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New mRNA treatment for rare metabolic disease moves to Long-Term safety check

NCT ID NCT05130437

Disease control Sponsor: ModernaTX, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looks at the long-term safety of an experimental medicine called mRNA-3927 for people with propionic acidemia, a rare genetic disorder that prevents the body from breaking down certain proteins and fats. About 50 participants who were in an earlier study will continue receiving the treatment. The goal is to see if it remains safe and helps control the disease over time.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 50 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2021

Expected to finish

Dec 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Acid-Base Imbalance Acidosis hereditary disease inborn disorder of amino acid metabolism inborn errors of metabolism metabolic disease propionic acidemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Propionic acidemia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    21 sites in 8 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • AP-HM- Hôpital de La Timone

    NOT_YET_RECRUITING

    Marseille, 13005, France

  • Birmingham Women's and Children's NHS Foundation Trust

    COMPLETED

    Birmingham, West Midlands, B4 6NH, United Kingdom

  • Duke University Medical System (Duke Health)

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Erasmus MC -Dr. Molewaterplein 40

    NOT_YET_RECRUITING

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Fujita Health University Hospital

    RECRUITING

    Toyoake-shi, Akita, 470-1192, Japan

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Hospital For Sick Children

    RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

  • Hospital Universitario 12 de Octubre

    NOT_YET_RECRUITING

    Madrid, 28041, Spain

  • Hospital Universitario Cruces

    NOT_YET_RECRUITING

    Barakaldo, Vizcaya, 48903, Spain

  • Hôpital Necker - Enfants Malades

    NOT_YET_RECRUITING

    Paris, 75019, France

  • Icahn School of Medicine at Mount Sinai

    RECRUITING

    New York, New York, 10029, United States

  • King Abdullah Children's Specialist Hospital

    NOT_YET_RECRUITING

    Riyadh, Ar Riya, 14611, Saudi Arabia

  • King Faisal Specialist Hospital & Research Centre

    NOT_YET_RECRUITING

    Riyadh, Ar Riya, 11211, Saudi Arabia

  • Ronald Reagan UCLA Medical Center

    NOT_YET_RECRUITING

    Los Angeles, California, 90095, United States

  • Texas Children's Hospital

    RECRUITING

    Houston, Texas, 77030, United States

  • The Children's Hospital of Philadelphia (CHOP)

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Tohoku University Hospital

    RECRUITING

    Sendai, Miyagi, 980-8574, Japan

  • Universitair Medisch Centrum Utrecht - PPDS

    NOT_YET_RECRUITING

    Utrecht, 3584 CX, Netherlands

  • University Hospital Birmingham NHS Foundation Trust

    RECRUITING

    Birmingham, West Midlands, B15 2TH, United Kingdom

  • University of Michigan Hospitals

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Stanford Medical Center

    RECRUITING

    Palo Alto, California, 94304-1503, United States

  • Willink Biochemical Genetics Unit - Manchester

    RECRUITING

    Manchester, England, M13 9WL, United Kingdom

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