New mRNA therapy aims to control rare metabolic disease Long-Term
NCT ID NCT05295433
First seen Nov 01, 2025 · Last updated Jun 23, 2026 · Updated 32 times
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.
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This is a summary of
the original study
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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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Altman Clinical and Translational Research Institution
RECRUITINGSan Diego, California, 92037, United States
Contact Email: •••••@•••••
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Birmingham Children's Hospital NHS Foundation Trust
TERMINATEDBirmingham, B4 6NH, United Kingdom
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Erasmus MC
RECRUITINGRotterdam, 3015 AA, Netherlands
Contact Email: •••••@•••••
Contact
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Hospital For Sick Children
RECRUITINGToronto, Ontario, M5G 1X8, Canada
Contact Email: •••••@•••••
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Hospital Universitario 12 de Octubre
RECRUITINGMadrid, 28041, Spain
Contact Email: •••••@•••••
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Hospital Universitario Cruces
RECRUITINGBarakaldo, Vizcaya, 48903, Spain
Contact Email: •••••@•••••
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Hôpital Necker - Enfants Malades APHP
RECRUITINGParis, 75015, France
Contact Email: •••••@•••••
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Lucile Packard Children's Hospital at Stanford
RECRUITINGPalo Alto, California, 94304, United States
Contact Email: •••••@•••••
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Royal Manchester Childrens Hospital
RECRUITINGManchester, M13 9WL, United Kingdom
Contact Email: •••••@•••••
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Stollery Children's Hospital University of Alberta
RECRUITINGEdmonton, Alberta, T6G 2R7, Canada
Contact Email: •••••@•••••
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UCLA Medical Center
RECRUITINGLos Angeles, California, 90095, United States
Contact Email: •••••@•••••
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Universitair Medisch Centrum Utrecht
RECRUITINGUtrecht, 3584 CX, Netherlands
Contact Email: •••••@•••••
Contact
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.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.