A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD

NCT06488313 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: ARCT-810-04
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.

Conditions

OTC Deficiency; Ornithine Transcarbamylase Deficiency; OTCD

Outcome Measures

Primary Outcomes (1)

• Incidence, severity and dose-relationship of adverse events (AEs)

  Safety and tolerability of ARCT-810 assessed by incidence, severity, and dose-relationship of AEs

  Day 85

Secondary Outcomes (5)

• Stable isotope ureagenesis assay values (AUC of first isotope)

  Up to Day 85

• Stable isotope ureagenesis assay values (AUC of second isotope)

  Up to Day 85

• Fasting plasma ammonia

  Up to Day 85

• Plasma Glutamine

  Up to Day 85

• Plasma pharmacokinetics

  Up to Day 57

Study Arms (1)

ARCT-810

EXPERIMENTAL

Participants will receive up to 5 IV infusions of ARCT-810 administered at 14-day intervals.

Biological: ARCT-810

Interventions

ARCT-810 BIOLOGICAL

ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) messenger RNA (mRNA) formulated in a lipid nanoparticle (LNP).

ARCT-810

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent.
• Males and Females aged ≥12 years, at Screening.
• Documented clinical diagnosis of OTC deficiency.
• History of symptomatic hyperammonemia or elevated plasma ammonia or glutamine with clinical stability for at least 1 month prior to Screening.
• Medically managed for OTC deficiency and receiving a stable protein-restricted diet, dietary supplements, and/or ammonia scavenger regimen (if applicable) for at least 28 days.
• Good general health with no clinically significant abnormal findings that would interfere with study procedures (including plasma ammonia within participant's historical range).
• Must be willing to adhere to contraception guidelines.

You may not qualify if:

• Uncontrolled hypertension.
• Symptoms of infection for at least 7 days prior to dosing.
• Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
• History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 2 years.
• History of any organ transplant.
• History of severe allergic reaction to a liposomal or PEG-containing product.
• History of congenital or acquired cardiac disorders.
• Abuse of medications, illicit drugs or alcohol.
• Blood donation of 50 to 499 mL within 30 days of screening or of \>499 mL within 60 days of screening.
• Clinically significant laboratory abnormalities on screening labs including INR \>1.5, eGFR\< 60 mL/min/1.73m2 or positive test results for HIV, HBV, or HCV.
• Inadequately controlled diabetes.
• Clinically significant anemia.
• Changes in maintenance therapies for OTC deficiency with 28 days prior to dosing.
• Medical history requiring continuous or intermittent systemic corticosteroid administration.
• Receipt of inhibitors of urea synthesis or drugs that significantly affect renal clearance.

Sponsors & Collaborators

• Arcturus Therapeutics, Inc.: lead

Study Sites (1)

Uncommon Cures

Chevy Chase, Maryland, 20815, United States

RECRUITING

MeSH Terms

Conditions

Ornithine Carbamoyltransferase Deficiency Disease

Condition Hierarchy (Ancestors)

Urea Cycle Disorders, Inborn; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: All participants will receive 5 doses of Study Drug via intravenous infusion at one of the three dose levels. Dose levels will be enrolled sequentially.

Study Record Dates

• First Submitted: June 28, 2024
• First Posted: July 5, 2024
• Study Start: November 4, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: March 17, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)