Clinical Trials on Hereditary Periodic Fevers

Total 6636 results

  • National Cancer Institute (NCI)
    Terminated
    Tocilizumab for Patients With Cancer and COVID-19 Disease
    Hematopoietic and Lymphoid Cell Neoplasm | Malignant Solid Neoplasm | Pneumonia | Symptomatic COVID-19 Infection Laboratory-Confirmed | Severe Acute Respiratory Distress Syndrome | Pneumonitis
    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
  • Arch Biopartners Inc.
    Completed
    LSALT Peptide vs. Placebo to Prevent ARDS and Acute Kidney Injury in Patients Infected With SARS-CoV-2 (COVID-19)
    Severe Acute Respiratory Syndrome | Acute Kidney Injury | COVID | Acute Respiratory Distress Syndrome | Sars-CoV2
    United States, Turkey, Canada
  • St. Jude Children's Research Hospital
    Recruiting
    Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
    Mucopolysaccharidosis I | Severe Combined Immunodeficiency | Neuroblastoma | Neoplasm | Malignancy, Hematologic
    United States
  • National Cancer Institute (NCI)
    Completed
    Ixabepilone in Treating Young Patients With Refractory Solid Tumors
    Recurrent Neuroblastoma | Recurrent Osteosarcoma | Recurrent Childhood Rhabdomyosarcoma | Recurrent Adult Soft Tissue Sarcoma | Recurrent Wilms Tumor and Other Childhood Kidney Tumors | Previously Treated Childhood Rhabdomyosarcoma | Recurrent Childhood Soft Tissue Sarcoma | Recurrent Ewing Sarcoma... and other conditions
    United States
  • City of Hope Medical Center
    Recruiting
    Early Detection of Advanced Adenomas and Colorectal Cancer (AACRC)
    Colorectal Neoplasms | Colorectal Cancer | Colorectal Disorders | Colorectal Adenocarcinoma | Colorectal Cancer Stage II | Colorectal Cancer Stage III | Colorectal Cancer Stage IV | Colorectal Polyp | Colorectal Neoplasms Malignant | Colorectal Adenomatous Polyp | Colorectal Cancer Stage I | Colorectal Adenoma... and other conditions
    United States, Italy, China, Spain, Japan
  • Yang I. Pachankis, PhD
    S for Science
    Active, not recruiting
    Psychiatric Orders in Psychoanalytic Treatment of ASD
    PTSD | Anxiety Disorders | OCD | Borderline Personality Disorder | Social Anxiety Disorder | Depression, Anxiety | Depression, Unipolar | ADHD - Combined Type | Grief | Depression Anxiety Disorder | Depression and Suicide | Autism Spectrum Disorder High-Functioning | Avoidant Personality Disorder | Agoraphobia With... and other conditions
    China
  • 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
  • University Hospital, Bordeaux
    Recruiting
    Functional Tests to Resolve Unsolved Rare Diseases. Rares. (RID)
    Intellectual Disability | Cystic Fibrosis | Neurodegeneration With Brain Iron Accumulation (NBIA) | Congenital Heart Defect | Albinism | Rubinstein-Taybi Syndrome | Periventricular Nodular Heterotopia
    France
  • Ashley Hill
    Completed
    Elucidating the Genetic Basis of the Pleuropulmonary Blastoma (PPB) Familial Cancer Syndrome (PPB)
    Sarcoma | Goiter | Pineoblastoma | Wilms Tumor | Medulloepithelioma | Pleuropulmonary Blastoma | Pituitary Tumors | Cystic Nephroma | Sertoli-Leydig Cell Tumor of Ovary | Embryonal Rhabdomyosarcoma of Cervix
    United States
  • University of South Florida
    Boston University; Yale University
    Completed
    Vasculitis Illness Perception (VIP) Study
    Giant Cell Arteritis | Microscopic Polyangiitis | Churg-Strauss Syndrome | Polyarteritis Nodosa | Takayasu's Arteritis | Behcet's Disease | CNS Vasculitis | Henoch-Schoenlein Purpura | Wegener Granulomatosis
    United States
  • University of South Florida
    Boston University; Duke University; Data Management and Coordinating Center (DMCC)
    Completed
    Reproductive Health in Men and Women With Vasculitis
    Giant Cell Arteritis | Vasculitis, Central Nervous System | Wegener's Granulomatosis | Microscopic Polyangiitis | Churg-Strauss Syndrome | Polyarteritis Nodosa | Takayasu's Arteritis | Kawasaki Disease | Behcet's Disease | Henoch-schoenlein Purpura | Drug-induced Necrotizing Vasculitis
    United States
  • Baylor College of Medicine
    The Methodist Hospital Research Institute; Center for Cell and Gene Therapy...
    Recruiting
    Most Closely Matched 3rd Party Rapidly Generated LMP, BARF1 And EBNA1 Specific CTL, EBV-Positive Lymphoma (MABEL) (MABEL)
    Nasopharyngeal Carcinoma | Hodgkin Disease | Non-Hodgkin Lymphoma | Severe Chronic Active Epstein Barr Virus | T/NK-lymphoproliferative Disease | Smooth Muscle Tumor
    United States
  • Children's Hospital Medical Center, Cincinnati
    Memorial Sloan Kettering Cancer Center; Fred Hutchinson Cancer Center
    Recruiting
    HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA)
    Fanconi Anemia | Myelodysplastic Syndrome (MDS) | Acute Myelogenous Leukemia (AML) | Severe Marrow Failure
    United States
  • McGill University Health Centre/Research Institute...
    Recruiting
    Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)
    Peroxisome Biogenesis Disorder | Zellweger Spectrum Disorder | RCDP - Rhizomelic Chondrodysplasia Punctata | D-Bifunctional Protein Deficiency | Alpha-Methylacyl-CoA Racemase Deficiency | Peroxisomal Acyl-CoA Oxidase Deficiency | Peroxisomal Acyl-CoA Oxidase 2 Deficiency | ATP Binding Cassette Subfamily... and other conditions
    Canada
  • University of South Florida
    Boston University; University of Pennsylvania; Data Management and Coordinating...
    Completed
    Educational Needs of Patients With Systemic Vasculitis
    Giant Cell Arteritis | Vasculitis, Central Nervous System | Microscopic Polyangiitis | Churg-Strauss Syndrome | Polyarteritis Nodosa | Takayasu's Arteritis | Behcet's Disease | Henoch-Schoenlein Purpura | Wegener Granulomatosis
    United States
  • Brigham and Women's Hospital
    Agency for Healthcare Research and Quality (AHRQ)
    Completed
    Improving Safety of Diagnosis and Therapy in the Inpatient Setting (PSLL2-0)
    Pneumonia | Sepsis | Delirium | Confusion | Hip Pain Chronic | Hypoxia | Back Pain | Vomiting | Abdominal Pain | Diarrhea | Cough | Asthma | Chest Pain | Neck Pain | Fever | Cellulitis | Syncope | Headache | Deep Vein Thrombosis | Venous Thromboembolism | Soft Tissue Infections | Failure to Thrive | COPD | Pulmonary Embolism | Dyspnea | Leg Pain | Mental... and other conditions
    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
  • Region Skane
    Not yet recruiting
    Checklists Resuscitation Emergency Department
    Pneumonia | Burns | Shock | Poisoning | Abdominal Pain | Chest Pain | Trauma | Fever | Bradycardia | Syncope | Seizures | Vertigo | Tachycardia | Cardiac Arrest | Diabetic Ketoacidosis | Shortness of Breath | Allergic Reaction | Asthma Acute | Altered Mental Status | COPD Exacerbation Acute | Pulmonary Edema - Acute | Hyperglycemic Hyperosmolar...
  • Joanne Kurtzberg, MD
    The Marcus Foundation
    Recruiting
    UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (DUOC-01)
    Mucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo Mucopolysaccharidoses
    United States
  • University of Pennsylvania
    Completed
    Journey of Patients With Vasculitis From First Symptom to Diagnosis
    Vasculitis | Giant Cell Arteritis | Microscopic Polyangiitis | Polyarteritis Nodosa | Systemic Vasculitis | Granulomatosis With Polyangiitis | Takayasu Arteritis | Cryoglobulinemic Vasculitis | Behcet's Disease | CNS Vasculitis | Temporal Arteritis | IgA Vasculitis | Urticarial Vasculitis | Wegener Granulomatosis | Eosinophilic... and other conditions
    United States
  • National Human Genome Research Institute (NHGRI)
    Recruiting
    A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
    Metabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditions
    United States
  • St. Jude Children's Research Hospital
    Recruiting
    B7-H3-Specific Chimeric Antigen Receptor Autologous T-Cell Therapy for Pediatric Patients With Solid Tumors (3CAR)
    Melanoma | Soft Tissue Sarcoma | Carcinoma | Osteosarcoma | Ewing Sarcoma | Rhabdoid Tumor | Neuroblastoma | Rhabdomyosarcoma | Wilms Tumor | Hepatoblastoma | Desmoplastic Small Round Cell Tumor | Pediatric Solid Tumor | Clear Cell Sarcoma | Malignant Peripheral Nerve Sheath Tumors | Adrenocortical Cancer | Germ Cell Cancer
    United States
  • Giselle Sholler
    Translational Genomics Research Institute; Dell, Inc.
    Completed
    Molecular-Guided Therapy for Childhood Cancer
    Glioma | Renal Cell Carcinoma | Osteosarcoma | Ependymoma | Medulloblastoma | Neuroblastoma | Meningioma | Craniopharyngioma | Choroid Plexus Neoplasms | Rhabdomyosarcoma | Wilms Tumor | Ewings Sarcoma | Malignant Rhabdoid Tumor | Liver Tumors | Germ Cell Tumors | Dysembryoplastic Neuroepithelial Tumor | Clear Cell Sarcoma | ... and other conditions
    United States, Lebanon
  • Masonic Cancer Center, University of Minnesota
    Completed
    Stem Cell Transplant for Inborn Errors of Metabolism
    Niemann-Pick Disease | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | GM1 Gangliosidosis | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Tay Sachs Disease | Sandhoff Disease
    United States
  • Ann & Robert H Lurie Children's Hospital of Chicago
    National Cancer Institute (NCI); Northwestern University
    Completed
    Intensity-Modulated Radiation Therapy in Treating Younger Patients With Lung Metastases
    Unspecified Childhood Solid Tumor, Protocol Specific | Unspecified Adult Solid Tumor, Protocol Specific | Lung Metastases | Recurrent Childhood Rhabdomyosarcoma | Recurrent Adult Soft Tissue Sarcoma | Stage IV Adult Soft Tissue Sarcoma | Recurrent Wilms Tumor and Other Childhood Kidney Tumors | Previously... and other conditions
    United States
  • St. Jude Children's Research Hospital
    Pfizer; Ipsen
    Recruiting
    Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma
    Refractory Malignant Solid Neoplasm | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Solid Neoplasm | Recurrent Neuroblastoma | Recurrent Osteosarcoma | Recurrent Peripheral Primitive Neuroectodermal Tumor | Recurrent Rhabdoid Tumor | Recurrent... and other conditions
    United States, Canada
  • University of Pennsylvania
    University of South Florida; Duke University
    Recruiting
    Vasculitis Pregnancy Registry (V-PREG)
    Vasculitis | Wegener's Granulomatosis | Systemic Vasculitis | Eosinophilic Granulomatosis With Polyangiitis (EGPA) | Churg-Strauss Syndrome (CSS) | Cryoglobulinemic Vasculitis | Behcet's Disease | CNS Vasculitis | IgA Vasculitis | Granulomatosis With Polyangiitis (GPA) | Microscopic Polyangiitis (MPA) | Polyarteritis... and other conditions
    United States
  • Masonic Cancer Center, University of Minnesota
    Terminated
    Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
    Sphingolipidoses | Niemann-Pick Disease, Type C | Krabbe Disease | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Hurler's Syndrome | Maroteaux-Lamy Syndrome | Wolman's Disease | Niemann-Pick Disease Type B
    United States
  • Children's Oncology Group
    National Cancer Institute (NCI)
    Recruiting
    Study of Kidney Tumors in Younger Patients
    Renal Cell Carcinoma | Wilms Tumor | Clear Cell Sarcoma of the Kidney | Papillary Renal Cell Carcinoma | Kidney Medullary Carcinoma | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Congenital Mesoblastic Nephroma | Renal Cell Carcinoma Associated With Xp11.2 Translocations... and other conditions
    United States, Canada, Puerto Rico, Australia, New Zealand, Israel, Switzerland
  • Assistance Publique - Hôpitaux de Paris
    Iltoo Pharma
    Completed
    Induction of Regulatory t Cells by Low Dose il2 in Autoimmune and Inflammatory Diseases (TRANSREG)
    Rheumatoid Arthritis | Systemic Lupus Erythematosus | Psoriasis | Systemic Sclerosis | Crohn's Disease | Ulcerative Colitis | Idiopathic Thrombocytopenic Purpura | Autoimmune Hepatitis | Wegener's Granulomatosis | Ankylosing Spondylitis | Behcet's Disease | Takayasu's Disease | Sclerosing Cholangitis | Gougerot-sj...
    France
  • Anagnostou, Evdokia, M.D.
    The Hospital for Sick Children; Canadian Institutes of Health Research (CIHR); Alberta Health services and other collaborators
    Not yet recruiting
    Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders (CALM)
    Anxiety Disorders | Generalized Anxiety Disorder | Anxiety | Tuberous Sclerosis | ADHD | Neurodevelopmental Disorders | Autism Spectrum Disorder | Fragile X Syndrome | Social Anxiety | Social Anxiety Disorder | Agoraphobia | Tourette Syndrome | Tic Disorders | Autism | ADHD Predominantly Inattentive Type | ADHD - Combined... and other conditions
    Canada
  • Dana-Farber Cancer Institute
    Recruiting
    InAdvance: Surveillance, Prevention, and Interception in a Population at Risk for Cancer
    Lung Cancer | Cirrhosis | Barrett Esophagus | Hematologic Malignancy | Non-Alcoholic Fatty Liver Disease | Cancer Risk | Vulvar Intraepithelial Neoplasia | Childhood Cancer Survivors | Lung; Node | Non Alcoholic Steatohepatitis | Osteochondroma | Cancer Predisposition Syndrome | Hereditary Cancer Prediction | Adult... and other conditions
    United States
  • Indiana University
    Recruiting
    Intervention to Change Affect Recognition and Empathy (ICARE)
    Traumatic Brain Injury | Concussion, Severe | Concussion, Intermediate | Concussion With Brief Loss of Consciousness | Traumatic Brain Injury With Brief Loss of Consciousness | Traumatic Brain Injury With No Loss of Consciousness | Concussion With LOC 31 to 59 Minutes | Traumatic Brain Injury With... and other conditions
    United States
  • Anova Enterprises, Inc
    Recruiting
    AnovaOS Network Powered Patient Registry
    Neoplasms | Infectious Disease | External Causes of Morbidity and Mortality | Pregnancy, Childbirth and the Puerperium | Diseases of the Blood and Blood-Forming Organs and Certain Disorders Involving the Immune Mechanism (D50-D89) | Endocrine, Nutritional and Metabolic Diseases (E00-E89) | Mental... and other conditions
    United States
  • West Virginia University
    Completed
    Unrelated Umbilical Cord Blood (UBC)Transplantation
    Multiple Myeloma | Hodgkin Lymphoma | Non-Hodgkin Lymphoma | Myelodysplastic Syndrome | Chronic Lymphocytic Leukemia (CLL) | Severe Aplastic Anemia | Acute Myelogenous Leukemia (AML) | Acute Lymphocytic Leukemia (ALL) | Chronic Myelogenous Leukemia (CML)
    United States
  • University of Nebraska
    Recruiting
    Integrated Cancer Repository for Cancer Research (iCaRe2)
    Melanoma | Sarcoma | Kidney Cancer | Leukemia | Breast Cancer | Laryngeal Cancer | Hypopharyngeal Cancer | Nasopharyngeal Cancer | Multiple Myeloma | Gastric Cancer | Pancreatic Cancer | Esophageal Cancer | Small Intestine Cancer | Ovarian Cancer | Lynch Syndrome | Lung Cancer | Rectal Cancer | Prostate Cancer | Gastrointestinal... and other conditions
    United States
  • RTI International
    University of North Carolina, Chapel Hill
    Enrolling by invitation
    Parent-Infant Inter(X)Action Intervention (PIXI)
    Tuberous Sclerosis | Down Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Rett Syndrome | Turner Syndrome | Williams Syndrome | Angelman Syndrome | Chromosome 22q11.2 Deletion Syndrome | Klinefelter Syndrome | Phelan-McDermid Syndrome | Dup15Q Syndrome | Smith Magenis Syndrome
    United States
  • Children's Hospital of Philadelphia
    Eli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaborators
    Recruiting
    The Myelin Disorders Biorepository Project (MDBP)
    Mucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditions
    United States
  • Yale University
    Rheumatology Research Foundation
    Recruiting
    Rheumatology Patient Registry and Biorepository
    Rheumatic Diseases | Rheumatoid Arthritis | Systemic Lupus Erythematosus | Systemic Sclerosis | Dermatomyositis | Polymyositis | Giant Cell Arteritis | Polymyalgia Rheumatica | Psoriatic Arthritis | Sjogren's Syndrome | Antiphospholipid Syndrome | Sarcoidosis | Lyme Disease | Ankylosing Spondylitis | Scleroderma | Reactive... and other conditions
    United States
  • Albert Einstein College of Medicine
    Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators
    Enrolling by invitation
    ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (ScreenPlus)
    Mucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditions
    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
  • Fairview University Medical Center
    Unknown
    Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
    Mucopolysaccharidosis I | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Pulmonary Complications | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Mannosidosis | I Cell Disease
    United States
  • Children's Hospital of Philadelphia
    Illumina, Inc.
    Active, not recruiting
    LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
    Mucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditions
    United States
  • University of Pittsburgh
    Recruiting
    Longitudinal Study of Neurodegenerative Disorders
    Lysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditions
    United States
  • Scripps Health
    Completed
    Unrelated Donor Stem Cell Transplantation
    Myelodysplastic Syndromes | Multiple Myeloma | Hodgkin's Lymphoma | Non-Hodgkin's Lymphoma | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Acute Myelogenous Leukemia | Chronic Myelogenous Leukemia | Small Lymphocytic Lymphoma | Paroxysmal Nocturnal Hemoglobinuria | Severe Aplastic Anemia | Myeloproliferative... 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
  • Talaris Therapeutics Inc.
    Duke University
    Terminated
    Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
    Niemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...
    United States
  • Children's Hospitals and Clinics of Minnesota
    M.D. Anderson Cancer Center; Washington University School of Medicine; Dana-Farber... and other collaborators
    Recruiting
    International PPB/DICER1 Registry
    Neuroblastoma | Thyroid Carcinoma | Pineoblastoma | Wilms Tumor | Embryonal Rhabdomyosarcoma | Ovarian Sarcoma | Sertoli-Leydig Cell Tumor | Pleuropulmonary Blastoma | Gynandroblastoma | DICER1 Syndrome | Cystic Nephroma | Renal Sarcoma | Nodular Hyperplasia of Thyroid | Nasal Chondromesenchymal Hamartoma | Ciliary Body... and other conditions
    United States
  • Cure CMD
    Recruiting
    Congenital Muscle Disease Study of Patient and Family Reported Medical Information (CMDPROS)
    Emery-Dreifuss Muscular Dystrophy | Congenital Myasthenic Syndrome | Limb-Girdle Muscular Dystrophy | Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency | Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy) and other conditions
    United States

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Portugal

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe