3927-P101 study. Participants will receive mRNA-3927 at the same dose and dosing interval as in the prior study. The study consists of a treatment period followed by a follow-up period lasting up to two years after the last dose of the study drug. The aim is to ensure the continued safety and efficacy of mRNA-3927 in this patient population.

Who should consider this trial

Good fit: Ideal candidates are individuals who have previously participated in the mRNA-3927-P101 study and completed the end-of-treatment visit.

Not a fit: Patients who are not expected to receive clinical benefit from continued mRNA-3927 administration, as determined by the investigator, will not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term safety and effectiveness of mRNA-3927 for patients with propionic acidemia.

How similar studies have performed: Other studies involving mRNA therapies have shown promising results, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Participated in Study mRNA-3927-P101.
* Completed the EOT/ET visit in Study mRNA-3927-P101 and enroll in this study such that the first dose in this study is planned to be within 14±3 days of the last dose of mRNA-3927 in the mRNA-3927-P101 study.

Exclusion Criteria:

* Not expected to receive clinical benefit from continued mRNA-3927 administration, in the opinion of the Investigator.
* Any clinical or laboratory abnormality or medical condition that, at the discretion of the Investigator, may put the individual at increased risk by participating in this study.
* History of liver and/or kidney transplant.

Where this trial is running

Los Angeles, California and 21 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Propionic AcidemiamRNA-3927Propionic AciduriaMetabolism, Inborn ErrorsGenetic DiseasesInborn Amino Acid Metabolism, Inborn ErrorsAcidosisAcid-Base Imbalance
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.