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New mRNA therapy aims to control rare metabolic disease Long-Term

NCT ID NCT05295433

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 is testing the long-term safety of an mRNA therapy called mRNA-3705 in people with methylmalonic acidemia (MMA), a rare genetic disorder that prevents the body from breaking down certain proteins and fats. Participants have already been in earlier studies of this drug. The goal is to see if the treatment remains safe and helps control the disease over time.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
mRNA-3705 (a messenger RNA therapy that helps produce a missing enzyme)
What this could lead to
If successful, this could show that mRNA-3705 is safe for long-term use and helps manage methylmalonic acidemia, potentially reducing metabolic crises.
What could go wrong
This is an early-phase extension study with only 56 participants, so results may not apply to everyone. Long-term safety and effectiveness are still uncertain.

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 56 people

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

Started

Mar 2022

Expected to finish

Apr 2034

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: * Completed the assigned dose regimen treatment time period in other clinical studies of mRNA-3705 or is eligible for early transition to this study because they missed more than 3 consecutive doses of study drug due to coronavirus disease 2019 (COVID-19) vaccination during Study mRNA-3705-P101 Part 1. * Completed the End of treatment (EOT) Visit (or End of Study Visit in the case of unscheduled dosing) in Study mRNA-3705-P101 within 10 days of their first dose of mRNA-3705 in this extension study. Exclusion Criteria: * Not expected to receive clinical benefit from continued mRNA-3705 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. NOTE: Other inclusion and exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

hereditary disease inborn errors of metabolism methylmalonic acidemia methylmalonic aciduria due to methylmalonyl-CoA mutase deficiency

As listed by the trial registrant

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

Methylmalonic acidemia

How to take part

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

  1. The places running it

    11 sites in 6 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

  • Altman Clinical and Translational Research Institution

    RECRUITING

    San Diego, California, 92037, United States

  • Birmingham Children's Hospital NHS Foundation Trust

    TERMINATED

    Birmingham, B4 6NH, United Kingdom

  • Erasmus MC

    RECRUITING

    Rotterdam, 3015 AA, Netherlands

  • Hospital For Sick Children

    RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

  • Hospital Universitario 12 de Octubre

    RECRUITING

    Madrid, 28041, Spain

  • Hospital Universitario Cruces

    RECRUITING

    Barakaldo, Vizcaya, 48903, Spain

  • Hôpital Necker - Enfants Malades APHP

    RECRUITING

    Paris, 75015, France

  • Lucile Packard Children's Hospital at Stanford

    RECRUITING

    Palo Alto, California, 94304, United States

  • Royal Manchester Childrens Hospital

    RECRUITING

    Manchester, M13 9WL, United Kingdom

  • Stollery Children's Hospital University of Alberta

    RECRUITING

    Edmonton, Alberta, T6G 2R7, Canada

  • UCLA Medical Center

    RECRUITING

    Los Angeles, California, 90095, United States

  • Universitair Medisch Centrum Utrecht

    RECRUITING

    Utrecht, 3584 CX, Netherlands

More trials for these conditions

Other studies related to the condition(s) this trial covers.