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Research Program

  1. In keeping with its mission, the ACHD Center maintains a dedicated commitment to its research program, resulting in numerous medical publications as well as national and international presentations. Faculty members examine the many issues in this growing field and place an emphasis on studies that can readily translate to patient care.

Current areas in which research endeavors are focused include:

  •  management of Eisenmenger's syndrome and pulmonary hypertension
  •  management of pulmonary pressures in Fontan patients
  •  management and predictors of pulmonary hypertension in adult CHD patients
  •  transcatheter valve replacement as an alternative strategy to surgical heart valve replacement
  •  mechanical support for the failing right ventricle and Fontan circulation 
  •  outcomes in the postoperative tetralogy of Fallot patients
  •  arrhythmia substrates in adult CHD
  •  outcomes of pregnancy in CHD
  •  stratification and management of high-risk pregnancy
  •  aortic compliance and effect on systemic ventricle, Marfan syndrome, bicuspid aortic valve, and tetralogy of Fallot patients
  •  contraceptive practices in women with CHD
  •  gene identification for congenital heart disease
 Clinical Trials
  1. Macitentan in Eisenmenger Syndrome to Restore Exercise Capacity (MAESTRO)

    This study is a multi-center, double-blind, randomized, placebo-controlled Phase 3 study. The goal of this study is to test whether the drug Macitentan improves exercise capacity in comparison with a placebo in patients with Eisenmenger Syndrome (ES). Following the Phase 3 trial, patients who complete the 16 week study will have the option to enroll in the open label study. The MAESTRO open-label extension study of the MAESTRO Phase 3 study will assess the long-term safety, tolerability and efficacy of Macitentan in subjects with Eisenmenger Syndrome.

  2. Efficacy of Remodulin in Adults with Congenital Heart Disease and Pulmonary Hypertension 

  3. This is a multi-center, open-label study to evaluate the efficacy of Remodulin in treatment of pulmonary hypertension in adults with congenital heart disease. Subjects will receive Remodulin for six months, with pre- and post-treatment assessments, including cardiopulmonary exercise tests and six minute walk tests. We anticipate that Remodulin will improve the functional capacity in these patients.


  4. Pulmonary Artery Repair with Covered Stents (PARCS) : The Use of Covered Cheatham Platinum Stents for Repair of Right Ventricular to Pulmonary Artery Conduits - Richard Ringel, MD John Hopkins University 

    The Food and Drug Administration (FDA) recently issued an investigational device exemption (IDE) for the Covered Cheatham Platinum Stents. In this study, the Covered Cheatham Platinum Stents are being for the treatment of Right Ventricle-Pulmonary Artery conduit disruption that can occur during cardiac catheterization procedures that would otherwise require emergency surgery. The purpose of this study is determine the efficacy of using Covered Cheatham Platinum Stents to treat RV-PA disruption during cardiac catheterizations. 

  5. Melody Transcather Pulmonary Valve Implantation within Bioprosthetic Valves       

The Food and Drug Administration (FDA) recently issued a humanitarian device exemption (HDE) for the Melody Transcatheter Pulmonary Valve (TPV). We plan to offer the Melody valve as an alternative to high-risk surgery in patients who require revision of a stenotic or regurgitant valve. The Melody valve may be used as an alternative to surgical pulmonary valve replacement in eligible patients. To date, melody valves have been implanted in 70 patients by the UCLA interventional pediatric/adult congenital cardiology team. The purpose of this study is to review a large cohort of Melody valve implantations within surgically placed bioprosthetic valves. 

  

Alliance for Adult Research in Congenital Cardiology (AARCC) Multi-Center Collaborations
  1. Tetralogy of Fallot: Echocardiographic Assessment of Pulmonary Regurgitation and the Right Ventricle (TEAR-RV) - Sasha Opotowsky, MD, Brigham & Women's Hospital

    This comprehensive evaluation of right ventricular function in patients with Tetralogy of Fallot will utilize echocardiography and MRI to identify parameters of prognostic value in this patient population. A comprehensive database of adults with Tetralogy of Fallot is also being generated under this project. Subject enrollment is ongoing.

  2. Health Education and Access Research Trial in Adults with Congenital Heart Disease (HEART-ACHD) - Michelle Gurvitz, MD, University of Washington

    The purpose of this study is to determine the prevalence of lapses in specialized congenital heart disease care in this patient population and to assess the impact of a targeted educational intervention at augmenting disease knowledge. We are continuing to enroll subjects in this study.

  3. Liver Health in Fontan Patients - Fred Wu, MD, Brigham & Women's Hospital

    This study is evaluating liver health in a multi-center cohort of adults with single ventricle congenital heart disease who have undergone the Fontan procedure. Specifically, the prognostic value of liver biopsy and newer non-invasive laboratory studies will be assessed. We are currently enrolling patients in this study.

Other Multi-Center Collaborations

 

1. Bicuspid Aortic Valve Aortopathy: Feasibility of a Compared Effectiveness Study - Craig Broberg, MD - Oregon Health & Science University??

Medical therapy is sometimes adopted to attenuate progressive aortic enlargement in patients with bicuspid aortic valve (BAV), yet no studies address the efficacy of such therapy. Based on data in patients with Marfan Syndrome, the potentially favored therapeutic strategies are beta blockade (BB) or anginotensin receptor blockade (ARB); however, BAV and Marfan syndrome are distinct clinical entities, and thus a randomized trial is needed to determine whether either strategy is superior to no treatment in altering the natural progression of aortic enlargement in patients with BAV. In order to accurately design such a study, this feasibility protocol was developed to obtain the necessary pilot data for a definitive trial. We hypothesize that superiority of one approach can be demonstrated. We will conduct a randomized, multi-center, prospective, open label trial evaluating beta blockade or angiotensin receptor blockade versus no treatment in patients with BAV and aortic dilatation.

2The Anticoagulation Therapy Study in Congenital Heart Disease (TACTIC) - Paul Khairy, MD,  PhD    Montreal Heart Institute

This is a multicenter retrospective study to describe and characterize the antiplatelet and anticoagulation     prescription drug patterns with the congenital heart disease population.  This study will also assess the incidence of bleeding complications and thromboembolic events in this patient populations.  The Ahmanson/UCLA Adult Congenital Heart Disease Center is collaborating with 15 other institutions across North America on this project in hopes to develop optimal management approaches and guidelines for antiplatelet and anticoagulant therapy. Two patient populations will be evaluated: 1) adults with congenital heart disease and atrial tachyarrhythmias, and 2) adults with congenital heart disease who have undergone Fontan palliation.

3. Adult Congenital Heart Disease / Eisenmenger Syndrome Quality Enhancement Research Initiate (ACHD/ES QuERI)

The ACHD QuERI is a multi-center, observational initiative targeting adult patients with repaired Congenital Heart Disease (CHD) at risk for developing Pulmonary Arterial Hypertension (PAH). The ACHD QuERI's target is to enroll 800 patients from 100 cardiology practices nation-wide over three years. The goal of ACHD QuERI is to characterize the demographics, clinical course and outcomes in a cohort of adult patients with repaired CHD at risk for developing PAH. The Eisenmenger Syndrome program (ES QuERI) will enroll 200 adult patients across the nation. The goal of ES QuERI is to characterize the demographics and clinical course of the patients with Eisenmenger Syndrome and to improve management of patients with Eisenmenger Syndrome through improved adherence to published guidelines for the management of patients with Eisenmenger Syndrome.

4. Congenital heart disease and neuroendocrine tumors - Alexander Opotowsky, MD/M.P.H, Boston Children's Hospital

This is a multi-center, retrospective study on patients with cyanotic congenital heart disease (CCHD). There have been a number of case reports suggesting an association between CCHD and neuroendocrine tumors, especially pheochromocytoma (pheo) and paraganglioma (PGL), neuroendocrine tumors arising from neural crest-derived cells or organs either in the adrenal gland (pheo) or along the central sympathetic and parasympathetic chains (PGL). These are rare tumors, with incidence of approximately 2 to 8 per million people a year. If there is a higher incidence of these tumors in patients with CCHD, this may suggest a causal mechanism. This study aims to 1) to characterize the demographics and clinical course of patients with congenital heart disease and neuroendocrine tumors, 2) to investigate possible mechanistic pathways, genetic and otherwise, associated with congenital heart disease and neuroendocrine tumors.

 5. Genetic Studies in Congenital Heart Disease - Joseph Wu, MD, Stanford University

Identifying the causal gene mutation is the first step in understanding a genetic disorder. The purpose of this study is to attempt to identify the genetic mutations responsible for specific forms of congenital heart disease. Specific diseases to be evaluated include Shone's complex and Tetralogy of Fallot. Patient enrollment is ongoing.

6. Contraception in Females with Congenital Heart Disease - Pamela Miner, MN, and Mary Canobbio, MN, UCLA

This study is being conducted for the Adult Congenital Cardiac Care Associate (ACCCA) Research Group, a forum for non-physician adult congenital heart disease providers and researchers to collaborate on multi-center clinical research. This study will evaluate the use of contraceptives in this patient population in an attempt to identify risk factors for adverse events.

7. Teen Survey of Lifelong Cardiac Care Knowledge

This multi-center study is being conducted in collaboration with the Adult Congenital Cardiac Care Associate (ACCCA) research group and will enroll subjects ages 13-18 with moderate and complex congenital heart disease.  The goal of the study is to assess adolescent understanding of the need for life-long cardiac care through administration of a standardized survey.  Children's Hospital Boston will serve as the lead site for this study.

  

 Translational Research
  1. Mechanical Support for the Failing Right Ventricle and Fontan Circulation

Ventricular assist devices have become the standard of care for patients with end-stage heart failure refractory to maximal medical management. However, few options exist for patients with single ventricle congenital heart disease and a failing Fontan circulation. We are currently conducting an animal study using the Jarvik 2000 left ventricular assist device for mechanical support of the failing right ventricle and the Fontan circulation. We believe the small size and versatility of this device will make it an ideal option for this application. After reviewing the safety and efficacy of the Jarvik 2000 in these animal studies, we hope to extend our findings to human patients with failing Fontan circulation that could potentially benefit from this application of the ventricular assist device.

 

 

For more information on research studies being conducted at the Ahmanson/UCLA Adult Congenital Heart Disease Center, please email us at ACHDC@mednet.ucla.edu.