MRNA therapy trial for rare acidemia halted early
NCT ID NCT04899310
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested an mRNA therapy called mRNA-3705 in 18 people with a rare genetic condition called methylmalonic acidemia, which causes harmful acid buildup. The therapy aimed to help the body produce a missing enzyme to lower acid levels. The trial was terminated early, so final conclusions about safety and effectiveness are limited.
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 the body produce a missing enzyme)
- What this could lead to
- If successful, this could lead to a treatment that reduces harmful MMA levels in people with methylmalonic acidemia, potentially easing disease burden.
- What could go wrong
- The trial was terminated early with only 18 participants, so results are limited. As an early-phase study, safety and effectiveness are not yet proven.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for ISOLATED METHYLMALONIC ACIDEMIA DUE TO METHYLMALONYL-COA MUTASE DEFICIENCY are added.
By submitting, you agree to our Terms of use
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.
Contacts and locations
Locations
-
Erasmus MC
Rotterdam, 3015 AA, Netherlands
-
Hospital For Sick Children
Toronto, Ontario, M5G 1X8, Canada
-
Hospital Universitario 12 de Octubre
Madrid, 28041, Spain
-
Hospital Universitario Cruces
Barakaldo, 48903, Spain
-
Hôpital Necker - Enfants Malades
Paris, 75015, France
-
Lucile Packard Children's Hospital at Stanford
Palo Alto, California, 94304, United States
-
Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
-
Stollery Children's Hospital University of Alberta
Edmonton, Alberta, T6G 2R7, Canada
-
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
-
UCLA Medical Center
Los Angeles, California, 90095, United States
-
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
More trials for these conditions
Other studies related to the condition(s) this trial covers.