Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

Completed · Phase 1 · Has a placebo group

Conditions studied: Ornithine Transcarbamylase Deficiency

In brief

Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in clinically stable patients (stable on standard of care treatment, e.g. diet ± ammonia scavengers) with ornithine transcarbamylase deficiency (OTCD).

Key facts

Study ID

NCT04442347

Run by

Arcturus Therapeutics, Inc.

People needed

16

Starts

2020-11-03

Expected to finish

2024-04-30

Last updated by the study team

2024-08-02

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Adequate cognitive ability to consent and recall symptoms over a 1-week time period
• Males and females ≥18 years of age with documented diagnosis of ornithine transcarbamylase deficiency (OTCD) confirmed with genetic testing, or willing to consent to OTC gene sequencing and deletion/duplication testing
• Subject's ornithine transcarbamylase deficiency (OTCD) is stable as evidenced by
• no clinical symptoms of hyperammonemia AND
• an ammonia level <100 µmol/L (170 µg/dL) at the screening evaluation
• Subjects must remain free from symptoms of hyperammonemia throughout the screening period.
• If using nitrogen ammonia scavenger therapy, must be on a stable regimen (no change in dose or frequency) for ≥ 28 days prior to providing informed consent and throughout the screening period
• Must have maintained a stable protein-restricted diet (+/- amino acid supplementation) for at least 28 days prior to providing informed consent and continue to maintain a stable diet for the duration of the study
• Good general health other than OTCD, in the opinion of the Investigator
• Willing to refrain from strenuous exercise/activity and alcohol for 72 hours before study visits
• Willingness to comply with procedures and visits
• Willingness to follow contraception guidelines

You may not qualify if…

• History of clinically significant disease(s), in the opinion of the Investigator
• Clinically significant screening laboratory values
• Uncontrolled diabetes
• Clinically significant anemia
• Subjects who develop infection during screening must be asymptomatic for at least 7 days prior to dosing
• Unwillingness to comply with study requirements
• History of positive HIV, hepatitis C, or chronic hepatitis B
• Uncontrolled hypertension
• Malignancy within 5 years prior to study
• Treatment with another investigational drug, biological agent, or device within 30 days of screening, or 5 half-lives of investigational drug
• Treatment with any oligonucleotide or mRNA within 6 months of screening, with exceptions for some vaccinations and investigational treatments
• History of gene therapy, hepatocyte or mesenchymal stem cell transplantation
• Prior organ transplant
• History of severe allergic reaction to a liposomal product
• Recent history of, or current, drug or alcohol abuse
• Dependence on inhaled (smoked or vaped) or oral cannabis products
• Systemic corticosteroids within 6 weeks prior to screening
• Blood donation of 50 to 499 mL within 30 days of screening or of .499 mL within 60 days of screening
• Other conditions, in the opinion of the Investigator, that would make the subject unsuitable for participation

Where it is running

• University of Florida — Gainesville, Florida, United States
• M Health Fairview Masonic Children's Hospital — Minneapolis, Minnesota, United States
• The Mount Sinai Hospital — New York, New York, United States
• Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• University of Texas Southwestern Medical Center at Dallas — Dallas, Texas, United States
• Baylor University — Waco, Texas, United States
• University of Utah — Salt Lake City, Utah, United States
• Children's Wisconsin - Milwaukee Hospital — Milwaukee, Wisconsin, United States

Full record on ClinicalTrials.gov

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