Clinical Trials on Inherited Metabolic Storage Diseases

Clear Cell Renal Cell Carcinoma | Stage IV Renal Cell Cancer AJCC v8 | Metastatic Malignant Neoplasm in the Bone | Chromophobe Renal Cell Carcinoma | Papillary Renal Cell Carcinoma | Collecting Duct Carcinoma | Kidney Medullary Carcinoma | Advanced Renal Cell Carcinoma | Unclassified Renal Cell Carcinoma

United States

Hereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditions

United States, Bulgaria, Czechia, Hungary, Spain, France, Germany, Poland, Canada, Israel

Cystic Fibrosis | Primary Ciliary Dyskinesia | Kartagener Syndrome | Pseudohypoaldosteronism

United States, Canada

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

Lymphoma | Unspecified Childhood Solid Tumor, Protocol Specific | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Small Intestine Cancer | Unspecified Adult Solid Tumor, Protocol Specific | Multiple Myeloma and Plasma Cell Neoplasm | Precancerous Condition | Nausea and Vomiting

United States

Obesity | Craniopharyngioma

Germany

Hepatitis B | Pertussis | Tetanus | Diphtheria | Poliomyelitis | Haemophilus Influenzae Type b Infection

Depression | Diabetes Mellitus | Cancer | Atrial Fibrillation | Fibromyalgia | Blood Pressure | Syncope | Coronary Heart Disease | Chronic Fatigue Syndrome | Panic Attack | Diastolic Heart Failure | POTS | Systolic Heart Failure | Vasovagal Syncope | Acute Myocardial Infarction (AMI) | Transient Ischemic Attack (TIA) | Acute... and other conditions

Colombia

Ataxia | Multiple System Atrophy | Spinocerebellar Ataxias | Ataxia With Oculomotor Apraxia | CANVAS

Italy

Multiple Sclerosis | Pneumonia | Rheumatoid Arthritis | Systemic Lupus Erythematosus | Anxiety Disorders | Psoriasis | Asthma | Type 1 Diabetes | Graves Disease | Celiac Disease | Allergic Rhinitis | Urinary Tract Infections | Crohn Disease | Influenza | Ulcerative Colitis | Herpes Zoster | Herpes Simplex | Atopic Dermatitis | V... and other conditions

United Kingdom

Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Brain and Central Nervous System Tumors | Unspecified Adult Solid Tumor, Protocol Specific | Multiple Myeloma and Plasma Cell Neoplasm | Lymphoproliferative Disorder | Myelodysplastic/Myeloproliferative Neoplasms

United States

Gastrointestinal Stromal Tumor | Recurrent Adult Soft Tissue Sarcoma | Stage III Adult Soft Tissue Sarcoma | Stage IV Adult Soft Tissue Sarcoma | Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor | Adult Rhabdomyosarcoma | Metastatic Ewing Sarcoma/Peripheral Primitive Neuroectodermal... and other conditions

Liver Cirrhosis | Quality of Life | Neoplasm Metastasis | Pulmonary Disease, Chronic Obstructive | Peripheral Vascular Diseases | Dementia | Kidney Failure, Chronic | Chronic Disease | Lung Diseases, Interstitial | Health Communication | Lung Neoplasm | Heart Failure，Congestive | Patient Care | Health Care Quality... and other conditions

United States

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 | Recurrent Adult Diffuse Small Cleaved Cell Lymphoma and other conditions

United States

Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Brain and Central Nervous System Tumors | Unspecified Adult Solid Tumor, Protocol Specific | Multiple Myeloma and Plasma Cell Neoplasm | Lymphoproliferative Disorder | Myelodysplastic/Myeloproliferative Neoplasms

United States

Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Brain and Central Nervous System Tumors | Unspecified Adult Solid Tumor, Protocol Specific | Multiple Myeloma and Plasma Cell Neoplasm | Lymphoproliferative Disorder | Myelodysplastic/Myeloproliferative Neoplasms

Korea, Republic of

BRCA1 Gene Mutation | BRCA2 Gene Mutation | Ductal Breast Carcinoma in Situ | Atypical Ductal Breast Hyperplasia | Lobular Breast Carcinoma in Situ

United States

Multiple Sclerosis | Lower Urinary Tract Symptoms | Parkinson Disease | Spinal Cord Injuries | Bladder Outlet Obstruction | Cauda Equina Syndrome | Detrusor Underactivity | Enlarged Prostate With Lower Urinary Tract Symptoms

United Kingdom, United States, France

Tumor | Neuroma | Mandibular Fractures | Treacher Collins Syndrome | Facial Fracture | Fibrous Dysplasia | Hemifacial Microsomia | Miller Syndrome | Osteoma of Mandibular Condyle | Cleft Face | Nager Syndrome

Argentina

Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Neutropenia | Unspecified Adult Solid Tumor, Protocol Specific | Infection | Multiple Myeloma and Plasma Cell Neoplasm | Precancerous Condition | Fever, Sweats, and Hot Flashes | Myelodysplastic/Myeloproliferative Neoplasms

France, Switzerland, Belgium, Germany, Italy, Israel, Slovakia, Turkey

Parkinson Disease | Dyskinesias | Temporomandibular Joint Disorders | Dystonia | Torticollis | Essential Tremor | Tic Disorders | Dysphonia | Myoclonus | Ulnar Nerve Entrapment | Drug Induced Dystonia

United States

Diabetic Retinopathy | Retinal Vein Occlusion | Retinitis Pigmentosa | Optic Atrophy | Retinal Detachment | Epiretinal Membrane | Macular Holes | Central Serous Chorioretinopathy | Retinal Artery Occlusion | Suspect Glaucoma | Pathologic Myopia | Nonexudative Age-related Macular Degeneration | Exudative Age Related...

China

Recurrent Endometrial Adenocarcinoma | Recurrent Endometrial Carcinoma | Recurrent Endometrial Clear Cell Adenocarcinoma | Recurrent Endometrial Endometrioid Adenocarcinoma | Recurrent Endometrial Mixed Cell Adenocarcinoma | Recurrent Endometrial Serous Adenocarcinoma | Recurrent Endometrial Undifferentiated... and other conditions

United States

Rheumatoid Arthritis | Uveitis | Vasculitis | Type 1 Diabetes | Crohn's Disease | Myositis | Healthy Volunteer | Ankylosing Spondylitis | FMF | Systemic Lupus Erythematosus/Antiphospholipid Syndrome | Cryopyrin-Associated Periodic Syndromes /TNF-receptor Associated Periodic Syndrome | Ulcerative Rectocolitis | Unclassified...

France

Recurrent Hodgkin Lymphoma | Chronic Myelomonocytic Leukemia | Previously Treated Myelodysplastic Syndrome | Recurrent Adult Acute Myeloid Leukemia | Acute Lymphoblastic Leukemia | Secondary Acute Myeloid Leukemia | Myelodysplastic Syndrome | Recurrent Acute Myeloid Leukemia | Myelodysplastic Syndrome... and other conditions

United States

Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Brain and Central Nervous System Tumors | Multiple Myeloma and Plasma Cell Neoplasm | Nonmalignant Neoplasm | Lymphoproliferative Disorder | Myelodysplastic/Myeloproliferative Neoplasms | Viral Infection

United States

Epilepsy | Renal Cell Carcinoma | Hypertension | Diabetes | Restless Legs Syndrome | Pancreatic Cancer | Neuropathic Pain | Chronic Pancreatitis | Renal Cancer | Renal Pelvis Cancer

Neoplasms | Neoplasms by Histologic Type | Lung Diseases | Neoplasms by Site | Adenocarcinoma | Carcinoma | Neoplasms, Glandular and Epithelial | Endocrine System Diseases | Gastrointestinal Neoplasms | Neoplasms, Germ Cell and Embryonal | Head and Neck Neoplasms | Carcinoma, Non-Small-Cell Lung | Respiratory Tract... and other conditions

France, Spain, United States, Korea, Republic of, United Kingdom, Taiwan, China, Belgium, Singapore, Netherlands, Germany, Italy, Hong Kong

Hepatitis B | Measles | Pertussis | Rubella | Mumps | Tetanus | Diphtheria | Varicella | Poliomyelitis | Haemophilus Infections

Germany, Italy

Pain | Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Brain and Central Nervous System Tumors | Unspecified Adult Solid Tumor, Protocol Specific | Multiple Myeloma and Plasma Cell Neoplasm | Precancerous Condition | Lymphoproliferative Disorder | Myelodysplastic/Myeloproliferative...

United States

Hepatitis B | Pertussis | Tetanus | Diphtheria | Poliomyelitis | Haemophilus Influenzae Type B Infection

Hepatitis B | Pertussis | Tetanus | Diphtheria | Poliomyelitis | Haemophilus Influenzae Type B Infection

Mexico

Castration-Resistant Prostate Carcinoma | Metastatic Prostate Carcinoma | Stage IV Prostate Cancer AJCC v8 | Aggressive Variant Prostate Carcinoma | Metastatic Prostate Neuroendocrine Carcinoma | Metastatic Prostate Small Cell Carcinoma

United States

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

Hepatocellular Carcinoma | Pancreatic Cancer | Ovarian Cancer | Cholangiocarcinoma | Triple Negative Breast Cancer | Metastatic Melanoma | Colon Cancer | Synovial Sarcoma | Breast Cancer Metastatic | Metastatic Colon Cancer | Melanoma Stage IV | Melanoma Recurrent | TNBC - Triple-Negative Breast Cancer | NSCLC, Metastatic and other conditions

United States

Ovarian Diseases | Ovarian Neoplasms | Ovarian Cancer | Ovarian Epithelial Cancer | Fallopian Tube Cancer | Endometrial Neoplasms | Endometrial Cancer | Endometrial Clear Cell Adenocarcinoma | Ovarian Carcinoma | Endometrial Adenocarcinoma | Peritoneal Cancer | Endometrioid Adenocarcinoma | Ovarian Adenocarcinoma | Ovarian Serous Adenocarcinoma and other conditions

United States

Pain | Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Brain and Central Nervous System Tumors | Unspecified Adult Solid Tumor, Protocol Specific | Multiple Myeloma and Plasma Cell Neoplasm | Precancerous Condition | Lymphoproliferative Disorder | Myelodysplastic/Myeloproliferative...

United States

Pain | Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Brain and Central Nervous System Tumors | Unspecified Adult Solid Tumor, Protocol Specific | Multiple Myeloma and Plasma Cell Neoplasm | Precancerous Condition | Lymphoproliferative Disorder | Myelodysplastic/Myeloproliferative...

United States

Lymphoma | Unspecified Childhood Solid Tumor, Protocol Specific | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Brain and Central Nervous System Tumors | Unspecified Adult Solid Tumor, Protocol Specific | Multiple Myeloma and Plasma Cell Neoplasm | Nonmalignant Neoplasm | Lymphoproliferative Disorder and other conditions

Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Brain and Central Nervous System Tumors | Unspecified Adult Solid Tumor, Protocol Specific | Metastatic Cancer | Multiple Myeloma and Plasma Cell Neoplasm | Precancerous Condition | Lymphoproliferative Disorder | Secondary...

United States

Cervical Carcinoma | Human Papillomavirus Infection | Health Status Unknown | Cervical Intraepithelial Neoplasia Grade 2/3 | Stage 0 Cervical Cancer | Atypical Squamous Cell of Undetermined Significance | Low Grade Cervical Squamous Intraepithelial Neoplasia

United States

Recurrent Hodgkin Lymphoma | Refractory Hodgkin Lymphoma | Recurrent Non-Hodgkin Lymphoma | Refractory Non-Hodgkin Lymphoma | Recurrent Breast Carcinoma | Recurrent Mycosis Fungoides | Recurrent Primary Cutaneous T-Cell Non-Hodgkin Lymphoma | Stage IV Breast Cancer AJCC v6 and v7 | Breast Adenocarcinoma and other conditions

United States

Myelodysplastic Syndromes | Vitamin D Deficiency | Pediatric Cancer | Aplastic Anemia | Stem Cell Transplant Complications | Sickle Cell Anemia in Children | Thalassemia in Children | Blood Disorder | Pediatric Acute Myeloid Leukemia | Pediatric Acute Lymphoid Leukemia

United States

Sepsis | Burns | Septic Shock | Trauma | Infectious Disease | Pancreatitis | Acute Respiratory Distress Syndrome | Liver Transplant; Complications | Drug Overdose | Acute Liver Failure | Cardiogenic Shock | Rhabdomyolysis | Acute on Chronic Liver Failure | Hemophagocytic Lymphohistiocytoses | Extracorporeal Life Support and other conditions

Spain, Italy, Germany, Portugal

Clear Cell Renal Cell Carcinoma | Fallopian Tube Cancer | Solid Tumor | Neuroendocrine Tumors | Epithelial Ovarian Cancer | Primary Peritoneal Carcinoma | Extensive-stage Small-cell Lung Cancer | MSI-H Cancer | Cancer With A High Tumor Mutational Burden

United States, Canada

Lymphoma | Unspecified Childhood Solid Tumor, Protocol Specific | Leukemia | Testicular Germ Cell Tumor | Breast Cancer | Unspecified Adult Solid Tumor, Protocol Specific | Multiple Myeloma and Plasma Cell Neoplasm

United States

Prader-Willi Syndrome | DiGeorge Syndrome | 22q11 Deletion Syndrome | Angelman Syndrome | Trisomy 21 | Trisomy 18 | Trisomy 13 | Monosomy X | Sex Chromosome Abnormalities | 1p36 Deletion Syndrome | Cri-du-Chat Syndrome

United States, Australia, Ireland, Spain, Sweden, United Kingdom

Non-Small Cell Lung Cancer | Colon Cancer | Medullary Thyroid Cancer | Any Solid Tumor

United States, Japan, Taiwan, Australia, Korea, Republic of, Israel, Spain, Switzerland, Singapore, France, Canada, Italy, Germany, United Kingdom, Hong Kong, Denmark

Lymphoma | Kidney Cancer | Myelodysplastic Syndromes | Leukemia | Testicular Germ Cell Tumor | Breast Cancer | Chronic Myeloproliferative Disorders | Ovarian Cancer | Gestational Trophoblastic Tumor | Neuroblastoma | Multiple Myeloma and Plasma Cell Neoplasm | Graft Versus Host Disease

United States

Coronary Artery Disease | Diabetes | Joint Diseases | Diabetic Foot Ulcer | Interstitial Lung Disease | Hyperaldosteronism | Kidney Stone | Intracranial Arteriovenous Malformations | Adrenal Incidentaloma | Parenchymatous; Pneumonia | Inner Ear Disease | Brain Stroke | Macroadenoma

France