New mRNA therapy aims to control rare metabolic disease Long-Term

NCT ID NCT05295433

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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.

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Conditions

The condition(s) this trial relates to.

hereditary disease inborn errors of metabolism methylmalonic acidemia methylmalonic aciduria due to methylmalonyl-CoA mutase deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

METHYLMALONIC ACIDEMIA

Contacts and locations

Locations

Altman Clinical and Translational Research Institution
RECRUITING

San Diego, California, 92037, United States
Birmingham Children's Hospital NHS Foundation Trust
TERMINATED

Birmingham, B4 6NH, United Kingdom
Erasmus MC
RECRUITING

Rotterdam, 3015 AA, Netherlands
Hospital For Sick Children
RECRUITING

Toronto, Ontario, M5G 1X8, Canada
Hospital Universitario 12 de Octubre
RECRUITING

Madrid, 28041, Spain
Hospital Universitario Cruces
RECRUITING

Barakaldo, Vizcaya, 48903, Spain
Hôpital Necker - Enfants Malades APHP
RECRUITING

Paris, 75015, France
Lucile Packard Children's Hospital at Stanford
RECRUITING

Palo Alto, California, 94304, United States
Royal Manchester Childrens Hospital
RECRUITING

Manchester, M13 9WL, United Kingdom
Stollery Children's Hospital University of Alberta
RECRUITING

Edmonton, Alberta, T6G 2R7, Canada
UCLA Medical Center
RECRUITING

Los Angeles, California, 90095, United States
Universitair Medisch Centrum Utrecht
RECRUITING

Utrecht, 3584 CX, Netherlands

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