Clinical trials: Metabolic Cardiomyopathy
Cardiomyopathy, Hypertrophic, Cardiomyopathy, Dilated
National Network for Cardiovascular Genomics: Advancing Cardiovascular Healthcare for Hereditary Diseases in Brazil's Unified Health System Through a Multicenter Registry
The goal of this observational study is to develop a registry of Brazilian patients with hereditary cardiovascular diseases, combining clinical and genomic data.
- Conditions
- Cardiomyopathy, Hypertrophic, Cardiomyopathy, Dilated, Cardiomyopathy Restrictive, Arrhythmogenic Right Ventricular Dysplasia
- Trial name
- RENOMICA-Hcor (NCT06546137)
- Testing
- whole genome sequencing
- Where
- 27 sites in Brazil
- Volunteers
- 1,211 people
- Sponsor
- Hospital do Coracao
Mitochondrial Disorders, Mitochondrial Genetic Disorders
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.
- Conditions
- Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes
- Trial name
- NAMDC (NCT01694940)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Columbia University
Cardiac Failure, Coronary Artery Disease
Evaluation of MyoStrain™ in Clinical Practice
Evaluate MyoStrain cardiac MRI pulse sequence in Clinical practice
- Conditions
- Cardiac Failure, Coronary Artery Disease, Sarcoidosis, Amyloidosis
- Trial name
- NCT03825224
- Testing
- Diagnostic Test: Myostrain
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- University of Kansas Medical Center
Diabetic Cardiomyopathy
Mechanisms Responsible for Cardiac and Skeletal Muscle Energetic Impairment in Diabetes
Diabetes increases the risk of heart failure. This is mainly due to a disease of the blood vessels supplying the heart muscle and/or high blood pressure, but abnormal metabolism may also contribute.
- Trial name
- DDCM (NCT00628056)
- Testing
- Perhexiline
- Where
- United Kingdom
- Volunteers
- 75 people
- Sponsor
- University Hospital Birmingham
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Fabry Disease, Heart Diseases
Effect of Migalastat on Cardiac Involvement in Fabry Disease
Anderson-Fabry Disease (AFD) is one of the rare lysosomal storage disorders for which a cause - specific therapy is available. Recently, a new specific drug has been marketed, namely Migalastat, a small-molecule pharmacological…
- Trial name
- MAIORA (NCT03838237)
- Testing
- Cardiological evaluation
- Where
- Italy
- Volunteers
- 18 people
- Sponsor
- Ospedale San Donato
Cardiovascular Diseases, Heart Diseases
Epidemiology of Idiopathic Dilated Cardiomyopathy (Washington, DC Dilated Cardiomyopathy Study)
To identify risk factors for idiopathic dilated cardiomyopathy and to examine prognostic factors over a follow-up period of two to three years.
- Conditions
- Cardiovascular Diseases, Heart Diseases, Myocardial Diseases, Asthma
- Trial name
- NCT00005262
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Metabolic Disease, Obesity
Characterization of Patients With Uncommon Presentations and/or Uncommon Diseases Associated With the Cardiovascular System
Background: Researchers are interested in studying individuals who have known or suspected metabolic, inflammatory or genetic diseases that may put them at a high risk for heart diseases or diseases of their blood vessels.
- Conditions
- Metabolic Disease, Obesity, Li-Fraumeni Syndrome, Cardiomyopathy
- Trial name
- NCT01143454
- Testing
- NSR IDE-MRI: Research pulse sequences
- Where
- 3 sites in United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Carcinomas, Amyloidosis
B-Receptor Signaling in Cardiomyopathy
We hope to determine the importance of different genes (including B receptors) in anthracycline-induced cardiomyopathy. This has important benefits to patients exposed to anthracyclines, as this could help determine whether…
- Conditions
- Carcinomas, Amyloidosis, Anal Cancer, Anemia
- Trial name
- NCT01135849
- Where
- United States
- Volunteers
- 99 people
- Sponsor
- Daniel Bernstein
Cardiomyopathies, Amyloidosis Cardiac
Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation
The study aims to identify new diagnostic and prognostic markers for CMP that can help predict disease progression. In particular, the study will focus on microRNAs (miRNAs) and spatial transcriptomics, which are emerging…
- Conditions
- Cardiomyopathies, Amyloidosis Cardiac, Fabry Disease, Arrhythmogenic Cardiomyopathy
- Trial name
- CMPMIRNA (NCT06813443)
- Where
- 3 sites in Italy
- Volunteers
- 700 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Rare Diseases, Amyloidosis
Institutional Registry of Rare Diseases
The goal of this observational study is to create a single macro registry system with data collection on common clinical features, grouping the different rare diseases (RD).
- Conditions
- Rare Diseases, Amyloidosis, Sarcoidosis, Phacomatosis
- Trial name
- NCT06573723
- Where
- Argentina
- Volunteers
- 380 people
- Sponsor
- Hospital Italiano de Buenos Aires
Fabry's Disease
Impact of Enzyme Replacement Therapy on Cardiac Function in Patients With Fabry's Cardiomyopathy (RECAFTURE Trial)
The purpose of this study is to evaluate the impact of ERT on LV diastolic function and flow in patients with Fabry's cardiomyopathy using diastolic stress echocardiography, LV vortex flow and CMR.
- Trial name
- NCT02469181
- Where
- South Korea
- Volunteers
- 20 people
- Sponsor
- Yonsei University
Amyloidosis, Amyloid
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
- Conditions
- Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy
- Trial name
- MED-hATTR (NCT03431896)
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- The Cleveland Clinic
Danon Disease
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B)…
- Trial name
- NCT06092034
- Testing
- RP-A501
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Danon Disease, Fabry Disease
Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
What is this study about? This research is focused on improving the care for people with rare heart muscle diseases, known as rare cardiomyopathies.
- Conditions
- Danon Disease, Fabry Disease, Cardiac Amyloidosis, Noonan Syndrome
- Trial name
- NCT07336394
- Where
- China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Chinese Academy of Medical Sciences, Fuwai Hospital
Chronic Heart Failure
Metabolic Manipulation in Chronic Heart Failure
Conventional measures used for the treatment of chronic heart failure act predominantly by reducing the work performed by the heart.
- Trial name
- NCT00841139
- Testing
- Perhexiline
- Where
- 2 sites in United Kingdom
- Volunteers
- 50 people
- Sponsor
- University Hospital Birmingham NHS Foundation Trust
Eye Diseases Hereditary, Retinal Disease
Inherited Retinal Degenerative Disease Registry
The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation.
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome
- Trial name
- MRTR (NCT02435940)
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Foundation Fighting Blindness
Barth Syndrome
Exercise Training in Barth Syndrome
Barth syndrome (BTHS) is a genetic disease that results in heart failure, muscle weakness and exercise intolerance. Several studies in non-BTHS heart failure suggest that endurance exercise training is beneficial in improving…
- Trial name
- NCT01194141
- Testing
- Exercise training
- Volunteers
- 4 people
- Sponsor
- Washington University School of Medicine
Mitochondrial Diseases, Kearns-Sayre Syndrome
Global Registry and Natural History Study for Mitochondrial Disorders
The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome…
- Conditions
- Mitochondrial Diseases, Kearns-Sayre Syndrome, MIDD, SANDO
- Trial name
- GENOMIT (NCT05554835)
- Where
- 34 sites in Austria, Germany and 1 more
- Volunteers
- 6,000 people
- Sponsor
- LMU Klinikum
Danon Disease
Danon Disease Natural History Study
The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patients (8 to 50 years of age).
- Trial name
- NCT06214507
- Testing
- No intervention
- Where
- 16 sites in United States, Australia and 5 more
- Volunteers
- 60 people
- Sponsor
- Rocket Pharmaceuticals Inc.