Trial Profile

A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 22 Sep 2025

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At a glance

02 Sep 2025 According to a Moderna media release, interim result data from this study have been accepted for presentation at the 2025 International Congress of Inborn Errors of Metabolism (ICIEM), which will be held September 2-6, 2025 in Kyoto, Japan, at the Kyoto International Conference Center.
31 Oct 2023 Planned number of patients changed from 33 to 63.
16 Aug 2021 According to a Moderna media release, the company announced the first patient has been dosed in this trial.

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