Long-term Follow-up Study of Participants in any iECURE Protocol using an Investigational Product
Research type
Research Study
Full title
Long-term Follow-up (LTFU) Study of Participants in any iECURE Protocol Using an Investigational Product (IP)
IRAS ID
1010080
Contact name
Maryann Toto
Contact email
Sponsor organisation
iECURE
Clinicaltrials.gov Identifier
Research summary
Children who have been participating in a clinical research study for a new gene editing drug, ECUR-506, for the treatment of neonatal onset Ornithine Transcarbamylase (OTC) deficiency, will be invited to voluntarily participate in a long-term follow-up (LTFU) clinical research study.
The purpose of this study is to look at the long-term effects of ECUR-506 that the child may have received during the previous study.
Often, gene editing therapy products, like ECUR-506 are designed to achieve therapeutic effect through permanent or long-acting changes in the body. Therefore, LTFU studies are important to monitor their long-term safety. Even if the child did not receive ECUR-506, information related to their condition will still be collected to help researchers better understand OTC deficiency. The child will not receive any study drug in this study.
Study procedures include tests to record electric signals in the heart (electrocardiogram), physical exams, tests to look at brain and nervous system and an imaging test that uses sound waves to see inside the belly (abdominal ultrasound). Samples of blood and urine will be taken for testing. If the child received study drug in the previous study but did not undergo a procedure to remove a small piece of iver tissue (a liver biopsy), this procedure will be done prior to 1 year of age for the LTFU study.
All patients (up to 12) from the previous study will be invited to participate in this LTFU study. This study is being conducted at several hospitals globally. The child’s participation in this study will last up to 15 years. Parents/carers will come to the study centre with their child for up to 27 visits. Monthly visits are planned for the first year, then every 3 months through year 2, every 6 months through year 5, then annually after year 5.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
24/SC/0174
Date of REC Opinion
5 Jul 2024
REC opinion
Further Information Favourable Opinion