Ignite Creation Date:
2024-05-06 @ 8:06 PM
Last Modification Date:
2024-10-26 @ 3:20 PM
Study NCT ID:
NCT06255782
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-06-28
First Post:
2023-12-19
Brief Title:
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency OTC-HOPE
Sponsor:
iECURE Inc
Organization:
iECURE Inc
Study Overview
Official Title:
A Phase III 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:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Ornithine Transcarbamylase OTC deficiency the most common urea cycle disorder is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia Individuals with OTC deficiency can build-up excess levels of ammonia in their blood potentially resulting in devastating consequences including cumulative and irreversible neurological damage coma and death The severe form of the condition emerges shortly after birth and is more common in boys than girls
This is a Phase 12 open-label multicenter safety and dose finding study of ECUR-506 in male babies with neonatal onset OTC deficiency The primary objective of this study is to evaluate the safety and tolerability of multiple dose levels of ECUR-506 following intravenous IV administration of a single dose
This is a Phase 12 open-label multicenter safety and dose finding study of ECUR-506 in male babies with neonatal onset OTC deficiency The primary objective of this study is to evaluate the safety and tolerability of multiple dose levels of ECUR-506 following intravenous IV administration of a single dose
Detailed Description:
The study drug ECUR-506 is an investigational gene editing therapy Gene editing is a way to repair replace or introduce new copies of genes that dont work The study drug contains a working copy of the OTC gene that will be delivered by an IV infusion It also contains a gene to encode the editing enzyme which is the part of the study drug that can cut DNA so that the OTC gene can be inserted The study drug was designed to introduce a working copy of the OTC gene and a gene to encode the editing enzyme A gene cannot enter cells by itself it needs a delivery mechanism to move the gene into the cells In this study a commonly used virus called adeno-associated virus AAV is used to enter the cells and deliver the genes
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| OTC HOPE | OTHER | iECURE Inc | None |