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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: 23 Jan 2026

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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

  • 12 Jan 2026 According to an iECURE media release, iECURE reached alignment with the U.S Food and Drug Administration (FDA) on the primary and key secondary efficacy endpoints, comparators and study size for the ongoing OTC-HOPE study which could support a Biologics License Application. iECURE expects to release data from the ongoing clinical trial in the first half of 2026.
  • 07 Jan 2026 According to an iECURE media release, encouraging clinical data from the ongoing OTC-HOPE clinical study, including a complete clinical response observed in the first infant treated with ECUR-506, supported the FDA's grant of RMAT designation; ECUR-506 has also previously received Rare Pediatric Disease and Orphan Drug designations for the treatment of neonatal-onset OTC deficiency.
  • 02 Oct 2025 According to an iECURE media release, company to present updated result data from this study at the American Society of Human Genetics (ASHG) Annual Meeting, taking place October 14-18 in Boston.

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