Search clinical trials for: congenital amegakaryocytic thrombocytopenia

Total 10151 results

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

Recruiting

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

Congenital Amegakaryocytic Thrombocytopenia | Paroxysmal Nocturnal Hemoglobinuria | Shwachman-Diamond Syndrome | Bone Marrow Failure Syndrome | Congenital Pure Red Cell Aplasia | Hereditary Sideroblastic Anemia | Hematologic Neoplasm With Germline GATA2 Mutation | Hematologic Neoplasm With Germline... and other conditions

United States
Paul Szabolcs

Recruiting

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC (PRO-RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias

United States
Mitchell Cairo

Recruiting

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Hodgkin Lymphoma | Sickle Cell Disease | Acute Leukemia | Beta-Thalassemia | Non-hodgkin Lymphoma | Severe Aplastic Anemia | Diamond Blackfan Anemia | Amegakaryocytic Thrombocytopenia | Kostmann

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
Paul Szabolcs

Recruiting

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT (HSCT+RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias | Systemic Juvenile Idiopathic Arthritis (sJIA)

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
New York Medical College

Completed

Human Placental-Derived Stem Cell Transplantation (HPDSC)

Mucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditions

United States
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
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
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
Children's Hospital Los Angeles

Completed

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Granuloma | Anemia | Thrombocytopenia | Neutropenia | Sickle Cell Disease | Thalassemia | Niemann-Pick Disease | Wiskott-Aldrich Syndrome | Osteopetrosis | Fucosidosis | Chediak Higashi Syndrome | Hurler Disease

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
National Center for Research Resources (NCRR)
University of California, Los Angeles

Completed

Stem Cell Transplantation (SCT) for Genetic Diseases

Thrombocytopenia | Inborn Errors of Metabolism | Metachromatic Leukodystrophy | Fanconi's Anemia | Thalassemia Major | Pure Red-Cell Aplasia

United States
Masonic Cancer Center, University of Minnesota

Not yet recruiting

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Acquired Pure Red Cell Aplasia | Paroxysmal Nocturnal Hemoglobinuria | Severe Aplastic Anemia | Acquired Amegakaryocytic Thrombocytopenia
UK Kidney Association

Recruiting

National Registry of Rare Kidney Diseases (RaDaR)

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom
Mario Negri Institute for Pharmacological Research

Recruiting

Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases (ANTHEM)

Healthy | Autosomal Dominant Polycystic Kidney | Atypical Hemolytic Uremic Syndrome | Membranoproliferative Glomerulonephritis

Italy
Takeda
Shire; Takeda Development Center Americas, Inc.

Recruiting

A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

Thrombotic Thrombocytopenic Purpura (TTP)

China, United States, France, Japan, Poland, Germany, Austria, Spain, Italy, Switzerland, United Kingdom
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
Mayo Clinic

Recruiting

Pre-myeloid Cancer and Bone Marrow Failure Clinic Study

Hematologic Neoplasms | Myeloid Malignancy | Cytopenia | Bone Marrow Failure Syndrome | Clonal Cytopenia of Undetermined Significance | Clonal Hematopoiesis of Indeterminate Potential | Low Risk Myelodysplastic Syndrome | Inherited Bone Marrow Failure Syndrome | Clonal Expansion | Hereditary Neoplastic... and other conditions

United States
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
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
Insel Gruppe AG, University Hospital Bern
Swiss National Science Foundation; Mach Gaensslen Foundation; Baxalta Innovations...

Recruiting

Genotype and Phenotype Correlation in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome) (TTP registry)

Thrombotic Thrombocytopenic Purpura | Congenital Thrombotic Thrombocytopenic Purpura | Familial Thrombotic Thrombocytopenic Purpura | Thrombotic Thrombocytopenic Purpura, Congenital | Upshaw-Schulman Syndrome

United States, Austria, Czechia, Germany, Japan, Norway, Switzerland
Sanguine Biosciences

Completed

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

Depression | Melanoma | Epilepsy | Lymphoma | Multiple Sclerosis | Kidney Cancer | Cervical Cancer | Diabetes Mellitus | Leukemia | Breast Cancer | Chronic Kidney Diseases | Chronic Obstructive Pulmonary Disease | Parkinson's Disease | Systemic Lupus Erythematosus | Multiple Myeloma | Hepatitis B | Insomnia | Colorectal Cancer | HIV/AID... 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
Takeda

Available

Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

Thrombotic Thrombocytopenic Purpura (TTP)
Baxalta now part of Shire

Completed

Prospective Psychometric Evaluation Study of a Patient-reported Outcomes (PRO) Instrument for Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP]

Congenital Thrombotic Thrombocytopenic Purpura

United States, United Kingdom
Medical College of Wisconsin

Completed

Registry for Vascular Anomalies Associated With Coagulopathy (VAC)

Hemangiomas | Multifocal Lymphangioendotheliomatosis With Thrombocytopenia | Cutaneovisceral Angiomatosis With Thrombocytopenia | Vascular Anomaly With Thrombocytopenia

United States
University Hospital, Bordeaux

Completed

Diagnostic of Chronic Thrombocytopenia (DIATROC)

Chronic Idiopathic Thrombocytopenic Purpura | Congenital Thrombocytopenia
Hadassah Medical Organization

Completed

Efficacy of Sialic Acid GNE Related Thrombocytopenia (SA-thrombo)

Thrombocytopenia
Singapore General Hospital
Shanghai Yueyang Integrated Medicine Hospital; Singapore Bao Zhong Tang TCM...

Completed

Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological Disorders

Myelodysplastic Syndrome (MDS) | Myelofibrosis (MF) | Thalassemia Intermedia | Aplastic Anaemia (AA)

Singapore
Children's Mercy Hospital Kansas City

Withdrawn

Single Incision Versus Standard Laparoscopic Splenectomy

Idiopathic Thrombocytopenic Purpura | Hereditary Spherocytosis

United States
Children's Hospital Medical Center, Cincinnati

Unknown

Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies

Tufted Angioma | Microcystic Lymphatic Malformation | Kaposiform Hemangioendotheliomas | Capillary Venous Lymphatic Malformation | Venous Lymphatic Malformation | Mucocutaneous Lymphangiomatosis and Thrombocytopenia | Capillary Lymphatic Arterial Venous Malformations | PTEN Overgrowth Syndrome With... and other conditions

United States
Children's National Research Institute

Withdrawn

Assessment of Opioid Analgesia in Sickle Cell

Sickle Cell Disease

United States
St. Olavs Hospital
Norwegian University of Science and Technology

Withdrawn

Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura

Purpura, Thrombotic Thrombocytopenic
Office of Rare Diseases (ORD)
Rare Diseases Clinical Research Network

Unknown

Identifying Characteristics of Bone Marrow Failure Syndromes

Myelodysplastic Syndromes | Purpura, Thrombocytopenic | Anemia, Aplastic | Red-Cell Aplasia, Pure | Bone Marrow Failure Syndromes | Hemoglobinuria, Paroxysmal | Leukemia, Lymphocytic

United States
Encysive Pharmaceuticals
GlaxoSmithKline

Completed

Evaluation of Argatroban Injection in Pediatric Patients Requiring Anticoagulant Alternatives to Heparin

Thrombosis | Thrombocytopenia

United States
National Center for Research Resources (NCRR)
James Whitcomb Riley Hospital for Children

Completed

Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

Thrombocytopenia | Dyskeratosis Congenita | Fanconi's Anemia | Shwachman Syndrome
University of Sao Paulo

Not yet recruiting

Interventions to Control Hypersensitivity Pain in Teeth With Insisive Molar Hypomineralization (LBIARHMI)

Molar Incisor Hypomineralization
Stanford University
American Heart Association

Not yet recruiting

Efficacy of Targeted Medical Therapy in Angina and Nonobstructive Coronary Arteries (MVP-ANOCA)

Angina Pectoris | Microvascular Angina | Vasospastic Angina | Myocardial Bridge of Coronary Artery

United States
Enyo Pharma

Not yet recruiting

Vonafexor ALPort Syndrome Efficacy & Safety TRIAl-1 (ALPESTRIA-1) (ALPESTRIA-1)

Alport Syndrome

Spain, United States, France, Germany
Simon Annaheim
University Children's Hospital, Zurich

Recruiting

Assessment of an Innovative Air Mattress On Critically Ill Infants (Prepicare)

Skin Abnormalities | Skin; Injury, Superficial | Skin; Ulcer, Decubitus

Switzerland
University of Texas Southwestern Medical Center
National Institute on Disability, Independent Living, and Rehabilitation...

Not yet recruiting

Monitoring System for Cranial Orthoses

Plagiocephaly | Brachycephaly | Deformational Plagiocephaly | Deformational Posterior Plagiocephaly
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Respiratory and Psychological Impact of Elective Surgery of Congenital Lung Malformations (MALFPULM2)

Congenital Lung Malformations
DK Medical Technology (Suzhou) Co., Ltd.

Not yet recruiting

Peripheral Scoring Drug-coated Balloon in the Treatment of Hemodialysis Arteriovenous Fistula Stenosis (SCDB)

Arteriovenous Fistula Stenosis

China
Amryt Pharma
Chiesi Farmaceutici S.p.A.

Not yet recruiting

A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez (FOSteR)

Epidermolysis Bullosa, Junctional | Epidermolysis Bullosa, Dystrophic
Rocket Pharmaceuticals Inc.

Not yet recruiting

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Pyruvate Kinase Deficiency

Spain, United States
Medical University of South Carolina

Not yet recruiting

QL vs LAI for Palatoplasty

Cleft Palate | Opioid Use | Post-operative Pain

United States
Regeneron Pharmaceuticals

Not yet recruiting

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body (FERVENT-1)

Non-transfusion Dependent Beta-thalassemia (NTDT)

Search clinical trials for: congenital amegakaryocytic thrombocytopenia

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