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MRNA therapy takes on rare metabolic disease

NCT ID NCT04159103

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests an experimental mRNA therapy called mRNA-3927 for propionic acidemia, a rare genetic disorder that causes dangerous metabolic crises. The trial involves about 77 participants of all ages, starting with older children and adults. Researchers will check safety, find the best dose, and see if it reduces the number of metabolic emergencies.

What this could mean

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

Active substance
mRNA-3927 (a messenger RNA therapy given by IV infusion)
What this could lead to
If successful, this could provide a new treatment option to reduce dangerous metabolic episodes in people with propionic acidemia.
What could go wrong
This is an early-phase trial with only 77 participants, so results may not apply to everyone. The therapy is still experimental, and side effects or lack of effectiveness are possible.

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 77 people

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

Started

Apr 2021

Expected to finish

Aug 2027

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

Children (under 18), adults (18 to 64) and older adults (65 and over)

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: Participants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply: * ≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants in Part 1. * ≥1 year of age at the time of consent/assent if enrolled after the first 2 participants in Part 1. * Confirmed diagnosis of PA based on diagnosis by molecular genetic testing via central laboratory (PCCA and/or PCCB mutations). * Part 2 only: At least one documented MDE in the 12-month period before consent. Participants \<1 Year of Age : * Identification by newborn screening shortly after birth or having suspected PA by presenting with a spectrum of metabolic symptoms, and having a sibling diagnosed with PA. Participant may enter the Screening Period while awaiting genetic testing results, provided that all other eligibility criteria are met but would not be enrolled until diagnosis of PA is confirmed. * For infants in the neonatal intensive care unit (NICU) only: ≥37 weeks gestational age at the time of birth without other conditions/comorbidities that in the opinion of the Investigator may interfere with the interpretation of study results. * Body weight ≥3 kilograms (kg) at Screening. * At least 1 documented PA-related event prior to Screening defined as the following criteria: * Clinical signs of metabolic deterioration consistent with PA (for example, vomiting, not feeding well/poor suck, heavy breathing, lethargy, absence of proper perfusion, abnormal movements including bicycling, abnormal tone, low body temperature, seizure\[s\]), OR * Meeting the criteria of MDE definition, OR * Evidence of laboratory abnormalities as evidenced by at least one of the following: * Metabolic acidosis with elevated anion gap. * Acute hyperammonemia. * Neutropenia or thrombocytopenia. Exclusion Criteria: Participants of all ages are excluded from the study if during Screening any of the following criteria apply: * Any individual with laboratory abnormalities considered to be clinically significant (for example, markedly out of range, associated with clinical symptoms) in the Investigator or Sponsor's opinion that could interfere with or limit the participation in the study. * Estimated glomerular filtration rate (eGFR) \<30 milliliters (mL)/minute/1.73 square meter (m\^2) for participants of all ages receiving chronic dialysis. * History of organ transplantation or planned organ transplantation during the period of study participation. * Corrected QT interval (QTc) \>480 milliseconds (ms) using Bazett's correction. * Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification. * Pregnant or breastfeeding. * Other clinically significant conditions that in the Investigator's opinion could interfere with the safety of the participant, the interpretation of study results, or limit the participation in the study.

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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

    24 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

  • Ann and Robert H Lurie Childrens Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Birmingham Children's Hospital

    COMPLETED

    Birmingham, B4 6NH, United Kingdom

  • Boston Children's Hospital

    COMPLETED

    Boston, Massachusetts, 02115, United States

  • CHU de Marseille - Hôpital de la Timone

    ACTIVE_NOT_RECRUITING

    Marseille, 13005, France

  • Children's Hospital of Philadelphia (CHOP)

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Cincinnati Children's Hospital Medical Center

    COMPLETED

    Cincinnati, Ohio, 45229, United States

  • Duke University Medical System (Duke Health)

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Erasmus MC

    RECRUITING

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Fujita Health University Hospital

    RECRUITING

    Toyoake-shi, Aichi-ken, 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 Sant Joan de Deu - PIN

    ACTIVE_NOT_RECRUITING

    Esplugues de Llobregat, Barcelona, 8950, Spain

  • Hospital Universitario 12 de Octubre

    RECRUITING

    Madrid, 28041, Spain

  • Hospital Universitario Cruces

    RECRUITING

    Barakaldo, Biscay, 48903, Spain

  • Hospital Universitario Virgen del Rocio - PPDS

    ACTIVE_NOT_RECRUITING

    Seville, 41013, Spain

  • Hôpital Necker - Enfants Malades

    RECRUITING

    Paris, 75019, France

  • Icahn School of Medicine at Mount Sinai - Clinical Research Unit

    RECRUITING

    New York, New York, 10029, United States

  • Johns Hopkins Hospital, Adult Outpatient Clinical Research Unit

    COMPLETED

    Baltimore, Maryland, 21287, United States

  • King Abdullah Children's Specialist Hospital

    NOT_YET_RECRUITING

    Riyadh, Ar Riya, 14611, Saudi Arabia

  • King Fahad Medical City

    NOT_YET_RECRUITING

    Riyadh, Ar Riya, 11564, Saudi Arabia

  • King Faisal Specialist Hospital & Research Center - Riyadh

    ACTIVE_NOT_RECRUITING

    Riyadh, Ar Riya, 11211, Saudi Arabia

  • Lucile Packard Children's Hospital Stanford

    RECRUITING

    Stanford, California, 94304, United States

  • National Center for Child Health and Development

    ACTIVE_NOT_RECRUITING

    Tokyo, 113-8519, Japan

  • Nicklaus Children's Hospital

    NOT_YET_RECRUITING

    Miami, Florida, 33155, United States

  • Ronald Reagan UCLA Medical Center

    RECRUITING

    Los Angeles, California, 90095, United States

  • Stollery Children's Hospital University of Alberta

    RECRUITING

    Edmonton, Alberta, T6G 2R7, Canada

  • Texas Children's Hospital

    RECRUITING

    Houston, Texas, 77030, United States

  • Tohoku University Hospital

    RECRUITING

    Sendai, Miyagi, 980-8574, Japan

  • UCSD Altman Clinical and Transalational Research Institute Building

    NOT_YET_RECRUITING

    Los Angeles, California, 90027, United States

  • Universitair Medisch Centrum Utrecht - PPDS

    ACTIVE_NOT_RECRUITING

    Utrecht, 3584 CX, Netherlands

  • University Hospital Birmingham NHS Foundation Trust

    RECRUITING

    Birmingham, B15 2TH, United Kingdom

  • University Hospitals Cleveland Medical Center - 11100 Euclid Ave

    ACTIVE_NOT_RECRUITING

    Cleveland, Ohio, 44106-2624, United States

  • University of Michigan Hospitals

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of South Florida - 12901 Bruce B Downs

    NOT_YET_RECRUITING

    Tampa, Florida, 33606-3603, United States

  • Willink Biochemical Genetics Unit - PPDS

    RECRUITING

    Manchester, M13 9WL, United Kingdom

More trials for these conditions

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