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Outcome Measures

Primary Outcomes (1)

Incidence, severity and dose-relationship of adverse events (AEs)
Safety and tolerability of ARCT-810 assessed by determining the number and severity of AEs by dose level
Week 23

Secondary Outcomes (11)

Plasma concentration area under the curve after first and last doses of ARCT-810
Up to 17 Weeks
Maximum observed plasma concentration (Cmax) after first and last doses of ARCT-810
Up to 17 Weeks

Study Arms (2)

ARCT-810

EXPERIMENTAL

Participants receive an initial intravenous (IV) infusion ARCT-810. If considered safe and well tolerated, participants will receive up to 5 additional IV infusions of ARCT-810 administered at 14-day intervals.

Biological: ARCT-810

Placebo, Normal Saline

PLACEBO COMPARATOR

Participants receive an initial IV infusion of placebo. If considered safe and well tolerated, participants receive up to 5 additional IV infusions of placebo administered at 14-day intervals.

Other: Placebo

Interventions

ARCT-810Biological

ARCT-810 is messenger RNA (mRNA) coding for Ornithine Transcarbamylase (OTC) formulated in a lipid nanoparticle (LNP).

ARCT-810

PlaceboOther

Normal Saline

Placebo, Normal Saline

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

Study Sites (14)

Cliniques Universitaires Saint Luc

Brussels, 1200, Belgium

Hôpitaux Universitaires de Marseille - Hôpital de la Timone

Marseille, 13005, France

Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades

Paris, France

Azienda Ospedaliera di Padova

Padova, 35128, Italy

IRCCS Ospedale Pediatrico Bambino Gesu

Rome, 00165, Italy

Hospital Clínic de Barcelona

Barcelona, Spain

Hospital Sant Joan de Déu

Barcelona, Spain

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

RANDOMIZED

Masking

QUADRUPLE

Who Masked

PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR

Masking Details

Double-Blinded

Purpose

TREATMENT

Intervention Model

SEQUENTIAL

Model Details: On Day 1, participants will receive a single dose of Study Drug via intravenous infusion. If the safety observations are considered acceptable, that participant will enter the multiple-dose portion of the study in which they will receive a further 5 doses of Study Drug on Days 15, 29, 43, 57 and 71.

Sponsor Type

INDUSTRY

Responsible Party

SPONSOR

Study Stopped

Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (11)

Study Arms (2)

ARCT-810

Placebo, Normal Saline

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (14)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (11)

Study Arms (2)

ARCT-810

Placebo, Normal Saline

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (14)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations