NIH Rare Diseases Clinical Research Network Opens 30 New Studies for Patient Accrual
There are now 58 studies open to enrollment. These studies are being conducted at 190 institutions and studying over 200 diseases.
| RDCRN Protocol Number | Protocol Title |
|---|---|
| 6101 | Peripheral Dopamine in Postural Tachycardia Syndrome |
| 6102 | Double blind, placebo controlled clinical trial to assess the efficacy, safety and tolerability of rifampicin in patients with Multiple System Atrophy |
| 6103 | Norepinephrine Transporter Blockade in Neurogenic Orthostatic Hypotension |
| 6104 | Prosthetic Baroreceptor System for Orthostatic Hypotension |
| 6201 | Modifier Genes in Cerebral Cavernous Malformations |
| 6202 | Innovative Approaches to Gauge Progression of Sturge-Weber Syndrome |
| 6203 | Cerebral Hemorrhage Risk in Hereditary Hemorrhagic Telangiectasia |
| 6204 | Establishing Reliability for Quantitative EEG, Transcranial Doppler, Behavioral Outcomes and Optical Coherence Tomography in Sturge-Weber Syndrome (SWS): The Next Step Toward Biomarker Development |
| 6301 | Dystonia Coalition Projects |
| 6401 | Tissue Bank Consortium for Salivary Gland Tumors
|
| 6501 | Longitudinal Study of Immune Mediated Disorders after Allogeneic HCT Protocol (Immune Mediated Disorders after Allo-HCT)
|
| 6502 | A Randomized Phase II Study of Imatinib and Rituximab for Cutaneous Sclerosis after Allogeneic Hematopoietic Cell Transplantation |
| 6601 | Charcot-Marie Tooth (CMT) Natural History Study: CMTIB, CMT2A, CMT4A and CMT4C |
| 6602 | Genetics of Charcot-Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT |
| 6603 | Development of Charcot-Marie Tooth (CMT) Peds Scale for Children with CMT |
| 6702 | Natural History and Structural Functional Relationships in Fabry Renal Disease |
| 6703 | Longitudinal Studies of Brain Structure and Function in Mucopolysaccharidosis (MPS) Disorders |
| 6704 | The Natural History of Mucolipidosis Type IV |
| 6705 | Longitudinal Study of Bone and Endocrine Disease in Children with Mucopolysaccharidosis (MPS) I, II, and VI: A Multicenter Study of the Lysosomal Disease Network |
| 6707 | Characterizing the Neurobehavioral Phenotype(s) in Mucopolysaccharidosis (MPS) III |
| 6713 | A Natural History Study of Hexosaminiidase Deficiency |
| 6714 | A Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I
|
| 6716 | Genotype-Phenotype Correlations of Late Infantile Neuronal Ceriod Lipofuscinosis |
| 6719 | Immune Response to Intrathecal Enzyme Therapy in Mucopolysaccharidosis I Patients |
| 6801 | The Nephrotic Syndrome Study Network (NEPTUNE) |
| 6901 | A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders |
| 7001 | Smith-Lemi-Opitz Syndrome: A Longitudinal Clinical Study of Patients Receiving Cholesterol Supplementation |
| 7101 | Tissue Bank Consortium for Salivary Gland Tumors |
| 7201 | Longitudinal Study of Porphyrias |
| 7301 | Natural History of and Genetic Modifiers in Spinocerebellar Ataxias |
| 5109 | Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders |
| 5524 | PEXIVAS - Plasma exchange and glucocorticoid dosing in the treatment of anti-neutrophil cytoplasm antibody associated vasculitis: an international randomised controlled trial |
| 5904 | Rare Genetic Disorders of the Airways: Cross-Sectional Comparison of Clinical Features, and Development of Novel Screening and Genetic Tests |
| 6105 | A Double- Blind, randomized, Placebo-Controlled Trial to evaluate the efficacy of Intravenous immunoglobulin Therapy in Autoimmune Autonomic Ganglionopathy |
| 6503 | Targeted Therapy of Bronchiolitis Obliterans Syndrome |
| 6706 | Epidemiology and Natural History of Woman and Cholesteryl Ester Storage Diseases |
| 6710 | Lysosomal Storage Disease: Health, Development, and Functional Outcomes in Preschool Children |
| 6712 | A Project to Investigate Clinical and Laboratory Features of Glycoprotein Storage Disorders |
| 6718 | Gene Therapy for Tay-Sachs Disease |
