MRNA therapy aims to tame rare metabolic disorder

NCT ID NCT04159103

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

First seen Oct 31, 2025 · Last updated Jun 23, 2026 · Updated 36 times

Summary

This study tests an experimental mRNA therapy called mRNA-3927 in people with propionic acidemia, a rare genetic condition that causes dangerous metabolic crises. The trial will enroll 77 participants of all ages to find the best dose and check if it safely reduces the frequency of these episodes. If it works, the therapy could help control the disease rather than cure it.

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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

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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

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 confirm benefit. Risks include unknown side effects and the possibility that the therapy may not effectively control the disease long-term.

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