ARCT-810 / Arcturus Therap - LARVOL DELTA

Study for Adolescents and Adults with Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 (clinicaltrials.gov) - P2 | N=8 | Terminated | Sponsor: Arcturus Therapeutics, Inc. | N=24 ➔ 8 | Trial completion date: Mar 2025 ➔ Nov 2024 | Active, not recruiting ➔ Terminated; The study was terminated after the completion of Cohort 1 (0.3 mg/kg) due to slow enrollment in this placebo-controlled study.

Enrollment change • Trial completion date • Trial termination • Genetic Disorders

ARCT-810-04: A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD (clinicaltrials.gov) - P2 | N=9 | Recruiting | Sponsor: Arcturus Therapeutics, Inc. | Initiation date: Jun 2024 ➔ Nov 2024

Trial initiation date • Genetic Disorders

Arcturus Therapeutics Announces Initiation of Dosing in Phase 2 Multiple Ascending Dose Studies for Cystic Fibrosis (CF) and Ornithine Transcarbamylase (OTC) Deficiency (Businesswire) - "Arcturus Therapeutics Holdings...announced that the first CF and OTC deficiency participants initiated dosing in December 2024, in the Company’s Phase 2 multiple ascending dose studies. Each participant in the Phase 2 CF study (NCT06747858) is expected to receive daily treatments of ARCT-032 over a period of 28 days. The first OTC deficient participant receiving 0.5 mg/kg ARCT-810 initiated dosing in December 2024 in the United States. Each participant is expected to receive five intravenous infusions administered over two months....Phase 2 interim data for both mRNA therapeutic programs on track for first half of 2025."

P2 data • Trial status • Cystic Fibrosis • Genetic Disorders

SAFETY AND TOLERABILITY OF ARCT-810 MRNA IN EARLY TRIALS FOR ORNITHINE TRANSCARBAMYLASE DEFICIENCY (SSIEM 2024) - "ARCT-810 is generally safe and well tolerated in single doses up to 0.5 mg/kg, supporting the conduct of multiple-dose studies in younger subjects using a slower infusion rate to mitigate IRRs. Another multiple-dose study is being planned to enrol patients with more severe OTCD. Combined, these studies are expected to inform the design of pivotal efficacy studies in symptomatic patients with OTCD."

Clinical • Genetic Disorders

Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency (clinicaltrials.gov) - P1 | N=16 | Completed | Sponsor: Arcturus Therapeutics, Inc. | Active, not recruiting ➔ Completed | Phase classification: P1b ➔ P1 | N=12 ➔ 16 | Trial completion date: Aug 2023 ➔ Apr 2024

Enrollment change • Phase classification • Trial completion • Trial completion date • Genetic Disorders

Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 (clinicaltrials.gov) - P2 | N=24 | Active, not recruiting | Sponsor: Arcturus Therapeutics, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2024 ➔ Mar 2025 | Trial primary completion date: Dec 2023 ➔ Dec 2024

Enrollment closed • Trial completion date • Trial primary completion date • Genetic Disorders

A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD (clinicaltrials.gov) - P2 | N=9 | Recruiting | Sponsor: Arcturus Therapeutics, Inc.

New P2 trial • Genetic Disorders

IT translates: An update on the ARCT-810 mRNA therapy for OTC deficiency (SIMD 2024) - "The results from ARCT-810-01 and ARCT-810-02 studies facilitated the initiation of a multiple ascending dose study of ARCT-810(ARCT-810-03) in OTC-deficient adolescents and adults which is ongoing in the UK and EU. Subjects are randomized to receive 6 doses of ARCT-810 or placebo (randomized 3:1) over 10 weeks, dosed every 14 days, at 2 dose levels."

Genetic Disorders

Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 (clinicaltrials.gov) - P2 | N=24 | Recruiting | Sponsor: Arcturus Therapeutics, Inc. | Trial completion date: Dec 2023 ➔ Jun 2024

Trial completion date • Genetic Disorders

Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency (clinicaltrials.gov) - P1b | N=12 | Active, not recruiting | Sponsor: Arcturus Therapeutics, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2022 ➔ Aug 2023 | Trial primary completion date: Dec 2022 ➔ Aug 2023

Enrollment closed • Trial completion date • Trial primary completion date • Genetic Disorders

Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 (clinicaltrials.gov) - P2 | N=24 | Recruiting | Sponsor: Arcturus Therapeutics, Inc.

New P2 trial • Genetic Disorders

Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency (clinicaltrials.gov) - P1b | N=12 | Recruiting | Sponsor: Arcturus Therapeutics, Inc. | Trial completion date: Dec 2021 ➔ Dec 2022 | Trial primary completion date: Nov 2021 ➔ Dec 2022

Trial completion date • Trial primary completion date • Genetic Disorders

Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects (clinicaltrials.gov) - P1; N=30; Completed; Sponsor: Arcturus Therapeutics, Inc.; Recruiting ➔ Completed

Clinical • Trial completion

Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects (clinicaltrials.gov) - P1; N=30; Recruiting; Sponsor: Arcturus Therapeutics, Inc.; Trial primary completion date: Sep 2020 ➔ Dec 2020

Clinical • Trial primary completion date

Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency (clinicaltrials.gov) - P1b; N=12; Recruiting; Sponsor: Arcturus Therapeutics, Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With OTC Deficiency (clinicaltrials.gov) - P1b; N=12; Not yet recruiting; Sponsor: Arcturus Therapeutics, Inc.

Clinical • New P1 trial

Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects (clinicaltrials.gov) - P1; N=30; Recruiting; Sponsor: Arcturus Therapeutics, Inc.

Clinical • New P1 trial