ECUR-506 in Neonatal OTC Deficiency Phase I/II

• Research type

  Research Study

• Full title

  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

• IRAS ID

  1008261

• Contact name

  Maryann Toto

• Contact email

  mtoto@iecure.com

• Sponsor organisation

  iECURE

• ISRCTN Number

  NA

• Research summary

  The purpose of this study is to look at how safe and how well one of two dose levels of a new investigational gene editing drug, ECUR-506, works for the treatment of neonatal onset (within the first four weeks of an infant's life) Ornithine Transcarbamylase Deficiency (OTC Deficiency). Gene editing means that a corrective gene which can produce a working OTC enzyme is introduced into DNA.
  Neonatal Onset OTC Deficiency is a rare genetic condition that causes a damaging substance, called ammonia, to build up in the blood because the enzyme, OTC, is missing from a child's body. It is more common in boys.
  Currently there is no approved medication that cures OTC Deficiency and a liver transplant is considered the only treatment that can cure the disease. Therefore, there is an urgent need for research into new treatment options for OTC Deficiency.

  Approximately 9 boys, up to 9 months old, who have been diagnosed with OTC Deficiency will be dosed with one of two dose levels in this study. The study is being conducted at several hospitals globally.
  There are 28 study visits over a period up to 42 weeks. Study drug will be given as an intravenous infusion (via a tube linked to a small needle in the child’s vein) over a period of approximately 1 to 2 hours. Study procedures include measurements such as blood pressure and weight, electrocardiogram, liver biopsy, ultrasound, blood test, tests on urine, faeces and questionnaires for parents/carers.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  23/LO/0846

• Date of REC Opinion

  8 Dec 2023

• REC opinion

  Further Information Favourable Opinion