Stopped: As part of ongoing efforts to refine the MMA program strategy, we are reevaluating clinical study plans for mRNA-3705, in order to further optimize the development program. As part of this process, Moderna will be closing the mRNA-3705-P101 study.
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.
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Parts 1 and 3: Number of Participants with Treatment-Emergent Adverse Events (TEAEs), Study Drug-related TEAEs, Adverse Events of Special Interest (AESIs), Serious Adverse Events (SAEs), and TEAEs Leading to Treatment Discontinuation
Timeframe: Up to 134 weeks
Part 2: Percentage Change in Plasma MMA Levels at Month 3
Timeframe: Baseline, Month 3