TerminatedPhase 2

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

Stopped: The study was terminated after the completion of Cohort 1 (0.3 mg/kg) due to slow enrollment in this placebo-controlled study.

Belgium, France, Italy8 participantsStarted 2022-10-17

Plain-language summary

The primary objective is to evaluate the safety and tolerability of repeated doses of intravenously administered ARCT-810.

Age range12 Years – 65 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

✕. History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 3 years
✕. 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)
✕. History of severe allergic reaction to liposomal or PEG-containing products
✕. Abuse of illicit drugs, medications or alcohol
✕. Clinically significant laboratory abnormalities on screening labs

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

Timeframe: Week 23

NCT IDNCT05526066

SponsorArcturus Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-10-31

Who can participate

Age range12 Years – 65 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

✕. History of any OTC gene therapy, or history of liver-derived stem cell therapy in the past 3 years
✕. 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)
✕. History of severe allergic reaction to liposomal or PEG-containing products
✕. Abuse of illicit drugs, medications or alcohol
✕. Clinically significant laboratory abnormalities on screening labs