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mRNA-3705 dose optimization study in isolated methylmalonic acidemia due to MUT deficiency
NL-OMON55911Phase 2Not yet recruiting
- Sponsor
- Moderna Inc
- Start
- Jan 2023
- Modality
- Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Countries
- 1
01
Overview
Phase 2, open-label trial of mRNA-3705 in Isolated Methylmalonic Acidemia (MMA) and Methylmalonic acidemia mutase type (MMA) (2 arms), sponsored by Moderna Inc. Primary endpoints: Incidence and severity of TEAEs, including study drug-related and unrelated TEAEs, SAEs, and TEAEs leading to treatment discontinuation and Annualized frequency of metabolic decompensation events after 12 months of treatment. Planned start Jan 2023; status: not yet recruiting.
02
Drugs
mRNA-3705
03
Conditions
Isolated Methylmalonic Acidemia (MMA)Methylmalonic acidemia mutase type (MMA)
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