Trials Sponsors Conditions Drugs Pricing

Conditions

Ornithine Transcarbamylase Deficiency OTC Deficiency OTCD

Outcome Measures

Primary Outcomes (1)

Incidence, severity and dose-relationship of adverse events (AEs)
Safety and tolerability of ARCT-810 assessed by incidence, severity, and dose-relationship of AEs
Day 85

Secondary Outcomes (5)

Stable isotope ureagenesis assay values (AUC of first isotope)
Up to Day 85
Stable isotope ureagenesis assay values (AUC of second isotope)
Up to Day 85
Fasting plasma ammonia
Up to Day 85
Plasma Glutamine
Up to Day 85
Plasma pharmacokinetics
Up to Day 57

Study Arms (1)

ARCT-810

EXPERIMENTAL

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

Biological: ARCT-810

Interventions

ARCT-810Biological

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

ARCT-810

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age12 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Willingness and ability to comply with all the protocol requirements, complete all study visits and sign informed consent.
Males and Females aged ≥12 years, at Screening.

Sponsors & Collaborators

Arcturus Therapeutics, Inc.
Industrylead

Study Sites (1)

Uncommon Cures

Chevy Chase, Maryland, 20815, United States

RECRUITING

Marshall Summar, MDCONTACT541-232-7818 arcturus@uncommoncures.com

MeSH Terms

Conditions

Ornithine Carbamoyltransferase Deficiency Disease

Condition Hierarchy (Ancestors)

Urea Cycle Disorders, InbornBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type: interventional
Phase: phase 2
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SEQUENTIAL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

June 28, 2024

First Posted

July 5, 2024

Study Start

November 4, 2024

Actual (reported)

Primary Completion

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (5)

Study Arms (1)

ARCT-810

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (5)

Study Arms (1)

ARCT-810

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations