Clinical Trials on Myasthenic Syndromes, Congenital

Total 40908 results

Memorial Sloan Kettering Cancer Center
Pediatric Brain Tumor Consortium

Terminated

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

Fanconi Anemia | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML)

United States
Natera, Inc.

Completed

Development of Non-invasive Prenatal Screening Test for Microdeletions Based on Fetal DNA Isolated From Maternal Blood (MAPS)

Trisomy 21 | Trisomy 18 | Trisomy 13 | Sex Chromosome Abnormalities | Microdeletion Syndromes

United States, Spain, Taiwan
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Effect of RAS/MAPK Pathway Hyperactivation on Growth' and Bone' Profile of the RASopathies (3776)

Noonan Syndrome | Costello Syndrome | RASopathy | Cardio-Facio-Cutaneous Syndrome

Italy
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Solid Tumors in RASopathies (4218)

Noonan Syndrome | Costello Syndrome | RASopathy | Cardio-Facio-Cutaneous Syndrome

Italy
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Prevalence and Characterization of Pain in RASopathies (3717)

Noonan Syndrome | Costello Syndrome | RASopathy | Cardio-Facio-Cutaneous Syndrome

Italy
Emory University

Completed

Single Ventricle Outcome

Hypoplastic Left Heart Syndrome | Single Ventricle | Heart Disease, Congenital

United States
Guangdong Provincial People's Hospital

Unknown

Study of Hemodynamics of Neonates by Echocardiography and USCOM (HNEU)

Sepsis | Congenital Heart Disease

China
National Cancer Institute (NCI)

Completed

Belinostat in Treating Patients With Myelodysplastic Syndromes

Previously Treated Myelodysplastic Syndromes | Secondary Myelodysplastic Syndromes | de Novo Myelodysplastic Syndromes

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Completed

Azacitidine and Etanercept in Treating Patients With Myelodysplastic Syndromes

Previously Treated Myelodysplastic Syndromes | Secondary Myelodysplastic Syndromes | de Novo Myelodysplastic Syndromes

United States
Carelon Research

Terminated

Use of Oxandrolone to Promote Growth in Infants With HLHS

Congenital Heart Disease | Hypoplastic Left Heart

United States, Canada
Assistance Publique - Hôpitaux de Paris
Ministry of Health, France

Not yet recruiting

Cardiac Resynchronization Therapy in Adult Congenital Heart Disease With Systemic Right Ventricle: RIGHT-CRT (RIGHT-CRT)

Congenital Heart Disease | Transposition of Great Vessels | Congenitally Corrected Transposition of the Great Arteries | Systemic Right Ventricle | Heart Failure Congenital

France
University of Sao Paulo General Hospital
InCor Heart Institute

Recruiting

Pentoxifylline as an Adjunct Therapy for Patients With Eisenmenger Syndrome

Eisenmenger Syndrome

Brazil
University of Rochester
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Long QT Syndrome-Population Genetics and Cardiac Studies (LQTS)

Heart Diseases | Cardiovascular Diseases | Ventricular Arrhythmia | Long QT Syndrome

United States
Baylor College of Medicine

Completed

Cardiac Biomarkers in Patients With Single Ventricle Physiology

Congenital Heart Disease | Cardiac Disease | Hypoplastic Left Heart | Cyanotic Congenital Heart Disease

United States
Progenity, Inc.
Obstetrix Medical Group

Unknown

Non-Invasive Screening for Fetal Aneuploidy

Down Syndrome | Edwards Syndrome

United States
University of Colorado, Denver

Completed

Micro RNAs as a Marker of Aortic Aneurysm in Hereditary Aortopathy Syndromes

Marfan Syndrome | Turner Syndrome | Loeys-Dietz Syndrome | Thoracic Aortic Aneurysm and Dissection Syndromes | Ehlers-Danlos Type IV Syndrome

United States
St. Justine's Hospital

Recruiting

Impact of NAVA Ventilation on Brain Oxygenation and Perfusion in Children With Congenital Heart Disease

Heart Defects, Congenital | Tetralogy of Fallot | Univentricular Heart | Hypoplastic Left Heart Syndrome

Canada
Novartis Pharmaceuticals

Completed

This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.

Myelodysplastic Syndrome | Thalassemia

Russian Federation
Sumitomo Pharma Switzerland GmbH

Recruiting

Congenital Athymia Patient Registry

Complete DiGeorge Anomaly | Complete DiGeorge Syndrome | Congenital Athymia

United States
Association pour le Développement et l'Organisation...

Not yet recruiting

Prevalence of Rest Dyspnea and Impact of Non Invasive Ventilation on Breathing Sensations in CCHS Patients (DyspnOndine)

Dyspnea | Congenital Central Hypoventilation Syndrome | CCHS

France
Academisch Medisch Centrum - Universiteit van Amsterdam...
Netherlands Organisation for Scientific Research

Recruiting

Towards Optimal Treatment of Marfan Syndrome (TOWER)

Marfan Syndrome

Netherlands
University Hospital, Toulouse

Not yet recruiting

Adapted Physical Activity Program (APA) for Effort Rehabilitation of Children and Teenagers With Marfan Syndrome (M&M's)

Marfan Syndrome

France
French Cardiology Society

Completed

Effects of Personalized Training at Home Combining Endurance and Resistance in Patients Suffering From Marfan Syndrome (MARF'HOME)

Marfan Syndrome

France
Assistance Publique - Hôpitaux de Paris
Hospices Civils de Lyon; Banque de cellules cochin

Completed

Clinical Variability in Marfan Syndrome (Variarfan)

Marfan Syndrome

France
University of British Columbia
Heart and Stroke Foundation of Canada

Completed

Losartan Versus Atenolol for the Treatment of Marfan Syndrome

Marfan Syndrome

Canada
National Cancer Institute (NCI)

Recruiting

Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathies

Noonan Syndrome | Legius Syndrome | Cardiofaciocutaneous Syndrome | Costello Syndrome | Capillary Arteriovenous Malformation Syndrome | RASopathy

United States
Radboud University Medical Center

Recruiting

Lymphatic Phenotype in Noonan Syndrome Spectrum Disorders (LENS)

Lymphatic Diseases | Noonan Syndrome | Lymphatic Disease | Lymphatic System Disorders Congenital

Netherlands
Baylor College of Medicine
Southern Star Research Pty Ltd.

Recruiting

Marfan Syndrome Moderate Exercise Trial II

Marfan Syndrome

United States
Nationwide Children's Hospital
National Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health... and other collaborators

Recruiting

Two-Year Study of the Safety and Efficacy of the Second-Generation Tissue Engineered Vascular Grafts (TEVG-2)

Heart Diseases | Cardiovascular Diseases | Heart Defects, Congenital | Cardiovascular Abnormalities | HLH - Hypoplastic Left Heart Syndrome | DORV | DILV - Double Inlet Left Ventricle | Mitral Atresia | Tricuspid Atresia | Unbalanced AV Canal | Single-ventricle

United States
University Hospital, Ghent
Agentschap voor Innovatie door Wetenschap en Technologie

Completed

Randomized, Double-blind Study for the Evaluation of the Effect of Losartan Versus Placebo on Aortic Root Dilatation in Patients With Marfan Syndrome Under Treatment With Beta-blockers

Marfan Syndrome

Belgium
Atılım University

Completed

Investigation of Cardiopulmonary Parameters, Motor Development and Muscle Strength in DS With and Without CHD

Down Syndrome | Gross Motor Development Delay | Congenital Heart Defects

Turkey
Stanford University
Midwestern University

Completed

Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond

Marfan Syndrome

United States
Ann & Robert H Lurie Children's Hospital of Chicago
Johns Hopkins University

Withdrawn

Circulating Transforming Growth Factor Beta (TGF-β) in Individuals With Marfan Syndrome

Marfan Syndrome

United States
University of Oxford
Oxford University Hospitals NHS Trust

Unknown

The Oxford Marfan Trial

Marfan Syndrome

United Kingdom
Brigham and Women's Hospital
Boston Children's Hospital

Completed

Effects of Losartan Versus Atenolol on Aortic and Cardiac Muscle Stiffness in Adults With Marfan Syndrome

Marfan Syndrome

United States
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

Severe Aplastic Anemia | Telomere Biology Disorders | Inherited Bone Marrow Failure Syndromes

United States
Assiut University

Not yet recruiting

Neurological and Psychological Assessment of Neurocutaneous Syndromes in Upper Egypt Children

Neurocutaneous Syndromes
IRCCS Eugenio Medea
University of Trieste

Recruiting

Body Perception and Representation in Overgrowth Syndromes, Behavioral Assessment and Neuropsychological Development (Body-PROBAND)

Body Image | Beckwith-Wiedemann Syndrome | Sotos Syndrome | Body Schema | Overgrowth Syndrome

Italy
St. Jude Children's Research Hospital

Completed

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

Anemia, Aplastic | Diamond-Blackfan Anemia | Amegakaryocytic Thrombocytopenia | Kostmann Syndrome

United States
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
National Eye Institute (NEI)

Terminated

Sunitinib Malate to Treat Advanced Eye Disease in Patients With Von Hippel-Lindau Syndrome (VHL3)

Von Hippel-Lindau Syndrome

United States
National Eye Institute (NEI)

Completed

Ranibizumab Injections to Treat Retinal Tumors in Patients With Von Hippel-Lindau Syndrome

Von Hippel-Lindau Syndrome

United States
Charite University, Berlin, Germany
NeuroCure Clinical Research Center, Charite, Berlin

Completed

The Role of the Thymus in Myasthenia Gravis

Thymoma | Myasthenia Gravis

Germany
University Hospital, Toulouse

Recruiting

Biological Collection for Marfan and Related Syndromes (MARFANS)

Marfan Syndrome

France
Baylor College of Medicine
The Marfan Foundation

Completed

Marfan Syndrome Moderate Exercise Pilot

Marfan Syndrome

United States
University Hospital, Toulouse

Completed

Cardiorespiratory and Muscular Rehabilitation of Children and Young Adults With Marfan Syndrome. (Marfanpower)

Marfan Syndrome

France
Samsung Medical Center

Completed

Comparison of Aliskiren vs Negative Controls on Aortic Stiffness in Patients With MFS

Marfan Syndrome

Korea, Republic of
Assistance Publique - Hôpitaux de Paris

Terminated

Study of the Efficacy of Losartan on Aortic Dilatation in Patients With Marfan Syndrome (MARFANSARTAN)

Marfan Syndrome

France
HealthCore-NERI
National Heart, Lung, and Blood Institute (NHLBI); FDA Office of Orphan Products... and other collaborators

Completed

Comparison of Two Medications Aimed at Slowing Aortic Root Enlargement in Individuals With Marfan Syndrome--Pediatric Heart Network

Marfan Syndrome

United States, Canada, Belgium
University of California, Los Angeles
Actelion

Terminated

Iloprost for the Treatment of Pulmonary Hypertension in Adults With Congenital Heart Disease

Pulmonary Arterial Hypertension | Congenital Heart Disease | Eisenmenger's Syndrome

United States

Clinical Trials on Myasthenic Syndromes, Congenital

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