A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
Stopped early · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Methylmalonic Acidemia
In brief
This is a study of mRNA-3705 in participants with isolated elevated methylmalonic acid (MMA) due to methylmalonyl-coenzyme A (CoA) mutase (MUT) deficiency. The main goal of the study is to assess safety, efficacy, pharmacokinetics, and pharmacodynamics of intravenously (IV)-infused mRNA-3705.
Key facts
- Study ID
- NCT04899310
- Run by
- ModernaTX, Inc.
- People needed
- 18
- Starts
- 2021-08-06
- Expected to finish
- 2026-04-01
- Last updated by the study team
- 2026-05-06
Who can join
Age: 1 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- UCLA Medical Center — Los Angeles, California, United States
- Lucile Packard Children's Hospital at Stanford — Palo Alto, California, United States
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- Stollery Children's Hospital University of Alberta — Edmonton, Alberta, Canada
- Hospital For Sick Children — Toronto, Ontario, Canada
- Hôpital Necker - Enfants Malades — Paris, France
- Erasmus MC — Rotterdam, Netherlands
- Universitair Medisch Centrum Utrecht — Utrecht, Netherlands
- Hospital Universitario Cruces — Barakaldo, Spain
- Hospital Universitario 12 de Octubre — Madrid, Spain
- Royal Manchester Children's Hospital — Manchester, United Kingdom
Full record on ClinicalTrials.gov
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