Clinical Trials on Síndrome de Weber

Total 34110 results

University College, London
Moorfields Eye Hospital NHS Foundation Trust; Targeted Genetics Corporation

Completed

Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

Retinal Degeneration

United Kingdom
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Donor Bone Marrow Transplant With or Without G-CSF in Treating Young Patients With Hematologic Cancer or Other Diseases

Juvenile Myelomonocytic Leukemia | Childhood Acute Myeloid Leukemia in Remission | Childhood Acute Lymphoblastic Leukemia in Remission | Childhood Chronic Myelogenous Leukemia | Childhood Myelodysplastic Syndromes | Chronic Phase Chronic Myelogenous Leukemia | Previously Treated Myelodysplastic... and other conditions

United States
Gesellschaft fur Medizinische Innovation - Hamatologie...

Recruiting

National Acute Promyelocytic Leukemia (APL) Observational Study NAPOLEON-Registry of the German AML Intergroup

Newly-diagnosed APL (de Novo or Therapy-related) | Relapsed APL

Germany
Imperial College London Diabetes Centre

Unknown

Delineation of Novel Monogenic Disorders in the United Arab Emirates Population

Mendelian Disorders | Genetic Disorder | Novel Mutation | Hereditary Disorder | De Novo Mutation | Inherited Disease | Single-Gene Defects

United Arab Emirates
Merck Sharp & Dohme LLC

Terminated

A Dose Exploration Study With Birabresib (MK-8628) in Participants With Selected Hematologic Malignancies (MK-8628-005)

DLBCL | AML Including AML de Novo and AML Secondary to MDS
KU Leuven
Agentschap voor Innovatie door Wetenschap en Technologie

Completed

Risk of QT-prolongation and Torsade de Pointes in Patients Treated With Acute Medication in a University Hospital (RISQ-PATH)

Delirium | QT-prolongation

Belgium
Stanford University

Completed

Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study

Epidermolysis Bullosa Simplex | Epidermolysis Bullosa Simplex Kobner | Weber-Cockayne Syndrome

United States
Universitätsklinikum Hamburg-Eppendorf

Recruiting

Clinical Cohort Study - TRUST (TRUST)

Atrial Fibrillation | Arrhythmias, Cardiac | Ventricular Tachycardia | Atrial Flutter | Supraventricular Tachycardia | Brugada Syndrome | Atrial Tachycardia | Torsades de Pointes | Long QT Syndrome | Arrhythmogenic Right Ventricular Dysplasia | Catecholaminergic Polymorphic Ventricular Tachycardia | Atrial Cardiomyopathy

Germany
Editas Medicine, Inc.

Completed

Natural History Study of CEP290-Related Retinal Degeneration

Eye Diseases | Retinal Degeneration | Eye Diseases, Hereditary | Vision Disorders | Blindness | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

United States, Netherlands, France, Germany
GenSight Biologics

Completed

RESCUE and REVERSE Long-term Follow-up (RESTORE)

Leber Hereditary Optic Neuropathy

United States, France, Italy, United Kingdom, Germany
GenSight Biologics

Active, not recruiting

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year (REFLECT)

Leber Hereditary Optic Neuropathy

Belgium, Spain, United States, France, United Kingdom, Taiwan, Italy
GenSight Biologics

Completed

REALITY LHON Registry (REALITY)

Leber Hereditary Optic Neuropathy

United States, France, Italy, Spain, United Kingdom
Huazhong University of Science and Technology
Shiyan Taihe Hospital

Active, not recruiting

A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy

Leber Hereditary Optic Neuropathy

China
GenSight Biologics

Completed

Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients

Leber Hereditary Optic Neuropathy

France
Santhera Pharmaceuticals
European Vision Institute Clinical Research Network

Completed

Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey

Leber Hereditary Optic Neuropathy (LHON)

Belgium, France, Denmark, Italy, Slovenia
Neurophth Therapeutics Inc

Recruiting

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

Leber Hereditary Optic Neuropathy (LHON)

China, United States
University Hospital, Angers

Unknown

Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy (CICLO-NOHL)

Leber Hereditary Optic Neuropathy

France
dr. Laura C. G. de Graaff-Herder

Recruiting

GROWing Up With Rare GENEtic Syndromes (GROW UR GENES)

Disorders of Sex Development | Congenital Adrenal Hyperplasia | Tuberous Sclerosis | Kallmann Syndrome | Prader-Willi Syndrome | Neurofibromatosis | Rett Syndrome | 22q11 Deletion Syndrome | Turner Syndrome | Noonan Syndrome | Allan-Herndon-Dudley Syndrome | Saethre-Chotzen Syndrome | Congenital Hypopituitarism | Cornelia... and other conditions

Netherlands
Xijing Hospital

Active, not recruiting

Paclitaxel-Coated Balloon Versus Zotarolimus-Eluting Stent for Treatment of De Novo Coronary Artery Lesions (CAGE-FREEIII)

Coronary Artery Disease | Percutaneous Coronary Intervention | De Novo Stenosis

China
Yazdanfar, Iraj, M.D.
RWTH Aachen University

Completed

Pilot Study,Diode Laser 808 nm Supported Direct Pulp Capping in Permanent Teeth (Laser in DPC)

Pain Reduction | Haemostatic | Bacterial De-contamination | Bio-stimulation | Reparative Dentine Formation
University of Wisconsin, Madison
National Cancer Institute (NCI)

Completed

Decitabine and Total-Body Irradiation Followed By Donor Bone Marrow Transplant and Cyclophosphamide in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Recurrent Adult Acute Myeloid Leukemia | Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult... and other conditions

United States
Yuzuncu Yıl University

Completed

Comparative Effects of Paraffin Bath Therapy and ESWT on Quality of Life and Sleep in De Quervain Tenosynovitis

De Quervain Disease

Turkey
Children's Hospital Medical Center, Cincinnati
Lymphangiomatosis and Gorham's Disease Alliance (LGDA); Klippel Trenaunay (KT)... and other collaborators

Recruiting

Identification of Biomarkers for Patients With Vascular Anomalies

Kaposiform Hemangioendothelioma | Kaposiform Lymphangiomatosis | Vascular Anomaly | Klippel Trenaunay Syndrome | Generalized Lymphatic Anomaly | Gorham-Stout Disease | Congenital Lipomatous Overgrowth, Vascular Malformations, and Epidermal Nevi

United States
Novartis Pharmaceuticals

Terminated

MYPROMS-ES02: Safety and Efficacy of Basiliximab, Cyclosporine Microemulsion and Enteric-coated Mycophenolate Sodium (EC-MPS) Versus EC-MPS and Steroid Therapy in Kidney Transplant Recipients Who Are Hepatitis C Positive

De Novo Kidney Transplant
Neurophth Therapeutics Inc

Active, not recruiting

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations

Leber Hereditary Optic Neuropathy (LHON)

United States
Masonic Cancer Center, University of Minnesota

Recruiting

Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia

Myelodysplastic Syndromes | Leukemia | Previously Treated Myelodysplastic Syndrome | Secondary Acute Myeloid Leukemia | Childhood Acute Myeloid Leukemia in Remission | Childhood Acute Lymphoblastic Leukemia in Remission | Recurrent Childhood Acute Myeloid Leukemia | Secondary Myelodysplastic Syndrome and other conditions

United States
Genoss Co., Ltd.

Completed

Compare the Efficacy and Safety of Genoss® DCB and IN.PACT Admiral® DCB in Patients With Peripheral Artery Disease

Peripheral Artery Disease | De Novo Stenosis

Korea, Republic of
Editas Medicine, Inc.

Active, not recruiting

Single Ascending Dose Study in Participants With LCA10

Retinal Degeneration | Eye Diseases, Hereditary | Vision Disorders | Leber Congenital Amaurosis 10 | Inherited Retinal Dystrophies | Retinal Disease | Eye Disorders Congenital

United States
University of Texas Southwestern Medical Center

Recruiting

Morphea in Adults and Children (MAC) Cohort Study: A Morphea Registry and DNA Repository (MAC)

Scleroderma, Localized | Morphea | Frontal Linear Scleroderma en Coup de Sabre | Scleroderma, Circumscribed | Scleroderma, Linear

United States
Sydney South West Area Health Service

Unknown

IVUS Controlled Stenting

Coronary Angioplasty and Stenting | Single de Novo Coronary Artery Stenoses

Australia
Assistance Publique - Hôpitaux de Paris

Completed

Experience of Adolescents With Anorexia Nervosa Towards Antidepressants (E3A)

Adolescents Cared for Anorexia Nervosa at Maison de Solenn

France
Mayo Clinic
National Cancer Institute (NCI)

Completed

Azacitidine and Sonidegib or Decitabine in Treating Patients With Myeloid Malignancies

Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Chronic Myelomonocytic Leukemia | Previously Treated Myelodysplastic Syndrome | Recurrent Adult Acute Myeloid Leukemia | Untreated Adult Acute Myeloid Leukemia | Myelodysplastic Syndrome | Recurrent Childhood Acute Myeloid Leukemia and other conditions

United States
National Cancer Institute (NCI)

Completed

Lenalidomide, Cytarabine, and Idarubicin in Treating Patients With Acute Myeloid Leukemia

Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome | Previously Treated Myelodysplastic Syndrome | Recurrent Adult Acute Myeloid Leukemia | Secondary Acute Myeloid Leukemia | Untreated Adult Acute Myeloid Leukemia | Secondary Myelodysplastic Syndrome | de Novo Myelodysplastic... and other conditions

United States
Korea University Ansan Hospital
B. Braun Korea Co., Ltd.

Recruiting

STabilization of Atheroma By Lipid-reducing Effect of Drug-Coated Balloon (STABLE-DCB) (STABLE-DCB)

Coronary Artery Disease | De Novo Stenosis | Atherosclerotic Plaque

Korea, Republic of
Yale University
Juvenile Diabetes Research Foundation

Terminated

Pathogenesis of the Cardiometabolic Risk in Youth With Type 1 Diabetes

Type 1 Diabetes | De Novo Lipogenesis

United States
Fundacion para la Investigacion Biomedica del Hospital...
Sociedad Española De Medicina Interna

Active, not recruiting

Genotype-phenotype Association in Hereditary Hemorrhagic Telangiectasia (Generate-HHT)

Hereditary Hemorrhagic Telangiectasia | HHT | Rendu Osler Weber Disease

Spain
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Completed

Natural Killer Cells Before and After Donor Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelogenous Leukemia

Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome | Myelodysplastic Syndrome | Recurrent Acute Myeloid Leukemia | Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Therapy-Related Acute Myeloid Leukemia and other conditions

United States
GenSight Biologics

Completed

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation (RESCUE)

Optic, Atrophy, Hereditary, Leber

United Kingdom, United States, France, Germany, Italy
TJ Biopharma Co., Ltd.

Recruiting

A Phase 3 Clinical Study to Evaluate Lemzoparlimab for Injection in Combination With Azacitidine (AZA) Versus AZA Monotherapy in Treatment-naïve Patients With Higher-risk Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndromes(MDS)

China
National Heart, Lung, and Blood Institute (NHLBI)
National Cancer Institute (NCI)

Recruiting

The National Myelodysplastic Syndromes (MDS) Study (MDS)

Myelodysplastic Syndromes (MDS)

United States, Israel
AbbVie
Celgene; Genentech, Inc.

Completed

A Study Evaluating Venetoclax Alone and in Combination With Azacitidine in Participants With Relapsed/Refractory Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes (MDS)

United States, Australia, Germany
AbbVie
Genentech, Inc.

Active, not recruiting

A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes (MDS)

United States, Australia, Canada, France, Germany, Italy, United Kingdom
The First Affiliated Hospital with Nanjing Medical...

Unknown

A Clinical Trial Evaluating the Efficacy of Ultra Low Dose of Decitabine in Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes (MDS)

China
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Completed

Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia

Recurrent Adult Acute Myeloid Leukemia | Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult... and other conditions

United States, Germany
SCRI Development Innovations, LLC
Novartis Pharmaceuticals

Terminated

LBH589 in Refractory Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes (MDS)

United States
PersImmune, Inc
University of California, San Diego

Unknown

Collection of Samples From Patients With MDS

Myelodysplastic Syndromes(MDS)

United States
Xijing Hospital

Recruiting

Intravascular Lithotripsy Versus Conventional Therapy for Severely Calcified Coronary Artery Lesions (REC-CHIPCAC)

De Novo Stenosis | Severely Calcified Coronary Stenoses

China
Fondazione I.R.C.C.S. Istituto Neurologico Carlo...

Completed

A Phase I Study of Immunotherapy With GSC -Loaded Dendritic Cells in Patients With Recurrent Glioblastoma (DENDR-STEM)

de Novo Glioblastoma
Bin Li
Huazhong University of Science and Technology

Completed

Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON) (rAAV2-ND4)

Leber Hereditary Optic Neuropathy

China
National Human Genome Research Institute (NHGRI)

Completed

Screening Behavior in Adults With Hereditary Hemorrhagic Telangiectasia

Telangiectasia, Hereditary Hemorrhagic | Osler-Rendu-Weber Disease | Osler-Rendu Disease

United States

Clinical Trials on Síndrome de Weber

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