New mRNA therapy aims to control rare metabolic disease Long-Term
NCT ID NCT05130437
First seen Jan 06, 2026 · Last updated May 01, 2026 · Updated 15 times
Summary
This study is for people with propionic acidemia who have already taken part in a previous study of the experimental drug mRNA-3927. The main goal is to see if the drug is safe over a longer period of time. Researchers will also track how often participants have serious metabolic events or need hospital care. About 50 people will take part.
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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.
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
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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AP-HM- Hôpital de La Timone
NOT_YET_RECRUITINGMarseille, 13005, France
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Birmingham Women's and Children's NHS Foundation Trust
COMPLETEDBirmingham, West Midlands, B4 6NH, United Kingdom
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Duke University Medical System (Duke Health)
RECRUITINGDurham, North Carolina, 27710, United States
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Erasmus MC -Dr. Molewaterplein 40
NOT_YET_RECRUITINGRotterdam, South Holland, 3015 GD, Netherlands
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Fujita Health University Hospital
RECRUITINGToyoake-shi, Akita, 470-1192, Japan
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Great Ormond Street Hospital for Children NHS Foundation Trust
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Hospital For Sick Children
RECRUITINGToronto, Ontario, M5G 1X8, Canada
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Hospital Universitario 12 de Octubre
NOT_YET_RECRUITINGMadrid, 28041, Spain
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Hospital Universitario Cruces
NOT_YET_RECRUITINGBarakaldo, Vizcaya, 48903, Spain
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Hôpital Necker - Enfants Malades
NOT_YET_RECRUITINGParis, 75019, France
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Icahn School of Medicine at Mount Sinai
RECRUITINGNew York, New York, 10029, United States
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King Abdullah Children's Specialist Hospital
NOT_YET_RECRUITINGRiyadh, Ar Riya, 14611, Saudi Arabia
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King Faisal Specialist Hospital & Research Centre
NOT_YET_RECRUITINGRiyadh, Ar Riya, 11211, Saudi Arabia
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Ronald Reagan UCLA Medical Center
NOT_YET_RECRUITINGLos Angeles, California, 90095, United States
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Texas Children's Hospital
RECRUITINGHouston, Texas, 77030, United States
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The Children's Hospital of Philadelphia (CHOP)
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Tohoku University Hospital
RECRUITINGSendai, Miyagi, 980-8574, Japan
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Universitair Medisch Centrum Utrecht - PPDS
NOT_YET_RECRUITINGUtrecht, 3584 CX, Netherlands
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University Hospital Birmingham NHS Foundation Trust
RECRUITINGBirmingham, West Midlands, B15 2TH, United Kingdom
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University of Michigan Hospitals
RECRUITINGAnn Arbor, Michigan, 48109, United States
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University of Stanford Medical Center
RECRUITINGPalo Alto, California, 94304-1503, United States
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Willink Biochemical Genetics Unit - Manchester
RECRUITINGManchester, England, M13 9WL, United Kingdom
Conditions
Explore the condition pages connected to this study.