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

Part of paid clinical trials in Chevy Chase, Maryland.

Sponsor
Arcturus Therapeutics, Inc.
Study ID
NCT06488313
Phase
PHASE2
Status
Recruiting
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Conditions

  • OTC Deficiency
  • OTCD
  • Ornithine Transcarbamylase Deficiency

Eligibility Criteria

Sex
ALL
Age
12 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • ARCT-810 — BIOLOGICAL
    ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) messenger RNA (mRNA) formulated in a lipid nanoparticle (LNP).

Study Details

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

Key Dates

Start date
Nov 4, 2024
Status verified
Mar 2025
Primary completion
Jun 1, 2026
Completion
Sep 1, 2026

Study Design

Enrollment
9 participants (estimated)
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: ARCT-810
    Participants will receive up to 5 IV infusions of ARCT-810 administered at 14-day intervals.

Primary Outcome Measure

Incidence, severity and dose-relationship of adverse events (AEs) [ Time Frame: Day 85 ]

Locations (1)

FacilityCityStateZIPSite coordinators
Uncommon CuresChevy ChaseMaryland20815
Marshall Summar, MD
[email protected]
541-232-7818

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By research site
Uncommon Cures· Chevy Chase, MD

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