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

Recruiting Phase 2 Interventional Study
OTC Deficiency Ornithine Transcarbamylase Deficiency OTCD
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
12 and older
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
9 (estimated)
Sponsor
Arcturus Therapeutics, Inc. · Industry
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About This Trial

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

Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent. 2. Males and Females aged ≥12 years, at Screening. 3. Documented clinical diagnosis of OTC deficiency. 4. History of symptomatic hyperammonemia or elevat…
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