49 studies found for:    Open Studies | "Rare Diseases"
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Rank Status Study
21 Recruiting Pyrimethamine, Sulfadiazine, and Leucovorin in Treating Patients With Congenital Toxoplasmosis
Condition: Toxoplasmosis
Interventions: Drug: Leucovorin calcium;   Drug: Pyrimethamine;   Drug: Spiramycin;   Drug: Sulfadiazine
22 Recruiting A Phase 3 Study of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7
Conditions: MPS 7;   Sly Syndrome;   Mucopolysaccharidosis;   MPS VII
Intervention: Drug: UX003
23 Unknown  Genetic Study of Insulin-Like Growth Factor-I Receptor Mutations in Patients With Intrauterine Growth Retardation
Condition: Intrauterine Growth Retardation
Intervention:
24 Recruiting Hereditary Inclusion Body Myopathy-Disease Monitoring (HIBM-DMP): A Combined Registry and Prospective Observational Natural History Study to Assess HIBM Disease
Conditions: Hereditary Inclusion Body Myopathy;   GNE Myopathy;   Nonaka Disease;   Quadriceps Sparing Myopathy (QSM);   Distal Myopathy With Rimmed Vacuoles (DMRV)
Intervention:
25 Unknown  Study of Energy Expenditure in Infants With Ventricular Septal Defects
Condition: Heart Septal Defects, Ventricular
Intervention:
26 Unknown  Phase I Study of Vasoactive Intestinal Peptide in Patients With Acute Respiratory Distress Syndrome and Sepsis
Conditions: Sepsis;   Respiratory Distress Syndrome;   Respiratory Distress Syndrome, Adult
Intervention: Drug: vasoactive intestinal peptide
27 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
28 Unknown  Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
Condition: Gaucher's Disease
Intervention: Drug: glucocerebrosidase
29 Unknown  Genetic Study of the FBN1 Gene and Fibrillin-1 Abnormalities in Choctaw Native Americans and Other Patients With Systemic Sclerosis
Condition: Systemic Sclerosis
Intervention:
30 Unknown  Ultraviolet Light Therapy for Systemic Lupus Erythematosus
Condition: Systemic Lupus Erythematosus
Intervention: Procedure: ultraviolet A-1 light treatment
31 Unknown  Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis
Condition: Cerebrotendinous Xanthomatosis
Interventions: Drug: chenodeoxycholic acid;   Drug: lovastatin
32 Unknown  Immunologic Evaluation in Patients With DiGeorge Syndrome or Velocardiofacial Syndrome
Conditions: DiGeorge Syndrome;   Shprintzen Syndrome;   Chromosome Abnormalities;   Abnormalities, Multiple;   Conotruncal Cardiac Defects
Intervention:
33 Unknown  Studies in Porphyria I: Characterization of Enzyme Defects
Condition: Porphyria
Intervention:
34 Unknown  Study of Energy Requirements in Critically Ill Newborns
Condition: Respiratory Distress Syndrome
Intervention:
35 Unknown  Study of Systemic Amyloidosis Presentation and Prognosis
Condition: Amyloidosis
Intervention:
36 Unknown  International Registry for Severe Chronic Neutropenia
Condition: Neutropenia
Intervention:
37 Unknown  Study of Neurobehavioral Outcome in Children or Adolescents With Closed Head Injuries
Condition: Head Injuries, Closed
Intervention:
38 Unknown  Study of Depression, Peptides, and Steroids in Cushing's Syndrome
Condition: Cushing's Syndrome
Intervention:
39 Recruiting Evaluation of an Advanced Lower Extremity Neuroprostheses
Conditions: Spinal Cord Injuries;   Stroke;   Paralysis;   Tetraplegia;   Paraplegia
Intervention: Device: IST-16 (16-channel implanted stimulator-telemeter)
40 Unknown  Gluconeogenesis in Very Low Birth Weight Infants Who Are Receiving Nutrition By Intravenous Infusion
Conditions: Infant, Low Birth Weight;   Hyperglycemia
Interventions: Drug: alanine;   Drug: amino acids;   Drug: glucagon;   Drug: glucose;   Drug: glutamine;   Drug: glycerol;   Drug: insulin;   Drug: leucine;   Drug: lipids;   Drug: urea

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Indicates status has not been verified in more than two years