Treatment Trials

9 Clinical Trials for Various Conditions

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COMPLETED
Pilot Randomized Trial With Flecainide in ARVC Patients
Description

Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) is an inherited arrhythmia disorder with high risk of ventricular tachycardia or fibrillation, and implantable cardioverter defibrillator remains as therapy of choice. Antiarrhythmic therapy with different agents including beta-blockers, sotalol and amiodarone are usually not effective in reducing risk of arrhythmic events. Recent data indicated that flecainide effectively prevented the arrhythmias observed in the experimental ARVC animals and in small series of ARVC patients. These observations provide a strong rationale for conducting a pilot randomized clinical trial to determine whether flecainide will reduce ventricular arrhythmias in high-risk ARVC patients. This pilot study is designed as randomized double-blinded placebo-controlled crossover trial with administration of 100 mg of Flecainide or matching placebo twice a day for 4 weeks each with a washout period. Primary specific aim of this pilot trial is to determine whether Flecainide administration is associated with a significant reduction of number of ventricular ectopic beats (VEBs) in ARVC patients with implantable cardioverter-defibrillator (ICD).

RECRUITING
Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
Description

This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.

RECRUITING
Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
Description

This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC.

RECRUITING
A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACM
Description

An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM.

ACTIVE_NOT_RECRUITING
IC14 (Atibuclimab) in Arrhythmogenic Cardiomyopathy
Description

The goal of this clinical trial is to test IC14 (atibuclimab) in patients with arrhythmogenic cardiomyopathy (ACM) and who have an implantable cardoverter/defibrillator in place. ACM is also called arrhythmogenic right ventricular dysplasia (ARV) or arrhythmogenic right ventricular cardiomyopathy (ARVC). The main questions the study aims to answer are the effect of treatment on blood markers of inflammation, safety, and pharmacokinetics. There will also be measurements of myocardial imaging of C-C chemokine receptor type 2 (CCR2+) immune cells (optional), monitoring of cardiac arrhythmias using the patient's pre-existing intracardiac cardioverter/defibrillator (ICD) and a Holter monitor, electrocardiogram (ECG), echocardiogram (ECHO), and blood tests. Results will be compared to baseline; there is no inactive placebo treatment group. Participants will be asked to undergo screening and baseline testing, then receive 4 intravenous infusions with blood measurements before and after the infusion (including 24, 48, and 72 hours and 7, 14, and 28 days). Participants will be offered specialized scanning of the heart muscle, and will be asked to provide recordings from their ICD, undergo Holter monitoring twice, and have electrocardiograms (ECG), echocardiograms (ECHO) and blood tests.

RECRUITING
Gene Therapy for ACM Due to a PKP2 Pathogenic Variant
Description

This is a Phase 1/2, first-in-human, open-label, intravenous, dose-escalating, multicenter trial that is designed to assess the safety and tolerability of LX2020 in adult patients with PKP2-ACM

RECRUITING
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Description

This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with PKP2-ACM.

COMPLETED
Diagnostic Value and Safety of Flecainide Infusion Test in Brugada Syndrome
Description

The study aims to use flecainide infusion test as diagnostic test to unmask concealed Brugada Syndrome cases. It proposes to assess the safety profile of this test in US patients and its higher sensitivity when compared to procainamide infusion (the conventional drug used in the USA). As a substudy it proposes to apply this test to early ARVC cases in order to evaluate if ECG changes similar to those seen in Brugada Syndrome could be unmasked by flecainide iv.

COMPLETED
Isoproterenol Challenge to Detect Arrhythmogenic Right Ventricular Cardiomyopathy
Description

This study will examine the usefulness of a new test called an isoproterenol challenge in patients with arrhythmogenic right ventricular cardiomyopathy (ARVC) and family members who may have the disease but do not have clear-cut evidence of it. ARVC is a rare condition that runs in families. Heart muscle is replaced with fatty, scar-like tissue, especially in the right ventricle (lower pumping chamber of the heart), and can sometimes extend to the left ventricle (the main pumping chamber). The fat can interfere with the heartbeat, producing abnormal heart rhythms, such as ventricular tachycardia (VT) - a very fast heartbeat that can cause sudden death, especially in young people. Isoproterenol is a drug that increases heart rate and heart muscle contractions. In isoproterenol challenge, subjects are given increasing doses of the drug through a catheter (see details below) to try to produce an abnormal heart rhythm. ARVC is hard to diagnose with current tests. This study will see if isoproterenol challenge provokes VT in patients with the disease and can confirm the diagnosis; if it can detect the disease in family members better than currently available tests; and if it provokes abnormal rhythms in healthy control subjects. In addition, the study will explore the genetics of ARVC and determine whether infection could contribute to its development. Patients with ARVC, their family members, and normal volunteers 18 years of age and older may be eligible for this study. Candidates are screened with a medical history and physical examination, electrocardiogram (EKG), treadmill and bicycle exercise testing, and an echocardiogram (ultrasound test of the heart). Participants undergo the following tests and procedures: * Blood tests - Blood is collected to study the genetics of ARVC, to test for evidence of old infections, and to measure brain natriuretic peptide - a hormone that can increase with development of heart failure. * Heart magnetic resonance imaging (MRI). This test looks at heart structure and function. MRI uses a magnetic field and radio waves to produce images of body tissues and organs. The subject lies on a table that is moved into the scanner (a narrow cylinder), wearing earplugs to muffle loud knocking sounds that occur during the scanning process. At some time during the test, the subject is given a contrast agent called gadolinium through a catheter (thin, flexible tube) in a vein to improve the scan pictures. The scan time varies from 30 to 90 minutes, with most scans lasting 60 minutes. (Control subjects do not undergo MRI.) * Isoproterenol challenge. Subjects are given increasing doses of isoproterenol through a catheter until the heart rate reaches 100 to 120 beats per minute for no more than 1 hour. A special EKG records heart rhythm during the test and an echocardiogram records right and left ventricular function. * QRST surface mapping EKG. This special EKG, done with 64 or 120 leads, maps abnormalities of heart rhythm and cardiac conduction during the isoproterenol challenge. These tests are like a regular EKG, except that more leads are placed on the chest, and on the back as well. Patients and family members who wish to have follow-up visits may return to the NIH Clinical Center once a year for 5 years for guidance about therapy based on clinical considerations and new information or investigations.