Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult S… (NCT04442347) | Clinical Trial Compass
CompletedPhase 1
Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency
United States16 participantsStarted 2020-11-03
Plain-language summary
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).
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. 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
✓. 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
Exclusion criteria
✕. History of clinically significant disease(s), in the opinion of the Investigator