A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

March 20, 2026 updated by: ModernaTX, Inc.

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

The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The study will assess long-term safety of mRNA-3927. Participants with PA who were previously enrolled and completed the end-of-treatment (EOT)/early termination (ET) visit of the mRNA-3927-P101 study will have the option to enroll into this extension study provided all eligibility criteria have been met.

The study will include 2 periods: 1) Treatment Period and 2) Follow-up Period (90 days after the EOT visit). All participants will enter the study receiving mRNA-3927 at the same dose and at the same dosing interval last received in the mRNA-3927-P101 study.

Study Type

Interventional

Enrollment (Estimated)

50

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Recruiting
        • Hospital for Sick Children
  • France
      • Marseille, France, 13005
        • Not yet recruiting
        • AP-HM- Hôpital de La Timone
      • Paris, France, 75019
        • Not yet recruiting
        • Hôpital Necker - Enfants Malades
  • Japan
    • Akita
      • Toyoake-shi, Akita, Japan, 470-1192
        • Recruiting
        • Fujita Health University Hospital
    • Miyagi
      • Sendai, Miyagi, Japan, 980-8574
        • Recruiting
        • Tohoku University Hospital
  • Netherlands
      • Utrecht, Netherlands, 3584 CX
        • Not yet recruiting
        • Universitair Medisch Centrum Utrecht - PPDS
    • South Holland
      • Rotterdam, South Holland, Netherlands, 3015 GD
        • Not yet recruiting
        • Erasmus MC -Dr. Molewaterplein 40
  • Saudi Arabia
    • Ar Riya
      • Riyadh, Ar Riya, Saudi Arabia, 14611
        • Not yet recruiting
        • King Abdullah Children's Specialist Hospital
      • Riyadh, Ar Riya, Saudi Arabia, 11211
        • Not yet recruiting
        • King Faisal Specialist Hospital & Research Centre
  • Spain
      • Madrid, Spain, 28041
        • Not yet recruiting
        • Hospital Universitario 12 de Octubre
    • Vizcaya
      • Barakaldo, Vizcaya, Spain, 48903
        • Not yet recruiting
        • Hospital Universitario Cruces
  • United Kingdom
      • London, United Kingdom, WC1N 3JH
        • Recruiting
        • Great Ormond Street Hospital for Children NHS Foundation Trust
    • England
      • Manchester, England, United Kingdom, M13 9WL
        • Recruiting
        • Willink Biochemical Genetics Unit - Manchester
    • West Midlands
      • Birmingham, West Midlands, United Kingdom, B15 2TH
        • Recruiting
        • University Hospital Birmingham NHS Foundation Trust
      • Birmingham, West Midlands, United Kingdom, B4 6NH
        • Completed
        • Birmingham Women's and Children's NHS Foundation Trust
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Not yet recruiting
        • Ronald Reagan UCLA Medical Center
      • Palo Alto, California, United States, 94304-1503
        • Recruiting
        • University of Stanford Medical Center
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan Hospitals
    • New York
      • New York, New York, United States, 10029
        • Recruiting
        • Icahn School of Medicine at Mount Sinai
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University Medical System (Duke Health)
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • The Children's Hospital of Philadelphia (CHOP)
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • Texas Children's Hospital

Participation Criteria

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.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: mRNA-3927
Participants will receive the applicable dose identified during Study mRNA-3927-P101 (NCT04159103) on Day 1. The dose can be adjusted based on Sponsor recommendation.
Biological: mRNA-3927
mRNA-3927 dispersion for IV infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), and AEs Leading to Discontinuation
Time Frame: Baseline through End of Study Visit (up to 8 years)
Baseline through End of Study Visit (up to 8 years)

Secondary Outcome Measures

Outcome Measure
Time Frame
Annualized Frequency of Investigator-reported Metabolic Decompensation Events (MDEs)
Time Frame: Baseline through End of Study Visit (up to 8 years)
Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported MDE-related Hospitalizations
Time Frame: Baseline through End of Study Visit (up to 8 years)
Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported PA-related Hospitalizations
Time Frame: Baseline through End of Study Visit (up to 8 years)
Baseline through End of Study Visit (up to 8 years)
Annualized Frequency of Investigator-reported PA-related Urgent Healthcare Encounters
Time Frame: Baseline through End of Study Visit (up to 8 years)
Baseline through End of Study Visit (up to 8 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

ModernaTX, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 9, 2021

Primary Completion (Estimated)

December 4, 2029

Study Completion (Estimated)

December 4, 2031

Study Registration Dates

First Submitted

November 3, 2021

First Submitted That Met QC Criteria

November 18, 2021

First Posted (Actual)

November 23, 2021

Study Record Updates

Last Update Posted (Actual)

March 23, 2026

Last Update Submitted That Met QC Criteria

March 20, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • mRNA-3927-P101-EXT
  • 2022-502911-12-00 (Other Identifier: Clinical Trials Information System (CTIS))

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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