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A Phase I/II First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of a Single Intravenous (IV) Administration of ECUR-506 in Males Less Than 9 Months of Age With Genetically Confirmed Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency

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Trial Profile

A Phase I/II First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of a Single Intravenous (IV) Administration of ECUR-506 in Males Less Than 9 Months of Age With Genetically Confirmed Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 20 Jun 2025

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At a glance

  • Drugs ECUR 506 (Primary)
  • Indications Ornithine carbamoyltransferase deficiency disease
  • Focus Adverse reactions; First in man
  • Acronyms OTC-HOPE
  • Sponsors iECURE

Most Recent Events

  • 07 May 2025 According to an iECURE media release, initial data from the first participant to complete the ongoing trial will be presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place May 12 - 17, 2025 in New Orleans and the European Society of Human Genetics (ESHG) Conference taking place May 24 - 27, 2025 in Milan, Italy.
  • 25 Mar 2025 According to an iECURE media release, data from this study were presented at the 2025 ACMG Annual Clinical Genetics Meeting from the ongoing OTC-HOPE Phase 1/2 study of ECUR-506 suggest partial restoration of functional ornithine transcarbamylase (OTC) enzyme activity in the first patient dosed.
  • 25 Mar 2025 Results presented in an iECURE Media Release.

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