Skip to main content
OpenTrials
Recruiting

NCT Number: NCT05130437

A Study to Assess 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).

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Propionic Acidemia Acid-Base Imbalance Acidosis Amino Acid Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

1 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Hospital For Sick Children, Toronto, Ontario, Canada

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

mRNA-3927

Biological

mRNA-3927 dispersion for IV infusion

Primary outcomes

  1. 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)

Secondary outcomes

  1. Annualized Frequency of Investigator-reported Metabolic Decompensation Events (MDEs)

    Time frame: Baseline through End of Study Visit (up to 8 years)

  2. Annualized Frequency of Investigator-reported MDE-related Hospitalizations

    Time frame: Baseline through End of Study Visit (up to 8 years)

  3. Annualized Frequency of Investigator-reported PA-related Hospitalizations

    Time frame: Baseline through End of Study Visit (up to 8 years)

  4. Annualized Frequency of Investigator-reported PA-related Urgent Healthcare Encounters

    Time frame: Baseline through End of Study Visit (up to 8 years)

Study contacts

Contact information is provided by the study sponsor or research team.

Moderna WeCare Team

CONTACT

[email protected]

1-866-663-3762

Sponsors and collaborators

Lead sponsor

ModernaTX, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2029
Study completion
2031
First posted
Nov 23, 2021
Registry last updated
Mar 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.