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TERMINATEDPHASE2

Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

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

NCT05526066•Arcturus Therapeutics, Inc.

View on ClinicalTrials.gov

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.

Eligibility

Age

12 - 65 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Adequate cognitive ability to understand study requirements and give informed consent
•2. Males and females aged 12 to 65 years inclusive, at Screening
•3. Documented diagnosis of OTC deficiency
•4. Clinical stability (no clinical symptoms of hyperammonemia within 1 month, no hospitalizations for metabolic decompensation within 3 months, ≤ 2 hospitalizations within 1 year)
•5. Stable protein-restricted diet, dietary supplements, and ammonia scavenger regimen (if applicable) for at least 28 days.
•6. BMI = 18.0 - 32.0 kg/m2, inclusive for adults, and \>5th percentile for adolescents ≥12 to 17 years
•7. Must be willing to adhere to contraception guidelines

Exclusion Criteria

•1. History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 3 years
•2. History of other medical conditions that may make the participant unsuitable for inclusion or could interfere with study participation (e.g., uncontrolled hypertension or diabetes, malignancy, HIV, hepatitis B or C)
•3. History of severe allergic reaction to liposomal or PEG-containing products
•4. Abuse of illicit drugs, medications or alcohol
•5. Clinically significant laboratory abnormalities on screening labs

Locations (14)

Cliniques Universitaires Saint Luc

Brussels, Belgium

Hôpitaux Universitaires de Marseille - Hôpital de la Timone

Marseille, France

Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades

Paris, France

Azienda Ospedaliera di Padova

Padua, Italy

IRCCS Ospedale Pediatrico Bambino Gesu

Rome, Italy

Hospital Clínic de Barcelona

Barcelona, Spain

Hospital Sant Joan de Déu

Barcelona, Spain

Hospital Universitario 12 de Octubre

Madrid, Spain

Complejo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario

Santiago de Compostela, Spain

Karolinska Universitetssjukhuset - Astrid Lindgrens Barnsjukhus

Stockholm, Sweden

Key Dates

Start Date

October 17, 2022

Primary Completion Date

October 31, 2024

Completion Date

October 31, 2024

Last Updated

September 26, 2025

Enrollment

ACTUAL participants

Conditions

Ornithine Transcarbamylase DeficiencyOTC DeficiencyOTCD

Interventions

ARCT-810

BIOLOGICAL

Placebo

OTHER

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 26, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

Inclusion Criteria

Exclusion Criteria

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

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