68 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 Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))
Condition: Erythropoietic Protoporphyria (EPP)
Intervention:
6 Recruiting Clinical and Genetic Examination of Usher Syndrome Patients' Cohort in Europe
Condition: Usher Syndrome
Intervention:
7 Recruiting Nephrotic Syndrome Study Network
Conditions: Minimal Change Disease (MCD);   Membranous Nephropathy;   Glomerulosclerosis, Focal Segmental
Intervention:
8 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:
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 The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
Condition: Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE)
Intervention:
11 Recruiting Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) Study
Condition: Fabry Disease
Interventions: Drug: agalsidase alfa;   Drug: Agalsidase beta
12 Unknown  Pulmonary Hypertension, Quality of Life and Psychosocial Factors
Condition: Pulmonary Hypertension
Intervention: Behavioral: Evaluation of quality of life
13 Recruiting Follow-up Protocol for Patients With Cancer/AIDS/Skin Disease
Conditions: Acquired Immunodeficiency Syndrome;   Neoplasm;   Skin Disease;   HIV Infections
Intervention:
14 Recruiting Cure Cystinosis International Registry
Conditions: Cystinosis;   Nephropathic Cystinosis;   Renal Fanconi Syndrome
Intervention:
15 Recruiting Diagnosis and Treatment of Patients With Inborn Errors of Metabolism
Condition: Metabolism
Intervention:
16 Recruiting Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
Condition: Angelman Syndrome
Interventions: Drug: MINOCYCLINE;   Drug: PLACEBO (for Minocycline)
17 Recruiting T-Cell Project: Prospective Collection of Data in Patients With Peripheral T-Cell Lymphoma
Condition: Lymphoma, T-Cell, Peripheral
Intervention:
18 Recruiting Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Conditions: Mucopolysaccharidosis Type I (MPS I);   Mucopolysaccharidosis Type II (MPS II);   Mucopolysaccharidosis Type III (MPS III);   Mucopolysaccharidosis Type VI (MPS VI);   Krabbe Disease
Intervention:
19 Recruiting Cross-sectional Characterization of Idiopathic Bronchiectasis
Conditions: Idiopathic Bronchiectasis;   Nontuberculous Mycobacterium (NTM) Infection
Intervention:
20 Unknown  Phase II Randomized Study of Physiologic Testosterone Replacement in Premenopausal, HIV-Positive Women
Conditions: HIV Infections;   Cachexia
Intervention: Drug: testosterone

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Indicates status has not been verified in more than two years