Ignite Creation Date:
2025-12-24 @ 4:23 PM
Ignite Modification Date:
2026-01-04 @ 1:08 AM
Study NCT ID:
NCT05526066
Status:
TERMINATED
Last Update Posted:
2025-09-26
First Post:
2022-08-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810
Sponsor:
Arcturus Therapeutics, Inc.
Organization:
Study Overview
Official Title:
Phase 2, Randomized, Double-Blind, Placebo-Controlled, Nested Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ARCT-810 in Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency
Status:
TERMINATED
Status Verified Date:
2025-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
The study was terminated after the completion of Cohort 1 (0.3 mg/kg) due to slow enrollment in this placebo-controlled study.
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The primary objective is to evaluate the safety and tolerability of repeated doses of intravenously administered ARCT-810.
Detailed Description:
This study is a Phase 2, randomized, placebo-controlled study of ARCT-810 in people living with OTC deficiency 12 years of age and older. After an at least 4 week screening and diet stabilization period, participants will be randomized 3:1 to receive ARCT-810 or placebo. Following the first dose and safety evaluation, participants will receive up to an additional 5 doses of ARCT-810 or placebo, each separated by 14 days. The treatment period is followed by a 12-week observation period.
Study Oversight
Has Oversight DMC:
True
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?: