An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705
Condition(s) studied
Investigational drug(s) / intervention(s)
mRNA-3705: A sterile liquid for injection
Study summary
The primary objective of this study is to evaluate the long-term safety and clinical activity of mRNA-3705 administered to participants with isolated methylmalonic acidemia (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency who have previously participated in other clinical studies of mRNA-3705.
Eligibility
Primary outcome measure(s)
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Drug-Related TEAEs, Unrelated TEAEs, Adverse Events of Special Interests (AESIs), Serious Adverse Events (SAEs) and TEAEs Leading to Discontinuation — Baseline up to follow-up period (up to 6 years + 6 months)
Trial sites (12)
| Facility | City | Region | Status |
|---|---|---|---|
| UCLA Medical Center | Los Angeles | California | Recruiting |
| Lucile Packard Children's Hospital at Stanford | Palo Alto | California | Recruiting |
| Altman Clinical and Translational Research Institution | San Diego | California | Recruiting |
| Stollery Children's Hospital University of Alberta | Edmonton | Alberta | Recruiting |
| Hospital For Sick Children | Toronto | Ontario | Recruiting |
| Hôpital Necker - Enfants Malades APHP | Paris | France | Recruiting |
| Erasmus MC | Rotterdam | Netherlands | Recruiting |
| Universitair Medisch Centrum Utrecht | Utrecht | Netherlands | Recruiting |
| Hospital Universitario Cruces | Barakaldo | Vizcaya | Recruiting |
| Hospital Universitario 12 de Octubre | Madrid | Spain | Recruiting |
| Birmingham Children's Hospital NHS Foundation Trust | Birmingham | United Kingdom | Terminated |
| Royal Manchester Childrens Hospital | Manchester | United Kingdom | Recruiting |
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Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05295433 on ClinicalTrials.gov ↗ ← All trials in France