Clinical Trials on Hereditary Transthyretin Amyloidosis With Polyneuropthy

Total 230 results

  • 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
  • University Hospital, Ghent
    Centre Hospitalier Universitaire Vaudois; University Ghent; Hopital Lariboisière and other collaborators
    Completed
    PIPAC Nab-pac for Stomach, Pancreas, Breast and Ovarian Cancer (PIPAC-nabpac)
    Breast Cancer Stage IV | Peritoneal Carcinomatosis | Ovarian Cancer Stage IIIC | Ovarian Cancer Stage IV | Breast Cancer Stage IIIB | Breast Cancer Stage IIIc | Ovarian Cancer Stage IIIB | Stomach Cancer Stage III | Stomach Cancer Stage IV With Metastases | Pancreas Cancer, Stage III | Pancreas Cancer, Stage...
    Belgium
  • Roswell Park Cancer Institute
    National Cancer Institute (NCI)
    Completed
    High-Dose Chemotherapy With or Without Total-Body Irradiation Followed by Autologous Stem Cell Transplant in Treating Patients With Hematologic Cancer or Solid Tumors
    Unspecified Childhood Solid Tumor, Protocol Specific | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell Lymphoma | Recurrent Adult Diffuse Mixed Cell Lymphoma and other conditions
    United States
  • National Cancer Institute (NCI)
    Completed
    Sunitinib Malate in Treating HIV-Positive Patients With Cancer Receiving Antiretroviral Therapy
    HIV Infection | Clear Cell Renal Cell Carcinoma | Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell... and other conditions
    United States
  • Boston Children's Hospital
    National Eye Institute (NEI); Howard Hughes Medical Institute
    Recruiting
    Genetic Studies of Strabismus, Congenital Cranial Dysinnervation Disorders (CCDDs), and Their Associated Anomalies
    Ocular Motility Disorders | Facial Palsy | Synkinesis | Sixth Nerve Palsy | Mobius Syndrome | Congenital Fibrosis of Extraocular Muscles | Duane Retraction Syndrome | Duane Radial Ray Syndrome | Brown Syndrome | Marcus Gunn Syndrome | Strabismus Congenital | Horizontal Gaze Palsy | Horizontal Gaze Palsy With Progressive... and other conditions
    United States
  • Fred Hutchinson Cancer Center
    National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...
    Completed
    Fludarabine Phosphate and Total-Body Radiation Followed by Donor Peripheral Blood Stem Cell Transplant and Immunosuppression in Treating Patients With Hematologic Malignancies
    Stage II Multiple Myeloma | Stage III Multiple Myeloma | 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... and other conditions
    United States, Germany, Italy
  • Assistance Publique - Hôpitaux de Paris
    Recruiting
    Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (MATRIX)
    Adrenoleukodystrophy | Adrenomyeloneuropathy | Metachromatic Leukodystrophy | Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
    France
  • Nemours Children's Clinic
    Recruiting
    Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases
    Charcot-Marie-Tooth Disease | Motor Neuron Disease | Amyotrophic Lateral Sclerosis | Muscular Dystrophy | Spinal Muscular Atrophy | Neuromuscular Disease | Spinal Muscular Atrophy With Respiratory Distress 1 | Peroneal Muscular Atrophy
    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
  • 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
  • RenJi Hospital
    Peking University First Hospital; Shanghai Zhongshan Hospital; First Affiliated... and other collaborators
    Recruiting
    Frequency, Clinical Phenotype and Genetic Analysis of Heritable Kidney Cancer Syndromes
    Kidney Cancer | Renal Cell Carcinoma | ALK Gene Mutation | MET Gene Mutation | HLRCC | Renal Tumor Histology | Cutaneous Leiomyoma | BAP1 Tumor Predisposition Syndrome | VHL Syndrome | Birt-Hogg-Dube Syndrome | Familial Renal Cancer | FLCN Gene Mutation | FH Gene Mutation | Cutaneous Leiomyomata With Uterine Leiomyomata
    China
  • Children's Hospital Medical Center, Cincinnati
    Recruiting
    RASopathy Biorepository
    Neurofibromatosis 1 | Noonan Syndrome | Legius Syndrome | Cardiofaciocutaneous Syndrome | Costello Syndrome | SYNGAP1-Related Intellectual Disability | DLG4 | RAS Mutation | Noonan Syndrome With Multiple Lentigines | Noonan Neurofibromatosis Syndrome | Smith-Kingsmore Syndrome | MTOR Gene Mutation | GATOR-1 Gene... and other conditions
    United States
  • University of South Florida
    Boston University; University of Pennsylvania; Wayne State University; University...
    Completed
    VCRC Patient Contact Registry Patient-Reported Data Validation Study
    Giant Cell Arteritis | Microscopic Polyangiitis | Churg-Strauss Syndrome | Polyarteritis Nodosa | Takayasu's Arteritis | Behcet's Disease | Wegener Granulomatosis
    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 South Florida
    University of Pennsylvania; University of Toronto; University of Western Ontario...
    Completed
    Impact of Vasculitis on Employment and Income
    Vasculitis | Microscopic Polyangiitis | Systemic Vasculitis | Takayasu's Arteritis | Cryoglobulinemic Vasculitis | Behcet's Disease | CNS Vasculitis | Temporal Arteritis | Polyarteritis Nodosa (PAN) | Urticarial Vasculitis | Henoch-Schoenlein Purpura | Wegener Granulomatosis | Eosinophilic Granulomatosis
    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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • City of Hope Medical Center
    National Cancer Institute (NCI)
    Completed
    Infection Prophylaxis and Management in Treating Cytomegalovirus (CMV) Infection in Patients With Hematologic Malignancies Previously Treated With Donor Stem Cell Transplant
    Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid... and other conditions
    United States
  • Fred Hutchinson Cancer Center
    National Cancer Institute (NCI)
    Completed
    Fludarabine and Total-Body Irradiation Followed By Donor Stem Cell Transplant and Cyclosporine and Mycophenolate Mofetil in Treating HIV-Positive Patients With or Without Cancer
    HIV Infection | Unspecified Childhood Solid Tumor, Protocol Specific | Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal... and other conditions
    United States
  • 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
  • Sanguine Biosciences
    Recruiting
    Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
    Heart Diseases | Stroke | Lymphoma | Multiple Sclerosis | Kidney Diseases | Diabetes | Leukemia | Age-Related Macular Degeneration | Cancer | Arthritis | Schizophrenia | Thyroid Diseases | Parkinson Disease | Hepatitis | Vasculitis | Psoriasis | Asthma | Amyloidosis | Fibromyalgia | Graves Disease | Alzheimer Disease | Celiac Disease | Cirr... and other conditions
    United States
  • RTI International
    Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators
    Enrolling by invitation
    Early Check: Expanded Screening in Newborns
    Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions
    United States
  • Fred Hutchinson Cancer Center
    National Cancer Institute (NCI)
    Completed
    Lithium Carbonate in Treating Patients With Acute Intestinal Graft-Versus-Host-Disease (GVHD) After Donor Stem Cell Transplant (GVHD)
    Primary Myelofibrosis | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal... and other conditions
    United States

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