Search clinical trials for: severe congenital neutropenia

Total 17902 results

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 Institute of Allergy and Infectious Diseases...

Recruiting

Empagliflozin as a Treatment for Severe Congenital Neutropenia Due to G6PC3 Deficiency

Crohn's Disease | Glycogen Metabolism | Inflammatory Bowel Disease (IBD)

United States
Federal Research Institute of Pediatric Hematology...

Not yet recruiting

Total Lymphoid Irradiation Pre-HSCT in Severe Congenital Neutropenia

Severe Congenital Neutropenia | GATA2 Deficiency

Russian Federation
St. Jude Children's Research Hospital
Baylor College of Medicine; Children's Hospital of Philadelphia; Dana-Farber... and other collaborators

Recruiting

Investigation of the Genetics of Hematologic Diseases

Myelodysplastic Syndromes | Sickle Cell Disease | Fanconi Anemia | Severe Congenital Neutropenia | Bone Marrow Failure Syndromes | Blood Coagulation Disorder | Erythrocyte Disorder | Leukocyte Disorder | Hemostasis | Dyskeratosis Congenita | Diamond-Blackfan Anemia | Congenital Thrombocytopenia | Myeloproliferative...

United States
Masonic Cancer Center, University of Minnesota

Recruiting

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Sickle Cell Disease | Severe Congenital Neutropenia | Paroxysmal Nocturnal Hemoglobinuria | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Diamond Blackfan Anemia | Transfusion Dependent Alpha- or Beta- Thalassemia | Non-Malignant Hematologic Disorders

United States
Diane George

Recruiting

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

Acute Lymphoblastic Leukemia (ALL) | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML) | Chronic Myeloid Leukemia (CML) | Juvenile Myelomonocytic Leukemia (JMML) | Lymphoma (Hodgkin's and Non-Hodgkin's)

United States
Roswell Park Cancer Institute

Active, not recruiting

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

Acute Myeloid Leukemia | Polycythemia Vera | Myelofibrosis | Chronic Myelomonocytic Leukemia | Waldenstrom Macroglobulinemia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Secondary Acute Myeloid Leukemia | Sickle Cell Disease | Myelodysplastic Syndrome | Plasma Cell Myeloma | Chronic Granulomatous... and other conditions

United States
University of Florida
Live Like Bella Pediatric Cancer Research

Completed

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

Common Variable Immunodeficiency | Severe Combined Immunodeficiency | Sickle Cell Disease | Hurler Syndrome | Chronic Granulomatous Disease | Thalassemia | Wiskott-Aldrich Syndrome | Adrenoleukodystrophy | X-linked Lymphoproliferative Disease | Diamond Blackfan Anemia | Bone Marrow Failure Syndrome | Hemophagocytic... and other conditions

United States
Aalborg University Hospital
Aalborg University

Enrolling by invitation

Comparison of Histamine and Local Heating for Evoking the Axon-reflex Flare Response in Diabetes (HistaHeat)

Diabetes | Diabetes Mellitus, Type 1 | Diabetic Neuropathy Peripheral | Small Fiber Neuropathy

Denmark
Diane George

Active, not recruiting

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Sickle Cell Disease | Hemoglobinopathies | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Macrophage Activation Syndrome | Bone Marrow Failure Syndrome | Amegakaryocytic Thrombocytopenia | Schwachman Diamond Syndrome | Primary Immunodeficiency Syndromes | Acquired Immunodeficiency... and other conditions

United States
Masonic Cancer Center, University of Minnesota
National Marrow Donor Program

Completed

Stem Cell Transplant for Hemoglobinopathy

Sickle Cell Disease | Thalassemia | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Shwachman-Diamond Syndrome

United States
Emory University

Completed

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

Sickle Cell Disease | Fanconi Anemia | Hurler Syndrome | Chronic Granulomatous Disease | Severe Congenital Neutropenia | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Thalassemia Major | Leukocyte... and other conditions

United States
Roswell Park Cancer Institute

Completed

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Primary Myelofibrosis | Polycythemia Vera | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditions

United States
University of California, San Francisco
National Cancer Institute (NCI)

Completed

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

Myelodysplastic Syndromes | Leukemia | Congenital Amegakaryocytic Thrombocytopenia | Severe Congenital Neutropenia | Diamond-blackfan Anemia

United States
Emory University
Children's Healthcare of Atlanta

Terminated

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

Sickle Cell Disease | Fanconi Anemia | Thalassemia | Severe Congenital Neutropenia | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Leukocyte Adhesion Deficiency | Dyskeratosis-congenita | Chediak-Higashi Syndrome | Chronic-granulomatous Disease | Schwachman-Diamond...

United States
University of California, San Francisco
National Cancer Institute (NCI)

Completed

Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission

Leukemia | Thrombocytopenia | Fanconi Anemia | Congenital Amegakaryocytic Thrombocytopenia | Severe Congenital Neutropenia | Diamond-blackfan Anemia

United States
University of California, San Francisco
National Cancer Institute (NCI)

Completed

Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease

Myelodysplastic Syndromes | Leukemia | Congenital Amegakaryocytic Thrombocytopenia | Severe Congenital Neutropenia

United States
Regeneron Pharmaceuticals
Intellia Therapeutics

Not yet recruiting

A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B (BEYOND-9)

Hemophilia B
Sensorion

Not yet recruiting

A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations (AUDIOGENE)

Otorhinolaryngologic Diseases | Ear Diseases | Hearing Disorders | Deafness | Hearing Loss, Sensorineural | Congenital Deafness | DFNB9 | OTOF Gene Mutation

France
Leuko Labs, Inc.

Not yet recruiting

Pivotal Study to Validate a Novel System to Non-Invasively Detect Severe Neutropenia (PC002)

Multiple Myeloma | Diffuse Large B Cell Lymphoma
Bioray Laboratories
First Affiliated Hospital of Guangxi Medical University

Not yet recruiting

Clinical Study of BRL-101 in Severe SCD

Sickle Cell Disease

China
Alireza Shamshirsaz

Not yet recruiting

A Pilot Study on the Role of Fetal Endotracheal Occlusion (FETO) in Fetuses With Severe Congenital Diaphragmatic Hernia

Congenital Diaphragmatic Hernia

United States
Erasmus Medical Center

Not yet recruiting

Learning by Heart: The Effectiveness of an EF Training Program for Pre-schoolers With a Severe CHD (LbH)

Congenital Heart Disease | Executive Dysfunction | Attention Disorder

Netherlands
Jiangsu HengRui Medicine Co., Ltd.

Not yet recruiting

A Trial of HHPG-19K Injection and Auto-HHPG-19K Injection in Prevention of Chemotherapy-related Moderate to Severe Neutropenia of Patients With Nonmyeloid Malignancies

Patients With Nonmyeloid Malignancies Receiving Antineoplastic Therapy Based on Chemotherapy Regimens at Moderate to High Febrile Neutropenia (FN) Risk

China
Guangdong Provincial People's Hospital

Recruiting

Efficacy of Triple-Combination Therapy in Severe PAH-CHD

Pulmonary Arterial Hypertension | Congenital Heart Disease

China
University of Wisconsin, Madison

Not yet recruiting

Fetoscopic Endoluminal Tracheal Occlusion for Severe Left and Right Congenital Diaphragmatic Hernia

Congenital Diaphragmatic Hernia

United States
National Cancer Center, Korea
Seoul National University Hospital

Not yet recruiting

Tripegfilgrastim Trial to Reduce the Risk of Severe Neutropenia in Patients With Unresectable Pancreaticobiliary Cancers (Dulastin)

Unresectable Pancreatic Cancer | Unresectable Biliary Tract Carcinoma | Unresectable Bile Duct Carcinoma
University Hospital, Lille

Not yet recruiting

Severe Congenital Hemostatic Defects, Cerebral MIcrobleeds and COGnition (HEMICOG)

Cerebral Microbleeds, Congenital Haemophilia, Congenital Von Willebrand Disease
Stanford University
Boston Children's Hospital; Duke University

Not yet recruiting

Multicenter Trial of ECMO in Children With Severe Cardiac Failure Using the Cardiohelp System

Heart Failure | Cardiomyopathies | Congenital Heart Disease | Cardiogenic Shock
Boston Children's Hospital
Children's Hospital Medical Center, Cincinnati

Recruiting

Shwachman Diamond Syndrome Registry and Study (SDSR)

Shwachman-Diamond Syndrome | Shwachman-Diamond Syndrome-Like

United States
X4 Pharmaceuticals

Not yet recruiting

A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

Neutropenia
Vertex Pharmaceuticals Incorporated

Not yet recruiting

Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/βC Genotype

Sickle Cell Disease
Assiut University

Not yet recruiting

Outcome Of Using Transfixing K Wire ln DDH With Hypoplastic Head And Severe Generalised Ligamentous Laxity

Developmental Dysplasia of the Hip
JenaValve Technology, Inc.

Not yet recruiting

TAVIS Registry - Trilogy Heart Valve System for Management of Patients With Aortic Valve Disease (TAVIS)

Aortic Valve Stenosis | Aortic Valve Disease | Heart Valve Diseases | Ventricular Outflow Obstruction | Constriction, Pathologic | Aortic Valve Stenosis With Insufficiency | Cardiovascular Diseases in Old Age | Aortic Stenosis, Severe | Aortic Valve Regurgitation

Germany
University Medical Center Groningen
Radboud University Medical Center

Recruiting

A GnRH Agonist IN Pre-menopausal Women STudy to Treat Severe Polycystic Liver Disease (AGAINST-PLD)

Autosomal Dominant Polycystic Kidney | Polycystic Liver Disease

Netherlands
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Hematologic Diseases | Genetic Diseases, Inborn | Sickle Cell Disease | Hemoglobinopathies | Sickle Cell Anemia | Thalassemia | Beta-Thalassemia

United States, Italy, Germany
Yair Blumenfeld

Recruiting

Fetal Endoscopic Tracheal Occlusion (FETO) for Severe Congenital Diaphragmatic Hernia (FETO)

Congenital Diaphragmatic Hernia

United States
Beth Israel Deaconess Medical Center

Recruiting

Brain Health With Inner Engineering Meditation (BLISS)

Meditation | Brain; Agenesis

United States
Beam Therapeutics Inc.

Recruiting

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease (BEACON)

Sickle Cell Disease

United States
Aimen F. Shaaban, MD

Recruiting

Trial of FETO for Severe Congenital Diaphragmatic Hernia (FETO)

Congenital Diaphragmatic Hernia

United States
Rigshospitalet, Denmark

Recruiting

BILACO Trial: Biliary Atresia - a Severe Complex Congenital Liver Disease (BILACO)

Cognitive Impairment | Biliary Atresia

Denmark
King's College Hospital NHS Trust
King's College London; University College London Hospitals; University of Sheffield and other collaborators

Recruiting

RElated Haplo-DonoR Haematopoietic stEm Cell Transplantation for Adults With Severe Sickle Cell Disease (REDRESS)

Sickle Cell Disease

United Kingdom
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Sickle Cell Disease | Hemoglobinopathies | Hematological Diseases | Hydroxyurea Failure | Hydroxyurea Intolerance

United States, Italy, United Kingdom, Germany
Baylor College of Medicine
The Methodist Hospital Research Institute; Center for Cell and Gene Therapy...

Recruiting

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion (HAPLOTAB)

Myelodysplastic Syndromes | Primary Immunodeficiency Diseases | Hemoglobinopathies | Chronic Myeloid Leukemia | Cytopenia | Severe Aplastic Anemia | Bone Marrow Failure Syndrome | Acute Myeloid Leukemia in Remission | Hemophagocytic Lymphohistiocytoses | Acute Lymphoblastic Leukemia in Remission | Severe...

United States
University Children's Hospital, Zurich

Recruiting

EF Intervention in Children With Severe Congenital Heart Disease (E-FIT)

Heart Defects, Congenital | Executive Dysfunction

Switzerland
Mayo Clinic
Hopital du Sacre-Coeur de Montreal

Recruiting

PB to Treat Hereditary Nephrogenic Diabetes Insipidus, ADPKD Treated With Tolvaptan, and Severely Polyuric Patients With Previous Lithium Administration (SerendipityPB1)

Autosomal Dominant Polycystic Kidney Disease | Nephrogenic Diabetes Insipidus | Acquired Nephrogenic Diabetes Insipidus | Congenital Nephrogenic Diabetes Insipidus

United States
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Severe Acute Respiratory Syndrome CoV 2 COVID-19 Survey and Vaccination Coverage in the Sickle Cell Population in Ile-De-France (COVIDO-DREP)

Sickle Cell Disease
University of Bordeaux

Recruiting

Percutaneous Edge To Edge Repair for the Treatment of Severe Tricuspid Valve Regurgitation in High-surgical Risk Patients With Systemic Right Ventricle : a Multicentric French Cohort (PETER-TRESRIV) (PETER-TRESRIV)

Congenital Heart Disease | Tricuspid Regurgitation | Systemic Right Ventricle

France
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
University Hospital, Lille
Ministry of Health, France

Recruiting

DIStinguishing ChildrEn at Low Risk of Severe infectioN in Case of Febrile Neutropenia-7: Impact Study of a Clinical Decision Rule (DISCERN-FN7)

Neutropenia, Febrile

France

Search clinical trials for: severe congenital neutropenia

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