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MRNA therapy aims to tame rare metabolic disease

NCT ID NCT04159103

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

First seen Oct 31, 2025 · Last updated May 01, 2026 · Updated 29 times

Summary

This study tests an experimental mRNA medicine called mRNA-3927 for people with propionic acidemia, a rare genetic condition that causes dangerous metabolic crises. The trial enrolls about 77 participants aged 1 year and older to evaluate safety and whether the drug can reduce the number of severe episodes. The approach uses mRNA to help the body produce a missing enzyme, aiming to control the disease rather than cure it.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

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

    Contact Email: •••••@•••••

  • 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

    Contact Email: •••••@•••••

  • Erasmus MC

    RECRUITING

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Fujita Health University Hospital

    RECRUITING

    Toyoake-shi, Aichi-ken, 470-1192, Japan

    Contact Email: •••••@•••••

    Contact

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    RECRUITING

    London, WC1N 3JH, United Kingdom

    Contact Email: •••••@•••••

    Contact

  • Hospital For Sick Children

    RECRUITING

    Toronto, Ontario, M5G 1X8, Canada

    Contact Email: •••••@•••••

  • 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

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • 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

    Contact Email: •••••@•••••

  • Stollery Children's Hospital University of Alberta

    RECRUITING

    Edmonton, Alberta, T6G 2R7, Canada

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Texas Children's Hospital

    RECRUITING

    Houston, Texas, 77030, United States

    Contact Email: •••••@•••••

  • Tohoku University Hospital

    RECRUITING

    Sendai, Miyagi, 980-8574, Japan

    Contact Email: •••••@•••••

    Contact

  • 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

    Contact Email: •••••@•••••

  • 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

Conditions

Explore the condition pages connected to this study.

PROPIONIC ACIDEMIA