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

NCT ID: NCT05130437

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-11-09

Study Completion Date

2031-12-04

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Propionic Acidemia

Keywords

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mRNA-3927 Propionic Aciduria Metabolism, Inborn Errors Genetic Diseases Inborn Amino Acid Metabolism, Inborn Errors Acidosis Acid-Base Imbalance Metabolic Diseases Organic Acidemias Moderna mRNA

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

mRNA-3927

Intervention Type BIOLOGICAL

mRNA-3927 dispersion for IV infusion

Interventions

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mRNA-3927

mRNA-3927 dispersion for IV infusion

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.
Minimum Eligible Age

1 Year

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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ModernaTX, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Ronald Reagan UCLA Medical Center

Los Angeles, California, United States

Site Status RECRUITING

University of Michigan Hospitals

Ann Arbor, Michigan, United States

Site Status RECRUITING

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Site Status RECRUITING

Duke University Medical System (Duke Health)

Durham, North Carolina, United States

Site Status RECRUITING

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Texas Children's Hospital

Houston, Texas, United States

Site Status RECRUITING

Hospital For Sick Children

Toronto, Ontario, Canada

Site Status RECRUITING

Fujita Health University Hospital

Toyoake-shi, Akita, Japan

Site Status NOT_YET_RECRUITING

Willink Biochemical Genetics Unit - Manchester

Manchester, England, United Kingdom

Site Status RECRUITING

University Hospital Birmingham NHS Foundation Trust

Birmingham, West Midlands, United Kingdom

Site Status RECRUITING

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, West Midlands, United Kingdom

Site Status COMPLETED

Great Ormond Street Hospital for Children NHS Foundation Trust

London, , United Kingdom

Site Status RECRUITING

Countries

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United States Canada Japan United Kingdom

Central Contacts

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Moderna WeCare Team

Role: CONTACT

Phone: 1-866-663-3762

Email: [email protected]

Other Identifiers

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2022-502911-12-00

Identifier Type: OTHER

Identifier Source: secondary_id

mRNA-3927-P101-EXT

Identifier Type: -

Identifier Source: org_study_id