- |||||||||| ARCT-021 / Arcturus Therap, CSL Behring, Meiji Seika
Phase classification: Open Label Extension Study to Assess the Safety and Long-Term Immunogenicity of ARCT-021 (clinicaltrials.gov) - Jan 4, 2025 P2, N=65, Terminated, Self-amplifying mRNA vaccines could provide a substantial contribution to pandemic preparedness and response, inducing robust immune responses with a lower dose of mRNA to allow wider and more equitable distribution. Phase classification: P2a --> P2
- |||||||||| Kostaive (ARCT-154) / Arcturus Therap
Journal, IO biomarker: Delivery vehicle and route of administration influences self-amplifying RNA biodistribution, expression kinetics, and reactogenicity. (Pubmed Central) - Sep 21, 2024 The approval of saRNA-based vaccines, such as the ARCT-154 for COVID-19 in Japan, underscores its potential for diverse therapeutic applications, including vaccine development, cancer immunotherapy, and gene therapy...Additionally, our research unveiled distinct biodistribution profiles and inflammatory responses contingent upon the chosen delivery formulation and route. This research illuminates the intricate dynamics governing saRNA delivery, biodistribution and reactogenicity, offering essential insights for optimizing therapeutic strategies and advancing the clinical and commercial viability of saRNA technologies.
- |||||||||| ARCT-810 / Arcturus Therap
Trial completion, Phase classification, Enrollment change, Trial completion date: Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency (clinicaltrials.gov) - Aug 2, 2024 P1, N=16, Completed, Data from this study will support advancement of ARCT-032 to a multiple-dose study in PwCF. Active, not recruiting --> Completed | Phase classification: P1b --> P1 | N=12 --> 16 | Trial completion date: Aug 2023 --> Apr 2024
- |||||||||| ARCT-165 / Arcturus Therap, Kostaive (ARCT-154) / Arcturus Therap, ARCT-021 / Arcturus Therap, CSL Behring, Meiji Seika
Trial completion, Trial completion date, Trial primary completion date: A Trial Evaluating the Safety and Immunogenicity of 3 COVID-19 SARS-CoV-2 RNA Vaccines in Healthy Adults (clinicaltrials.gov) - Jul 30, 2024 P1/2, N=72, Completed, Combined, these studies are expected to inform the design of pivotal efficacy studies in symptomatic patients with OTCD. Recruiting --> Completed | Trial completion date: Mar 2023 --> Dec 2023 | Trial primary completion date: Mar 2023 --> Dec 2023
- |||||||||| ARCT-154 / Arcturus Therap
Clinical, Clinical Trial,Phase I, Clinical Trial,Phase II, Clinical Trial,Phase III, Journal: Safety, immunogenicity and efficacy of the self-amplifying mRNA ARCT-154 COVID-19 vaccine: pooled phase 1, 2, 3a and 3b randomized, controlled trials. (Pubmed Central) - May 15, 2024 P2/3 Efficacy of ARCT-154 was 56.6% (95% CI: 48.7- 63.3) against any COVID-19, and 95.3% (80.5-98.9) against severe COVID-19. ARCT-154 vaccination is well tolerated, immunogenic and efficacious, particularly against severe COVID-19 disease.
- |||||||||| Comirnaty (tozinameran) / Pfizer, Fosun Pharma, BioNTech, ARCT-154 / Arcturus Therap, Meiji Seika
Journal: Self-amplifying RNA COVID-19 vaccine. (Pubmed Central) - Apr 17, 2024 Clinical trials showed comparable safety and efficacy using a lower dose compared to the mRNA vaccine BNT162b2. To view this Bench-to-Bedside, open or download the PDF.
- |||||||||| Comirnaty (tozinameran) / Pfizer, Fosun Pharma, BioNTech, ARCT-154 / Arcturus Therap, Meiji Seika
P3 data, Journal, Head-to-Head: Immunogenicity and safety of a booster dose of a self-amplifying RNA COVID-19 vaccine (ARCT-154) versus BNT162b2 mRNA COVID-19 vaccine: a double-blind, multicentre, randomised, controlled, phase 3, non-inferiority trial. (Pubmed Central) - Mar 25, 2024 Increased immune responses at 28 days might provide increased likelihood of protection against these strains during this period and could also result in longer duration of protection. Further studies will assess the immunogenicity induced against more recent SARS-CoV-2 variants.
- |||||||||| ARCT-032 / Arcturus Therap
Inhaled LUNAR (Clyde Auditorium) - Mar 22, 2024 - Abstract #ECFS2024ECFS_699; Single doses of ARCT-032 were generally safe and well-tolerated in healthy adults. Unblinded safety data will be presented at the conference, as well as preliminary data from Part 2 CF participants.
- |||||||||| Kalydeco (ivacaftor) / Vertex, ARCT-032 / Arcturus Therap
LUNAR (105 C (West)) - Jul 27, 2023 - Abstract #NACFC2023NACFC_630; This study demonstrated that the LUNAR platform is a robust vehicle for therapeutic mRNA delivery into airway epithelial cells in vivo and that LUNAR-CFTR can correct the CF-associated MCC defect in ferrets. Furthermore, CF ferrets with the ROSA26mT/mG transgenic background are a robust tool to evaluate the efficiency of gene transfer and gene editing in mucus-laden airways.
- |||||||||| Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex, ARCT-032 / Arcturus Therap
LUNAR (105 C (West)) - Jul 27, 2023 - Abstract #NACFC2023NACFC_399; Absolute current magnitude after LUNAR-CFTR treatment was comparable with that in wt controls in the presence of a Cl gradient and equivalent to ETI in its absence. Immunohistochemistry characterization is in progress to evaluate CFTR-transfected cell popula-tions contributing to Cl transport in CF hBECs.
- |||||||||| Kostaive (ARCT-154) / Arcturus Therap
Trial completion, Trial completion date: The ARCT-154 Self-Amplifying RNA Vaccine Efficacy Study (ARCT-154-01) (clinicaltrials.gov) - Apr 28, 2023 P2/3, N=19467, Completed, Immunohistochemistry characterization is in progress to evaluate CFTR-transfected cell popula-tions contributing to Cl transport in CF hBECs. Active, not recruiting --> Completed | Trial completion date: Aug 2023 --> Jan 2023
- |||||||||| ARCT-810 / Arcturus Therap
Enrollment closed, Trial completion date, Trial primary completion date: Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency (clinicaltrials.gov) - Apr 7, 2023 P1b, N=12, Active, not recruiting, Not yet recruiting --> Recruiting Recruiting --> Active, not recruiting | Trial completion date: Dec 2022 --> Aug 2023 | Trial primary completion date: Dec 2022 --> Aug 2023
- |||||||||| UX053 / Ultragenyx, Arcturus Therap
Enrollment closed, Enrollment change, Trial completion date, Trial primary completion date: Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) (clinicaltrials.gov) - Feb 21, 2023 P1/2, N=8, Active, not recruiting, Active, not recruiting --> Terminated; Sponsor decision not related to safety concerns Recruiting --> Active, not recruiting | N=18 --> 8 | Trial completion date: Jun 2024 --> Mar 2023 | Trial primary completion date: Jun 2024 --> Mar 2023
- |||||||||| Comirnaty (tozinameran) / Pfizer, Fosun Pharma, BioNTech, ARCT-021 / Arcturus Therap
Journal, IO biomarker: Immune gene expression analysis indicates the potential of a self-amplifying Covid-19 mRNA vaccine. (Pubmed Central) - Nov 29, 2022 Moreover, the ARCT-021 signature correlated with day 7 YF17D live-attenuated vaccine transcriptomic responses. Altogether, our findings show that sa-mRNA vaccination induces innate immune responses that are associated with the development of adaptive immunity from other forms of vaccines, supporting further development of this vaccine platform for clinical application.
- |||||||||| UX053 / Ultragenyx, Arcturus Therap
Enrollment change, Trial completion date, Trial primary completion date: Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) (clinicaltrials.gov) - Oct 24, 2022 P1/2, N=18, Recruiting, Altogether, our findings show that sa-mRNA vaccination induces innate immune responses that are associated with the development of adaptive immunity from other forms of vaccines, supporting further development of this vaccine platform for clinical application. N=30 --> 18 | Trial completion date: Sep 2023 --> Jun 2024 | Trial primary completion date: Sep 2023 --> Jun 2024
- |||||||||| ARCT-021 / Arcturus Therap, CSL Behring, Meiji Seika
Enrollment change, Trial completion date, Trial termination, Trial primary completion date: Open Label Extension Study to Assess the Safety and Long-Term Immunogenicity of ARCT-021 (clinicaltrials.gov) - Jul 8, 2022 P2a, N=65, Terminated, N=106 --> 65 | Trial completion date: Jun 2022 --> Dec 2021 | Recruiting --> Terminated | Trial primary completion date: Jun 2022 --> Dec 2021; A decision was made to terminate the study for operational/business reasons. This study was not terminated for reasons of safety or immunogenicity.
- |||||||||| Kostaive (ARCT-154) / Arcturus Therap
Enrollment closed: The ARCT-154 Self-Amplifying RNA Vaccine Efficacy Study (ARCT-154-01) (clinicaltrials.gov) - Jan 11, 2022 P2/3, N=19400, Active, not recruiting, Trial completion date: Dec 2021 --> Dec 2022 | Trial primary completion date: Nov 2021 --> Dec 2022 Recruiting --> Active, not recruiting
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