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A Phase 1 Dose Escalation Trial Evaluating An Intravenously Administered Recombinant Adeno-associated Virus Serotype Rh.74 (AAVrh.74) Vector Containing The Human BCL2-associated Athanogene 3 (BAG3) Gene Coding Sequence (RP-A701) In Subjects With Dilated Cardiomyopathy Arising From Pathogenic BAG3 Variants (BAG3-DCM)
Rochester, MN
This is a Phase 1, open-label, dose-escalation trial to characterize the safety, tolerability, and preliminary efficacy of RP-A701 following a single IV administration in high-risk adult patients with BAG3-DCM.
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A Phase 1/2, First-in-Human, Open-Label, Multicenter, Single-Dose Study To Evaluate The Safety, Tolerability, And Preliminary Efficacy Of NVC-001, A Novel Adeno-Associated Virus (Serotype 9) Vector Expressing A Dominant Negative SUN1 Transgene, In Patients With LMNA-Related Dilated Cardiomyopathy (SUNBEAM-LMNA)
Rochester, MN
This study is designed to evaluate the safety and tolerability of NVC-001 in adults with LMNA-related dilated cardiomyopathy (LMNA-DCM). The study will also assess exploratory measures of efficacy.
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A Phase 1b, Multicenter, Open-Label, Dose Finding Study To Investigate The Safety And Tolerability Of A Single Intravenous Dose Of SGT-501 In Patients With Catecholaminergic Polymorphic Ventricular Tachycardia
Rochester, MN
The purpose of this study is to investigate the safety and tolerability of a single intravenous (IV) dose of SGT-501.
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Evaluation of a Novel Diagnostic Test for Calcium Release Deficiency Syndrome
Rochester, MN
The purpose of this study is to investigate the response to either an atrial or ventricular pacing train as a diagnostic test for Calcium Release Deficiency Syndrome (CRDS).
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First-in-Human, Open-Label, Safety, Tolerability, Dose Finding, Pharmacodynamic And Cardiac Transgene Expression Study Of TN-401, A Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Plakophilin-2 (PKP2) Transgene, In Adults With PKP2 Mutation-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
Rochester, MN
The purpose of this study is to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-401 in adult patients with symptomatic PKP2 mutation-associated ARVC. The trial will consist of 2 escalating dose cohorts. All patients will receive active drug.
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First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic And Cardiac Transgene Expression Study Of TN-201, A Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Myosin Binding Protein C Transgene, In Adults With MYBPC3 Mutation-associated Hypertrophic Cardiomyopathy (HCM)
Rochester, MN
This is a first-in-human, non-randomized, open-label study designed to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-201 in adult patients with symptomatic hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.
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International CRDS Registry
Rochester, MN
The purpose of this research study is to establish a large registry of individuals diagnosed with CRDS, along with individuals that have genetic changes that could potentially place them at risk for a CRDS-like condition, from around the world.
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A Phase 2/3, Two-part, Dose-ranging, Adaptive Study To Evaluate Efficacy And Safety Of THRV-1268 In Participants Diagnosed With Long QT Syndrome Type 2 (LQTS 2) And Type 3 (LQTS 3)
Rochester, MN
The goal of this clinical trial is to learn whether the study drug THRV-1268 can safely and effectively shorten the QT interval in people diagnosed with Long QT Syndrome Type 2 (LQTS 2) and Long QT Syndrome Type 3 (LQTS 3). The study will also learn about the safety and tolerability of THRV-1268 at different doses.
The main questions this study aims to answer are:
Does THRV-1268 reduce the QTc interval (a measure of the heart's electrical recovery time)?
What side effects or medical problems occur when participants take THRV-1268?
Which dose of THRV-1268 works best and is safest?
Participants will:
Complete a 3-week observation period with ECG and Holter monitoring to establish baseline QTc measurements Take THRV-1268 twice daily at two dose levels for 6 weeks (Part A) or be randomly assigned to a dose group for 6 weeks (Part B - cohort 1)
Have clinic visits and tests to monitor safety and changes in their heart rhythm
May continue taking THRV-1268 for up to 1 year for ongoing safety and efficacy evaluation
Researchers will compare changes in QTc over time and evaluate side effects to determine whether THRV-1268 can help reduce the risk of abnormal heart rhythms and sudden cardiac events in people with LQTS 2 and LQTS 3.
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Identification Of Circulating Biomarkers For PRKAG2- And PLN-Related Cardiomyopathies
Rochester, MN
The purpose of this study is to identify circulating biomarkers specific to two rare genetic cardiomyopathies associated with disease-causative variants in PRKAG2 and PLN.
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