Phase 2 study of ARCT-810 in Participants with OTC Deficiency
Research type
Research Study
Full 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
IRAS ID
296910
Contact name
Robin Lachmann
Contact email
Sponsor organisation
Arcturus Therapeutics, Inc.
Eudract number
2021-001081-38
Duration of Study in the UK
1 years, 6 months, 0 days
Research summary
This trial intends to study a novel investigational medicinal product for the treatment of a rare genetic medical disease: ornithine transcarbamylase deficiency. Patients with this disease are missing an important enzyme called OTC in a body system named the urea cycle, leading to an accumulation of molecules to levels which are toxic for the human body. The current treatments available do not show an adequate reduction of the morbidity and mortality related with the disease, and more than 60% of surviving children treated with the commonly available treatments have disabling neurological complications.
The drug under study in the present trial, ARCT-810, aims to treat this disease by providing the liver cells with a molecule of messenger RNA encoding the deficient OTC enzyme, which upon entering the cells will lead to the production of the corrected protein. In this way the defective or missing enzyme will be replaced by a functional one, thereby reinstating the body’s normal functions.
The main purpose of the trial is to evaluate the safety and tolerability of the study drug in adult and adolescent (≥12 years) participants with ornithine transcarbamylase deficiency. The trial is randomized, which means patients are assigned to the treatment by chance (i.e., like the flip of a coin) and placebo controlled, which means not all participants will receive the study drug. Two treatment groups will be studied, which differ in the dose of study drug administered, and participants will have a 3:1 chance of receiving the study drug vs. placebo within each of these groups. Upon confirmation of the requirements to participate, the participant will receive a first administration of the study drug. After verification of the safety of this administration, the participant will progress to receive five additional doses at 14-day intervals. The overall study duration for each participant will be approximately 28 weeks.REC name
London - West London & GTAC Research Ethics Committee
REC reference
21/LO/0372
Date of REC Opinion
14 Jul 2021
REC opinion
Further Information Favourable Opinion