Cardiomyopathy clinical trials at UCLA
11 in progress, 4 open to eligible people
Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
open to eligible people ages 12-17
The purpose of this study is to evaluate the efficacy, safety and PK of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
Los Angeles, California and other locations
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
open to eligible people ages 18-75
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like substance that accumulates in different organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart. One way occurs as a person ages, where the normal TTR protein can fall apart and form amyloid that may no longer be sufficiently cleared by the body. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way occurs when a person inherits a defective TTR gene that causes the TTR protein to spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and can be detected in adults by a genetic test of their TTR gene before they age. Amyloid build-up in the heart causes the heart wall to become thick and stiff and can result in heart failure and even death. Accumulation of TTR amyloid in the heart is known as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve tissues leading to nerve problems. Accumulation of TTR in the nerves is known as transthyretin amyloid polyneuropathy or ATTR-PN. Acoramidis is an experimental drug designed to bind tightly to TTR in the blood and stabilize its structure, so it does not form the harmful amyloid plaques that can cause damage to organs. This study is intended to determine if treatment with acoramidis in participants with ATTRv who have not yet developed any symptoms of disease can prevent or delay the development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene are treated early before any of the symptoms of disease have developed, it may be possible to delay the onset or prevent the disease entirely.
Los Angeles, California and other locations
PIVATAL Study -Study of Ventricular Arrhythmia (VTA) Ablation in Left Ventricular Assist Device (LVAD) Patients
open to eligible people ages 18 years and up
To investigate the effect of VTA ablation at the time of LVAD implant to see if it can reduce the incidence of VTA after surgery
Los Angeles, California and other locations
Cardiac Sympathetic Denervation for Prevention of Ventricular Tachyarrhythmias
open to eligible people ages 18 years and up
The purpose of this research study is to examine the effect of cardiac sympathetic denervation (CSD) surgery on life threatening abnormal heart rhythms called ventricular tachycardia or ventricular fibrillation that can lead to sudden cardiac death. Subjects will be asked to participate in this research study if they have recurrent ventricular tachycardia (at least one ICD shock for ventricular tachycardia) and have undergone at least one catheter ablation procedure or have ventricular tachycardia or fibrillation that is not ablatable. The goal of this study is to determine whether cardiac sympathetic denervation can prevent these abnormal heart rhythms from occurring and therefore, prevent, ICD shocks which are not only painful, but have been shown to reduce quality of life and/or lead to depression, particularly in the period immediately after the shock.
Los Angeles, California and other locations
Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Sorry, in progress, not accepting new patients
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
Los Angeles, California and other locations
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
Sorry, in progress, not accepting new patients
This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy. The study will evaluate up to three doses of single administration of LX2006 (AAVrh.10hFXN), an adeno-associated virus (AAV) gene therapy designed to intravenously deliver the human frataxin (hFXN) gene to cardiac cells over a 52-week period. Long-term safety and efficacy will be evaluated for an additional 4-years for a total of 5-years post LX2006 treatment.
Los Angeles, California and other locations
DCM Precision Medicine Study
Sorry, in progress, not accepting new patients
The aims of the DCM Precision Medicine Study are to test the hypothesis that DCM has substantial genetic basis and to evaluate the effectiveness of a family communication intervention in improving the uptake and impact of family member clinical screening.
Los Angeles, California and other locations
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Sorry, in progress, not accepting new patients
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young men in their late twenties. DMD is the most common fatal genetic disorder diagnosed in childhood. It affects approximately 1 in every 3,500 live male births across all races and cultures, and results in 20,000 new cases each year worldwide.Significant advances in respiratory care have unmasked CM as the leading cause of death. As there are yet no specific cardiac treatments to extend life, the current study aims to address this unmet medical need using a new therapeutic strategy for patients with DMD. Funding Source - FDA OOPD
Los Angeles, California and other locations
Aficamten Compared to Placebo in Adults With Symptomatic nHCM
Sorry, in progress, not accepting new patients
This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.
Los Angeles, California and other locations
AT-001 in Patients With Diabetic Cardiomyopathy
Sorry, in progress, not accepting new patients
This is a multicenter, randomized, placebo-controlled, 2-part study to evaluate the safety and efficacy of AT-001 in adult patients (N=675) with Diabetic Cardiomyopathy at high risk of progression to overt heart failure.
Torrance, California and other locations
IVTCC 2.0: a Prospective Multicenter Ventricular Tachycardia Catheter Ablation Registry
Sorry, accepting new patients by invitation only
This is a prospective multi-center international registry. The objective of this registry is to collect prospective data on patients undergoing catheter ablation for Ventricular Tachycardia (VT) and Premature Ventricular Contractions (PVC). The registry will be used for clinical monitoring, research, and quality improvement purposes.
Los Angeles, California and other locations
Our lead scientists for Cardiomyopathy research studies include Marmar Vaseghi, MD, PhD Kalyanam Shivkumar Nancy Halnon Jason Bradfield, MD.
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