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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

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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 Aug 2024

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At a glance

  • Drugs MRNA-3705 (Primary)
  • Indications Methylmalonic acidaemia
  • Focus Adverse reactions; First in man
  • Acronyms Landmark study
  • Sponsors Moderna Therapeutics

    • Most Recent Events

    • 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.
    • 02 Aug 2021 Planned initiation date (estimated date for recruitment of the first subject) changed from 28 Jun 2021 to 6 Aug 2021.

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