Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency
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|ClinicalTrials.gov Identifier: NCT04442347|
Recruitment Status : Recruiting
First Posted : June 22, 2020
Last Update Posted : October 8, 2020
|Condition or disease||Intervention/treatment||Phase|
|Ornithine Transcarbamylase Deficiency||Biological: ARCT-810 Other: Placebo||Phase 1|
This is a single ascending dose study of ARCT-810 in which approximately 12 (up to a maximum of 20) subjects are planned to be enrolled. The length of each study participant is approximately 8 weeks from screening to last study visit. The study comprises an up to 4-week Screening and a 4-week Diet Run-in period to run concurrently followed by a 1-day dosing period and a 28-day Post Treatment Period.
Study participants will be allocated to one of the three different study groups (also called cohorts), to test different doses of ARCT-810. There will be 4 participants in each group. Within each cohort, subjects will be randomized 3 :1 to receive ARCT-810 or placebo as an IV infusion.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||12 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Masking Description:||Double Blinded, Placebo Controlled|
|Official Title:||A Phase 1b Randomized, Double Blinded, Placebo Controlled, Ascending Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Doses of ARCT-810 in Clinically Stable Patients With Ornithine Transcarbamylase Deficiency|
|Estimated Study Start Date :||October 2020|
|Estimated Primary Completion Date :||November 2021|
|Estimated Study Completion Date :||December 2021|
Ascending single doses of ARCT-810 administered intravenously
ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) mRNA formulated in a lipid nanoparticle (LNP) under development.
Placebo Comparator: Placebo
Single doses of 0.9% Saline administered intravenously
The placebo for this study is 0.9% sterile saline.
- Incidence, severity and dose-relationship of adverse events (AEs) [ Time Frame: 4 weeks ]Safety and tolerability of ARCT-810 assessed by determining the incidence, severity and dose-relationship of AEs by dose
- Change in area under the curve after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]Area under the plasma concentration versus time curve (AUC) from time zero to the last quantifiable time point
- Maximum observed plasma concentration (Cmax) after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]The maximum observed plasma concentration (Cmax)
- Time at which Cmax occurred after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]The time at which Cmax occurred (Tmax)
- AUC0-inf after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]AUC from time zero extrapolated to infinity
- AUCExtrap after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]The relative portion of AUC0-inf extrapolated beyond AUC0-t
- T1/2 after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]Terminal half-life
- MRT0-inf after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]The mean residence time extrapolated to infinity
- CL after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]Total body clearance, calculated as dose divided by AUC0-inf
- Vss after single dose of ARCT-810 [ Time Frame: Up to 4 weeks ]Volume of distribution
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04442347
|Contact: Steve Hughes, MDemail@example.com|
|Contact: Manely Yafeh||858-900-2660||Manely@arcturusrx.com|
|United States, Texas|
|University of Texas Southwestern Medical Center at Dallas||Recruiting|
|Dallas, Texas, United States, 75390|