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- || Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment open, Trial completion date, Trial primary completion date: MUSIC-DMD: Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy (clinicaltrials.gov) - Feb 26, 2025
P1, N=12, Recruiting, Sponsor: Sardocor Corp.
Not yet recruiting --> Recruiting | Trial completion date: Feb 2030 --> Oct 2030 | Trial primary completion date: Feb 2027 --> Oct 2027
- Mydicar (AAV1/SERCA2a) / Armata, Theragene
A Phase 1 Clinical Trial of High Dose AAV1.SERCA2a in Patients with Heart Failure: Modulation of SERCA2a of Intra-myocytic Calcium trafficking in Heart Failure with Reduced Ejection Fraction (MUSIC-HFrEF) (Ballroom 3) - Apr 22, 2024 - Abstract #ASGCT2024ASGCT_2496;
The early clinical efficacy at a dose of 3E13vg/patient of AAV1.SERCA2a will be followed by enrollment of patients at a higher dose at 4.5E13 vg/patient. These encouraging results of high dose AAV1.SERCA2a in patients with HFrEF not observed previously may offer alternative treatments to patients with severe heart failure where a large unmet need remains.
- || Mydicar (AAV1/SERCA2a) / Armata, Theragene
New P1 trial: MUSIC-DMD: Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy (clinicaltrials.gov) - Jan 25, 2024
P1, N=12, Not yet recruiting, Sponsor: Sardocor Corp.
- || Mydicar (AAV1/SERCA2a) / Armata, Theragene
Trial primary completion date: MUSIC-HFrEF1: Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction (clinicaltrials.gov) - Jan 22, 2024
P1/2, N=57, Recruiting, Sponsor: Sardocor Corp.
These encouraging results of high dose AAV1.SERCA2a in patients with HFrEF not observed previously may offer alternative treatments to patients with severe heart failure where a large unmet need remains. Trial primary completion date: Dec 2023 --> Dec 2025
- || Mydicar (AAV1/SERCA2a) / Armata, Theragene
New P1 trial: MUSIC-HFpEF: Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Preserved Ejection Fraction (clinicaltrials.gov) - Sep 29, 2023
P1b, N=10, Recruiting, Sponsor: Sardocor Corp.
- Mydicar (AAV1/SERCA2a) / Armata, Theragene
AAV.Cas9 and AAV.SERCA2a strategies to mitigate heart failure in DMD pigs (Station 3) - May 13, 2023 - Abstract #ESC2023ESC_3929;
Hence, AAV.SERCA2a combined with antitachycardic pacemaker protection may serve as a safe treatment option for DMD cardiomyopathy, whereas Cas9-mediated gene editing may provide additional therapy of electrical vulnerability. Further translational studies will establish novel gene therapeutic options for DMD patients.
- ||| Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment open: MUSIC-HFrEF1: Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction (clinicaltrials.gov) - Jun 21, 2022
P1/2, N=56, Recruiting, Sponsor: Sardocor Corp.
Further translational studies will establish novel gene therapeutic options for DMD patients. Not yet recruiting --> Recruiting
- Mydicar (AAV1/SERCA2a) / Armata, Theragene
AAV1.SERCA2a gene therapy for treating pulmonary hypertension in a pig model: efficacy of endo-bronchial versus intra-tracheal aerosolization to address the lung delivery bottleneck (Station 7) - Jun 15, 2022 - Abstract #ESC2022ESC_3972;
SERCA2a gene therapy delivered via endo-bronchial aerosolization ameliorate PH in diseased pigs. A direct comparison between endo-bronchial and intra-tracheal aerosolization revealed that the endo-bronchial delivery is a more efficient delivery method for AAV based PH gene therapy.
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene
Preclinical, Journal: Endo-bronchial aerosolized AAV1.SERCA2a gene therapy in a pulmonary hypertension pig model: addressing the lung delivery bottleneck. (Pubmed Central) - May 20, 2022
In part two of the study, we directly compared the endo-bronchial aerosolization gene delivery to the intra-tracheal aerosolization in PH pigs. Endo-bronchial delivery demonstrated higher viral expression (6,719 ± 927 vs 1,444 ± 402 vg copy/100ng DNA, p=0.0017), suggesting this delivery modality is a promising method for clinical AAV gene therapy for PH.
- || Mydicar (AAV1/SERCA2a) / Armata, Theragene
Trial initiation date: MUSIC-HFrEF1: Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction (clinicaltrials.gov) - Sep 21, 2021
P1/2, N=56, Not yet recruiting, Sponsor: Sardocor Corp.
Endo-bronchial delivery demonstrated higher viral expression (6,719 ± 927 vs 1,444 ± 402 vg copy/100ng DNA, p=0.0017), suggesting this delivery modality is a promising method for clinical AAV gene therapy for PH. Initiation date: Jun 2021 --> Oct 2021
- || Mydicar (AAV1/SERCA2a) / Armata, Theragene
Clinical, Journal: Investigation of the safety and feasibility of AAV1/SERCA2a gene transfer in patients with chronic heart failure supported with a left ventricular assist device - the SERCA-LVAD TRIAL. (Pubmed Central) - Aug 20, 2021
There were no safety concerns in this small cohort. This trial identified some of the challenges of performing gene therapy trials in this LVAD patient cohort which may help guide future trial design.
- |||| Mydicar (AAV1/SERCA2a) / Armata, Theragene
New P1/2 trial: MUSIC-HFrEF1: Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction (clinicaltrials.gov) - Jan 10, 2021
P1/2, N=56, Not yet recruiting, Sponsor: Sardocor Corp.
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene Pharma
Journal: AAV1.SERCA2a Gene Therapy Reverses Pulmonary Fibrosis by Blocking the STAT3/FOXM1 Pathway and Promoting the SNON/SKI Axis. (Pubmed Central) - Dec 30, 2020
Collectively, our results demonstrate that SERCA2a gene transfer attenuates bleomycin (BLM)-induced PF by blocking the STAT3/FOXM1 pathway and promoting the SNON/SKI Axis. Thus, SERCA2a gene therapy may be a potential therapeutic target for PF.
- [VIRTUAL] Impact of pulmonary hypertension on the left ventricular stiffness: a large animal study (Channel 5) - Jul 15, 2020 - Abstract #ERS2020ERS_4248;
- Mydicar (AAV1/SERCA2a) / Armata, Theragene Pharma
[VIRTUAL] SERCA2a gene therapy ameliorates pulmonary hypertension in a pig model: comparison of different delivery methods and therapeutic effect (Pre-congress content) - Jul 15, 2020 - Abstract #ERS2020ERS_1253;
Gene delivery using a bronchoscope is an efficient method for PH gene therapy. AAV1SERCA2 gene therapy using this delivery method ameliorates PH in a large animal model.
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene Pharma
Journal: Intra-tracheal gene delivery of aerosolized SERCA2a to the lung suppresses ventricular arrhythmias in a model of pulmonary arterial hypertension. (Pubmed Central) - Jan 10, 2020
Aerosolized AAV1.S2a gene delivery selectively to the lungs ameliorates myocardial EP remodeling and VT susceptibility at rapid heart rates. Our findings highlight for the first time the utility of a non-cardiac gene therapy approach for arrhythmia suppression.
- bleomycin / Generic Mfg., BMS, Mydicar (AAV1/SERCA2a) / AmpliPhi, Theragene Pharma
Serca2a Gene Therapy Inhibits Pulmonary Fibrosis By Blocking The Stat3/foxm1 Pathway And Promoting The Snon/ski Axis (Zone 2, Science and Technology Hall) - Aug 21, 2019 - Abstract #AHA2019AHA_7765;
Our findings highlight for the first time the utility of a non-cardiac gene therapy approach for arrhythmia suppression. Collectively, our in vitro and in vivo results further confirmed our hypothesis that SERCA2a gene therapy may represent a promising therapeutic strategy for treating patients with pulmonary fibrosis.
- Mydicar (AAV1/SERCA2a) / Armata, Theragene
Trial primary completion date, Metastases: A Phase 1/2 Study of High-dose Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure (clinicaltrials.gov) - Jan 31, 2017
P1/2, N=9, Terminated, Sponsor: Celladon Corporation
Collectively, our in vitro and in vivo results further confirmed our hypothesis that SERCA2a gene therapy may represent a promising therapeutic strategy for treating patients with pulmonary fibrosis. Trial primary completion date: Nov 2016 --> Jun 2015
- Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment change, Trial termination, Gene therapy: AGENT-HF: AAV1-CMV-Serca2a GENe Therapy Trial in Heart Failure (clinicaltrials.gov) - May 10, 2016
P2, N=10, Terminated, Sponsor: Assistance Publique - H
Trial primary completion date: Nov 2016 --> Jun 2015 N=44 --> 10 | Active, not recruiting --> Terminated
- |||| Mydicar (AAV1/SERCA2a) / Armata, Theragene
Trial completion, Metastases: CUPID-2b: A Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure (clinicaltrials.gov) - Mar 24, 2016
P2, N=250, Completed, Sponsor: Celladon Corporation
N=44 --> 10 | Active, not recruiting --> Terminated Active, not recruiting --> Completed
- Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment change, Trial termination, Trial primary completion date: SERCA-LVAD: Investigation of the Safety and Feasibility of AAV1/SERCA2a Gene Transfer in Patients With Chronic Heart Failure (clinicaltrials.gov) - Feb 24, 2016
P2, N=5, Terminated, Sponsor: Imperial College London
Active, not recruiting --> Completed N=24 --> 5 | Active, not recruiting --> Terminated | Trial primary completion date: Jan 2016 --> Sep 2015; Early termination following Trial Steering Committee recommendation
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment closed: SERCA-LVAD: Investigation of the Safety and Feasibility of AAV1/SERCA2a Gene Transfer in Patients With Chronic Heart Failure (clinicaltrials.gov) - Sep 10, 2015
P2, N=24, Active, not recruiting, Sponsor: Imperial College London
N=24 --> 5 | Active, not recruiting --> Terminated | Trial primary completion date: Jan 2016 --> Sep 2015; Early termination following Trial Steering Committee recommendation Recruiting --> Active, not recruiting
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment closed, Trial primary completion date, Gene therapy: AGENT-HF: AAV1-CMV-Serca2a GENe Therapy Trial in Heart Failure (clinicaltrials.gov) - Aug 11, 2015
P2, N=44, Active, not recruiting, Sponsor: Assistance Publique - H
Recruiting --> Active, not recruiting Recruiting --> Active, not recruiting | Trial primary completion date: Dec 2015 --> Jul 2015
- Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment change, Trial termination, Metastases: A Phase 1/2 Study of High-dose Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure (clinicaltrials.gov) - Jul 8, 2015
P1/2, N=9, Terminated, Sponsor: Celladon Corporation
Recruiting --> Active, not recruiting | Trial primary completion date: Dec 2015 --> Jul 2015 N=36 --> 9 | Recruiting --> Terminated; Sponsor has suspended clinical development of MYDICAR for heart failure.
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment open, Metastases: A Phase 1/2 Study of High-dose Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure (clinicaltrials.gov) - Apr 27, 2015
P1/2, N=36, Recruiting, Sponsor: Celladon Corporation
N=36 --> 9 | Recruiting --> Terminated; Sponsor has suspended clinical development of MYDICAR for heart failure. Not yet recruiting --> Recruiting
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene
New P1/2 trial, Metastases: A Phase 1/2 Study of High-dose Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure (clinicaltrials.gov) - Jan 28, 2015
P1/2, N=36, Not yet recruiting, Sponsor: Celladon Corporation
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment open: SERCA-LVAD: Investigation of the Safety and Feasibility of AAV1/SERCA2a Gene Transfer in Patients With Chronic Heart Failure (clinicaltrials.gov) - Aug 15, 2014
P2, N=24, Recruiting, Sponsor: Imperial College London
Not yet recruiting --> Recruiting Not yet recruiting --> Recruiting
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment open, Gene therapy: AGENT-HF: AAV1-CMV-Serca2a GENe Therapy Trial in Heart Failure (clinicaltrials.gov) - Apr 10, 2014
P2, N=44, Recruiting, Sponsor: Assistance Publique - H
Not yet recruiting --> Recruiting Not yet recruiting --> Recruiting
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene
Trial initiation date: SERCA-LVAD: Investigation of the Safety and Feasibility of AAV1/SERCA2a Gene Transfer in Patients With Chronic Heart Failure (clinicaltrials.gov) - Mar 12, 2014
P2, N=24, Not yet recruiting, Sponsor: Imperial College London
Not yet recruiting --> Recruiting Initiation date: Jan 2010 --> May 2014
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment change: SERCA-LVAD: Investigation of the Safety and Feasibility of AAV1/SERCA2a Gene Transfer in Patients With Chronic Heart Failure (clinicaltrials.gov) - Mar 12, 2014
P2, N=24, Not yet recruiting, Sponsor: Imperial College London
Initiation date: Jan 2010 --> May 2014 N=16 --> 24
- ||| Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment closed, Metastases: CUPID-2b: A Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure (clinicaltrials.gov) - Mar 9, 2014
P2, N=250, Active, not recruiting, Sponsor: Celladon Corporation
N=16 --> 24 Recruiting --> Active, not recruiting
- ||| Mydicar (AAV1/SERCA2a) / Armata, Theragene
Enrollment change, Metastases: CUPID-2b: A Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure (clinicaltrials.gov) - Nov 9, 2013
P2, N=250, Active, not recruiting, Sponsor: Celladon Corporation
Recruiting --> Active, not recruiting N=200 --> 250
- | Mydicar (AAV1/SERCA2a) / Armata, Theragene
New P2 trial, Gene therapy: AGENT-HF: AAV1-CMV-Serca2a GENe Therapy Trial in Heart Failure (clinicaltrials.gov) - Oct 20, 2013
P2, N=44, Recruiting, Sponsor: Assistance Publique - H
- |||| Mydicar (AAV1/SERCA2a) / Armata, Theragene
Trial completion, Metastases: CUPID: Efficacy and Safety Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure (clinicaltrials.gov) - Jul 31, 2013
P1/2, N=51, Completed, Sponsor: Celladon Corporation
N=200 --> 250 Active, not recruiting --> Completed
- ||||| Mydicar (AAV1/SERCA2a) / Armata, Theragene
New P2 trial, Metastases: CUPID-2b: A Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure (clinicaltrials.gov) - Jul 16, 2012
P2, N=250, Active, not recruiting, Sponsor: Celladon Corporation