AREA[OverallStatus] Red-Cell Aplasia, Pure, Open or Pending: 15 Clinical Trials, Page 1 of 2

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National Registry of Rare Kidney Diseases

Condition(s):Adenine Phosphoribosyltransferase Deficiency; AH Amyloidosis; AHL Amyloidosis; AL Amyloidosis; Alport Syndrome; Atypical Hemolytic Uremic Syndrome; Autoimmune Distal Renal Tubular Acidosis; Autosomal Recessive Proximal Renal Tubular Acidosis; Autosomal Recessive Distal Renal Tubular Acidosis; Autosomal Dominant Polycystic Kidney Disease; Autosomal Recessive Polycystic Kidney Disease; Bartter Syndrome; BK Nephropathy; C3 Glomerulopathy With Monoclonal Gammopathy; C3 Glomerulopathy; Calciphylaxis; Crystalglobulinaemia; Crystal-storing Histiocytosis; Cystinosis; Cystinuria; Dense Deposit Disease; Dent Disease; Denys-Drash Syndrome; Dominant Hypophosphataemia With Nephrolithiasis and/or Osteoporosis; Drug Induced Fanconi Syndrome; Drug-Induced Hypomagnesemia; Drug-Induced Nephrogenic Diabetes Insipidus; Epilepsy, Ataxia, Sensorineural Deafness and Tubulopathy; Fabry Disease; Familial Hypomagnesemia With Hypercalciuria and Nephrocalcinosis; Familial Primary Hypomagnesemia With Hypocalcuria; Familial Primary Hypomagnesaemia With Normocalciuria; Familial Renal Glucosuria; Fanconi Renotubular Syndrome 1; Fanconi Renotubular Syndrome 2; Fanconi Renotubular Syndrome 3; Fibrillary Glomerulonephritis; Fibromuscular Dysplasia; Focal Segmental Glomerulosclerosis; Generalised Pseudohypoaldosteronism Type 1; Gitelman Syndrome; Heavy-Metal-Induced Fanconi Syndrome; Hepatocyte Nuclear Factor 1-Beta-Associated Monogenic Diabetes; Hereditary Renal Hypouricemia; Hereditary Hypophosphatemic Rickets With Hypercalciuria; Hyperuricaemic Nephropathy; IgA Nephropathy; Immunotactoid Glomerulonephritis With Organised Microtubular Mononoclonal Immunoglobulin Deposits; Inherited Renal Cancer Syndromes; Intracapillary Monoclonal IgM Without Cryoglobulin; Intraglomerular/Capillary Lymphoma/Leukaemia; Isolated Autosomal Dominant Hypomagnesaemia Glaudemans Type; Liddle Syndrome; Light Chain Cast Nephropathy; Light Chain Proximal Tubulopathy Without Crystals; Light Chain Proximal Tubulopathy With Crystals; Lowe Syndrome; Membranous Nephropathy; Membranoproliferative Glomerulonephritis; Medullary Cystic Kidney Disease; Minimal Change Nephropathy; Mitochondrial Disease Of The Kidney; Monoclonal Immunoglobulin Deposition Disease; Nail Patella Syndrome; Nephrogenic Diabetes Insipidus; Nephrogenic Syndrome of Inappropriate Antidiuresis; Nephronophthisis; Primary Hypomagnesemia With Secondary Hypocalcemia; Primary Hyperoxaluria; Proliferative Glomerulonephritis With Monoclonal IgG Deposits; Proximal Tubulopathy Without Crystals; Pseudohypoaldosteronism Type 1, 2A-2E; Pure Red Cell Aplasia; Retroperitoneal Fibrosis; Sickle Cell Nephropathy; Shiga Toxin Associated Haemolytic Uraemic Syndrome; Steroid Resistant Nephrotic Syndrome; Steroid-Sensitive Nephrotic Syndrome; Thin Basement Membrane Nephropathy; Thrombotic Microangiopathy With Monoclonal Gammopathy; Type 1 Cryoglobulinaemic Glomerulonephritis; Tuberous Sclerosis; Unclassified Monoclonal Gammopathy Of Renal Significance; VasculitisLast Updated:October 4, 2023Recruiting

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

Condition(s):Bone Marrow Failure Syndrome; Congenital Amegakaryocytic Thrombocytopenia; Congenital Pure Red Cell Aplasia; Hereditary Sideroblastic Anemia; Paroxysmal Nocturnal Hemoglobinuria; Shwachman-Diamond Syndrome; Hematologic Neoplasm With Germline GATA2 Mutation; Hematologic Neoplasm With Germline SAMD9 Mutation; Hematologic Neoplasm With Germline SAMD9L MutationLast Updated:November 22, 2023Recruiting

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Baby Detect : Genomic Newborn Screening

Condition(s):Congenital Adrenal Hyperplasia; Familial Hyperinsulinemic Hypoglycemia 1; Phosphoglucomutase 1 Deficiency; Maturity Onset Diabetes of the Young; Cystic Fibrosis; Hypophosphatasia, Infantile; Congenital Hypothyroidism; Deficit in Anterior Pituitary Function and Variable Immunodeficiency; Pituitary Hormone Deficiency, Combined; Diamond Blackfan Anemia; Wiskott-Aldrich Syndrome; Fanconi Anemia; Hemophilia A; Hemophilia B; Glucose 6 Phosphate Dehydrogenase Deficiency; Alpha-Thalassemia; Sickle Cell Disease; Shwachman-Diamond Syndrome; Alpha 1-Antitrypsin Deficiency; Inflammatory Bowel Disease 25, Autosomal Recessive; Wilson Disease; Progressive Familial Intrahepatic Cholestasis; Crigler-Najjar Syndrome; Familial Chylomicronemia; Lysosomal Acid Lipase Deficiency; Familial Hemophagocytic Lymphocytosis; Griscelli Syndrome; Chediak-Higashi Syndrome; Severe Congenital Neutropenia; Severe Combined Immune Deficiency; Chronic Granulomatous Disease; Menkes Disease; Adrenoleukodystrophy; Smith-Lemli-Opitz Syndrome; Ataxia With Vitamin E Deficiency; Thiamine Metabolism Dysfunction Syndrome 5 (Episodic Encephalopathy Type); Thiamine Metabolism Dysfunction Syndrome 4 (Bilateral Striatal Degeneration and Progressive Polyneuropathy Type); Thiamine-Responsive Megaloblastic Anemia; Thiamine Metabolism Dysfunction Syndrome 2; Deficiency of GOT2; Cerebral Folate Transport Deficiency; Segawa Syndrome, Autosomal Recessive; Congenital Myasthenic Syndrome; Metachromatic Leukodystrophy; Sepiapterin Reductase Deficiency; Dopamine Beta Hydroxylase Deficiency; Glut1 Deficiency Syndrome; Late-Infantile Neuronal Ceroid Lipofuscinosis; Aromatic L-amino Acid Decarboxylase Deficiency; Charcot-Marie-Tooth Disease, Type 6C; Hereditary Hyperekplexia; Brain Dopamine-Serotonin Vesicular Transport Disease; Very Long Chain Hydroxy Acyl Dehydrogenase Deficiency; Tyrosinemia, Type I; Disaccharide Intolerance I; Beta Ketothiolase Deficiency; Phosphoglycerate Dehydrogenase Deficiency; Succinyl-Coa:3-Ketoacid Coa-Transferase Deficiency; Pyridoxine-5′-Phosphate Oxidase Deficiency; Pyridoxine-Dependent Epilepsy; Propionic Acidemia; Pompe Disease; Phenylalanine Hydroxylase Deficiency; Ornithine Transcarbamylase Deficiency; N Acetyl Glutamate Synthetase Deficiency; Riboflavin Deficiency; Maple Syrup Urine Disease; Medium Chain Acyl CoA Dehydrogenase Deficiency; Malonic Acidemia; Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency; Isovaleric Acidemia; Phosphoserine Aminotransferase Deficiency; Phosphoserine Phosphatase Deficiency; Hyperornithinemia-Hyperammonemia-Homocitrullinuria; S-Adenosylhomocysteine Hydrolase Deficiency; Mucopolysaccharidosis VII; Mucopolysaccharidosis VI; Mucopolysaccharidosis IV A; Mucopolysaccharidosis II; Mucopolysaccharidosis I; Transcobalamin Deficiency; Isolated Methylmalonic Acidemia; Cobalamin Deficiency; Homocystinuria; Holocarboxylase Synthetase Deficiency; Fanconi Bickel Syndrome; Glycogen Storage Disease; Glycine Encephalopathy; Glutaric Acidemia I; Glucose Galactose Malabsorption; Gaucher Disease, Type 1; Galactosemias; Fructosemia; Fructose-1,6-Diphosphatase Deficiency; Carbamoyl Phosphate Synthase 1 Deficiency; Citrullinemia Type II; Citrullinemia 1; Creatine Deficiency Syndrome; Systemic Primary Carnitine Deficiency; Carnitine Palmitoyltransferase Deficiency 2; Carnitine Palmitoyltransferase Deficiency 1; Carnitine Acylcarnitine Translocase Deficiency; Riboflavin Transporter Deficiency; Branched-Chain Keto Acid Dehydrogenase Kinase Deficiency; Andersen Tawil Syndrome; Timothy Syndrome; Jervell-Lange Nielsen Syndrome; Catecholaminergic Polymorphic Ventricular Tachycardia; Familial Hypertrophic Cardiomyopathy Type 4; Pseudohypoaldosteronism, Type II; Pseudohypoaldosteronism Type 1; Primary Hyperoxaluria; X Linked Hypophosphatemia; Hereditary Nephrogenic Diabetes Insipidus; Cystinosis; Congenital Nephrotic Syndrome, Finnish Type; Alport Syndrome; Hereditary Retinoblastoma; Biotinidase Deficiency; Aciduria, Argininosuccinic; Argininemia; Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of; 3-Hydroxy 3-Methyl Glutaric Aciduria; 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 DeficiencyLast Updated:October 25, 2023Recruiting

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