Cardiomyopathy clinical trials at University of California Health
32 in progress, 19 open to eligible people
RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
open to eligible people ages 18 years and up
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.
at UCSD
EDG-7500 in Adults with Hypertrophic Cardiomyopathy (CIRRUS-HCM)
open to eligible people ages 18 years and up
This study is being conducted in order to understand the safety and effects of different doses of EDG-7500 as a single dose in adults with obstructive hypertrophic cardiomyopathy and as multiple doses in adults with obstructive or nonobstructive hypertrophic cardiomyopathy.
at UCSF
Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
open to eligible people ages 18 years and up
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
at UCSD
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.
at UCLA
AccuCinch® Ventricular Restoration System in Patients Who Present With Symptomatic Heart Failure With Reduced Ejection Fraction (HFrEF): The CORCINCH-HF Study
open to eligible people ages 18 years and up
Prospective, randomized, open-label, international, multi-center clinical study to evaluate the safety and efficacy of the AccuCinch Ventricular Restoration System in patients with heart failure and reduced ejection fraction (HFrEF).
at UCSD UCSF
Exercise Intolerance in Non-obstructive Hypertrophic Cardiomyopathy
open to eligible people ages 18-80
The EXCITE-HCM study is a randomized, controlled, blinded clinical trial designed to evaluate the effect of moderate intensity exercise training versus usual physicial activity on the improvement of HCM-related symptoms and cardiac function. About 70 participants will be recruited and randomized on a 1:1 ratio to either moderate intensity training or usual physicial activity interventions. Patients will be followed during a period of 24 weeks and assesesments as physical examination, questionnaires, 12 lead ecg's, biomarker levels, echocardiogram, Cardiac Magnetic resonance, PET and CPET will be performed to evaluate their response to the intervention.
at UCSF
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
open to eligible people ages 18-50
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.
at UCLA
Bromocriptine on Clinical Outcomes for Peripartum Cardiomyopathy
open to eligible females ages 18 years and up
The study will enroll 200 women newly diagnosed with peripartum cardiomyopathy within 5 months postpartum in a randomized placebo controlled trial of bromocriptine therapy to evaluate its impact on myocardial recovery and clinical outcomes. Given that bromocriptine prevents breastfeeding, an additional 50 women with peripartum cardiomyopathy excluded from the trial due to a desire to continue breastfeeding but meeting all other entry criteria will be followed in an observational cohort.
at UC Irvine UCSD
Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
open to eligible people ages 18-65
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.
at UCSF
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
open to eligible males ages 7 years and up
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
at UCLA
Aficamten Compared to Placebo in Adults With Symptomatic nHCM
open to eligible people ages 18-85
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.
at UCLA UCSD UCSF
Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure
open to eligible people ages 18 years and up
This is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized, multi-center trial study to evaluate the safety and efficacy of a single dose of AB-1002, administered via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and NYHA Class III symptoms of HF. Subjects will be randomized into one of three treatment groups in a 1:1:1
at UCSD
ALXN2220 Versus Placebo in Adults With ATTR-CM
open to eligible people ages 18-90
The primary objective of this study is to access the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the total occurrences of ACM and CV clinical events.
at UCSF
TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
open to eligible people ages 18-65
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 MYBPC3 mutation-associated nonobstructive hypertrophic cardiomyopathy (nHCM).
at UCSD UCSF
Registry Study to Assess Real-world Patient Characteristics, Treatment Patterns, and Longitudinal Outcomes in Patients Receiving Mavacamten and Other Treatments for Symptomatic Obstructive Hypertrophic Cardiomyopathy (Obstructive-HCM)
open to eligible people ages 18 years and up
This registry evaluates patient characteristics, real-world treatment patterns, and short- and long-term outcomes in a population of patients in the United States and Europe with symptomatic obstructive hypertrophic cardiomyopathy (HCM) who are receiving mavacamten, receiving other treatment for obstructive HCM, or not receiving treatment for obstructive HCM due to intolerance or failure of prior treatment. United States Sub-Study: The purpose of this study is to evaluate the safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting. Europe Sub-Study: The purpose of this study is to evaluate the effectiveness and safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting.
at UCSD UCSF
Danon Disease Natural History Study
open to eligible males ages 8 years and up
The goal of this international observational study is to learn about the natural history of Danon disease in male patients >8 years of age. The key objectives include assessing change over time in cardiac structure (left ventricular mass and wall thickness), cardiac biomarkers, symptoms, and quality of life and the incidence of clinical events such as HF hospitalization, cardiac transplantation, and death.
at UCSD
Evaluating Pulse Oximetry Bias in Children With Darker Skin Pigmentation
open to eligible people ages up to 21 years
In this prospective study, the investigators will enroll 154 children with arterial lines to determine the accuracy of pulse oximeters in children with darker skin pigmentation. Studies in adults suggest pulse oximeters may overestimate the true level of oxygenation in the blood as measured directly by co-oximetry. However, pediatric data are relatively limited. This study, which is funded by the FDA through the Stanford-UCSF (University of California San Francisco) Clinical Excellence in Regulatory Science and Innovation (CERSI) Program, will determine if the error/bias is associated with skin pigmentation and whether the error falls outside FDA standards. The broader purpose of the study is to work toward eliminating health disparities.
at UCSF
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy
open to eligible people ages 0-18
The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality of life (QoL). This study will also collect information on treatments, procedures and outcome in infants and children up to 18 yrs who have this mutation.
at UC Davis
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)
open to eligible people ages 14-65
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.
at UCSF
Mavacamten in Adults Who Have Completed MAVERICK-HCM or EXPLORER-HCM
Sorry, in progress, not accepting new patients
Approximately 30 sites that enrolled participants in the MAVERICK-HCM (MYK-461-006) study in the United States (US) will initiate this study. Approximately 90 sites that enrolled participants in the EXPLORER-HCM (MYK-461-005) study in the US, Europe, and Israel will initiate this study. Note: Approximately 30 centers overlap between MAVERICK and EXPLORER.
at UCSF
ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia
Sorry, not yet accepting patients
Friedreich Ataxia is a rare condition that causes damage to the nervous system and muscles. People with Friedreich Ataxia have difficulty walking, lose sensation in their arms and legs, and have slurred speech. It can also affect the heart and many people with Friedrich Ataxia develop serious heart problems. Friedreich Ataxia is a genetic condition which means a faulty gene is passed down through families. This type of gene therapy treats a genetic condition by providing a healthy copy of the gene. At the time this study started, there was no approved treatment for heart problems in people with Friedreich Ataxia. In this study, ASP2016 is being tested in humans for the first time. The people taking part are adults with Friedreich Ataxia who have heart problems. The main aims of the study are to check the safety of ASP2016 and how people cope with (tolerate) ASP2016. ASP2016 is given as a slow injection into a vein. This is called an infusion. People will also take tablets of a medicine called prednisolone. This is taken to stop the immune system interfering with ASP2016. Each person in the study will be given 1 single infusion of ASP2016. Different small groups will receive lower or higher doses of ASP2016. Each person will stay overnight in the clinic for at least 1 night after their infusion. For the first few months, people will visit the clinic regularly. There may be the option of home visits by a study nurse at some visits. At the 6-month and 12-month visits extra tests, procedures, and scans will be done. One of these is an ECHO (echocardiogram) scan. This is like an ultrasound scan for the heart. Another is an endomyocardial biopsy. A tiny piece of their heart tissue is removed (biopsy). A flexible hollow tube (catheter) goes into the blood vessels up to the heart. Then, a small device on the end of the catheter takes a tiny piece of heart tissue (about the size of a pencil tip). Another is a cardiac MRI. This takes pictures of the inside of the heart using a powerful magnet. Another is a cardiopulmonary exercise test (CPET). This involves moving a specially designed set of bicycle pedals using hands and arms. This will check how the lungs, heart and muscles are affected during exercise. After the 12-month visit, people will visit the clinic every few months for up to a few years.
at UCLA
Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy
Sorry, in progress, not accepting new patients
The purpose of this study is to evaluate the safety, tolerability, and efficacy of mavacamten compared with placebo in participants with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).
at UCSF
Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Sorry, not currently recruiting here
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
at UCLA
CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Sorry, in progress, not accepting new patients
To evaluate the efficacy of eplontersen compared to placebo in participants with ATTR-CM receiving available standard of care (SoC). For more information, please visit https://www.cardio-ttransform.com.
at UCSD UCSF
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.
at UCLA
Early Feasibility Evaluation of the AccuCinch® Ventricular Restoration System in Patients With Heart Failure and Reduced Ejection Fraction
Sorry, in progress, not accepting new patients
This is a non-randomized, prospective, multi-center Early Feasibility Study to evaluate the AccuCinch® Ventricular Restoration System in Patients with Heart Failure and Reduced Ejection Fraction (HFrEF).
at UCSF
Long-term Safety and Tolerability of Aficamten in Adults With HCM
Sorry, accepting new patients by invitation only
The purpose of this study is to collect long-term safety and tolerability data for aficamten.
at UCSD UCSF
AG10 in Patients With Cardiomyopathy
Sorry, in progress, not accepting new patients
This prospective, multicenter open-label study will evaluate the long-term safety, tolerability, PK and PD of AG10 administered on a background of stable heart failure therapy.
at UCSF
Aficamten Compared to Metoprolol Succinate in Adults With Symptomatic oHCM
Sorry, in progress, not accepting new patients
The purpose of this study is to compare the efficacy and safety of aficamten (CK-3773274) compared with metoprolol succinate in adults with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction
at UCSD
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.
at UC Irvine UCLA UCSD
Analysis of Both Sex and Device Specific Factors on Outcomes in Patients With Non-Ischemic Cardiomyopathy
Sorry, in progress, not accepting new patients
The purpose of this registry is to prospectively assess outcomes of device-treated ventricular tachyarrhythmias and all-cause mortality in non-ischemic cardiomyopathy patients indicated for ICD or CRT-D implantation for the primary prevention of sudden cardiac death. Differences in outcomes will be evaluated by sex and by device type.
at UC Davis
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.
at UC Davis UCLA UCSF
Our lead scientists for Cardiomyopathy research studies include Theodore P Abraham, M.D Liviu Klein, MD Afshan Hameed, MD Kalyanam Shivkumar, MD, PhD Barry Greenberg, MD Hilary Shapiro, MD Jason Bradfield, MD Jorge Silva Enciso, MD Marmar Vaseghi Eric Adler.
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