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

NCT04442347 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2024-08-02

No results posted yet for this study

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).

Conditions

  • Ornithine Transcarbamylase Deficiency

Interventions

BIOLOGICAL

ARCT-810

ARCT-810 is an investigational medicinal product comprising Ornithine Transcarbamylase (OTC) mRNA formulated in a lipid nanoparticle (LNP) under development.

OTHER

Placebo

The placebo for this study is 0.9% sterile saline.

Sponsors & Collaborators

  • Arcturus Therapeutics, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-11-03
Primary Completion
2023-08-30
Completion
2024-04-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04442347 on ClinicalTrials.gov