Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
69 studies found for:    Open Studies | "Rare Diseases"
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Rank Status Study
1 Recruiting Rare Disease Patient Registry: Coordination of Rare Diseases at Sanford
Conditions: Rare Disorders;   Undiagnosed Disorders;   Disorders of Unknown Prevalence
Intervention:
2 Not yet recruiting Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in GRD
Condition: Genetic Rare Disease
Intervention: Drug: Busulfan
3 Recruiting Biobank Protocol, Rare Diseases Clinical Research Network
Conditions: Primary Hyperoxaluria;   Dent Disease;   APRT Deficiency;   Cystinuria
Intervention:
4 Recruiting Decision Support for Parents Receiving Information About Child's Rare Disease
Conditions: Disorder of Sex Development;   Intersex Conditions;   Congenital Adrenal Hyperplasia;   Hypospadias
Intervention: Behavioral: Decision Support Tool
5 Recruiting Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
Condition: Rare Genetic Disorders
Intervention:
6 Recruiting Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))
Condition: Erythropoietic Protoporphyria (EPP)
Intervention:
7 Recruiting The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
Condition: Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
Intervention:
8 Recruiting Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL)
Conditions: FTLD;   Progressive Supranuclear Palsy (PSP);   Frontotemporal Dementia (FTD);   Corticobasal Degeneration (CBD);   PPA Syndrome;   Behavioral Variant Frontotemporal Dementia (bvFTD);   Semantic Variant Primary Progressive Aphasia (svPPA);   Nonfluent Variant Primary Progressive Aphasia (nfvPPA);   FTD With Amyotrophic Lateral Sclerosis (FTD/ALS);   Amyotrophic Lateral Sclerosis (ALS);   Oligosymptomatic PSP (oPSP);   Corticobasal Syndrome (CBS)
Intervention:
9 Unknown  High Dose Intravenous N-Acetylcysteine Versus Iloprost for Early, Rapidly Progressive Diffuse Systemic Sclerosis
Condition: Scleroderma, Diffuse
Intervention: Drug: N-acetylcysteine (NAC)
10 Recruiting Clinical and Genetic Examination of Usher Syndrome Patients' Cohort in Europe
Condition: Usher Syndrome
Intervention:
11 Recruiting Nephrotic Syndrome Study Network
Conditions: Minimal Change Disease (MCD);   Membranous Nephropathy;   Glomerulosclerosis, Focal Segmental
Intervention:
12 Recruiting Pediatric Vasculitis Initiative
Conditions: Wegeners Granulomatosis (Granulomatosis With Polyangiitis);   Microscopic Polyangiitis;   Churg Strauss Syndrome (Eosinophilic Granulomatosis With Polyangiitis);   Polyarteritis Nodosa;   Takayasu Arteritis;   Primary CNS Vasculitis;   Unclassified Vasculitis
Intervention:
13 Recruiting Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) Study
Condition: Fabry Disease
Interventions: Drug: agalsidase alfa;   Drug: Agalsidase beta
14 Unknown  Pulmonary Hypertension, Quality of Life and Psychosocial Factors
Condition: Pulmonary Hypertension
Intervention: Behavioral: Evaluation of quality of life
15 Unknown  Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
Conditions: Gaucher's Disease;   Osteopenia
Interventions: Drug: alendronate sodium;   Drug: calcium carbonate;   Drug: cholecalciferol
16 Unknown  Study of Energy Requirements in Critically Ill Newborns
Condition: Respiratory Distress Syndrome
Intervention:
17 Unknown  Study of Systemic Amyloidosis Presentation and Prognosis
Condition: Amyloidosis
Intervention:
18 Recruiting Study of Total Body Irradiation in Combination With Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation Followed By Cyclosporine and Mycophenolate Mofetil in High Risk-Patients With Human Immunodeficiency Virus-1
Condition: HIV Infections
Interventions: Drug: Cyclosporine;   Drug: Mycophenolate mofetil;   Procedure: Total body irradiation and stem cell or bone marrow transplantation
19 Unknown  Phase I Study of Alpha-Melanocyte Stimulating Hormone in Patients With Acute Renal Failure
Condition: Acute Renal Failure
Intervention: Drug: alpha-melanocyte stimulating hormone
20 Unknown  Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
Condition: Gaucher's Disease
Intervention: Drug: glucocerebrosidase

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Indicates status has not been verified in more than two years