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A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency

Trial Profile

A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 06 Nov 2019

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At a glance

  • Drugs MRNA-3704 (Primary)
  • Indications Methylmalonic acidaemia
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Sponsors Moderna Therapeutics
  • Most Recent Events

    • 06 Nov 2019 According to a Moderna Therapeutics media release, the company recently received the Medicines and Healthcare products Regulatory Agency (MHRA) approval in the U.K for this study.
    • 06 Nov 2019 According to a Moderna Therapeutics media release, the company is actively recruiting patients for the first cohort at U.S. sites following a protocol amendment expanding the eligibility criteria to patients 8 years and older.
    • 05 Jun 2019 Status changed from not yet recruiting to recruiting.
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