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Found 96 studies with search of:   "Sphingolipidoses"
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Rank Status Study
1 Recruiting Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
Conditions: Hurler's Syndrome;   Maroteaux-Lamy Syndrome;   Sly Syndrome;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Sphingolipidoses;   Krabbe Disease;   Wolman's Disease;   Niemann-Pick Disease Type B;   Niemann-Pick Disease, Type C
Interventions: Procedure: Stem Cell Transplantation;   Drug: Campath, Busulfan, Cyclophosphamide
2 Active, not recruiting Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Conditions: I Cell Disease;   Fucosidosis;   Globoid Cell Leukodystrophy;   Adrenoleukodystrophy;   Mannosidosis;   Niemann-Pick Disease;   Pulmonary Complications;   Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Metachromatic Leukodystrophy;   Gaucher's Disease;   Wolman Disease
Intervention:
3 Completed Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
Condition: Gaucher Disease
Intervention: Procedure: Blood sample
4 Completed Stem Cell Transplantation (SCT) for Genetic Diseases
Conditions: Thrombocytopenia;   Metachromatic Leukodystrophy;   Fanconi's Anemia;   Thalassemia Major;   Pure Red-Cell Aplasia;   Inborn Errors of Metabolism
Intervention: Procedure: Stem Cell Transplantation
5 Recruiting 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
6 Recruiting Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
Condition: Gaucher's Disease
Intervention: Drug: glucocerebrosidase
7 Completed Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease
Condition: Gaucher's Disease
Intervention: Gene Transfer: human glucocerebrosidase gene into autologous peripheral blood stem cells
8 Completed A Study of AT1001 in Patients With Fabry Disease
Condition: Fabry Disease
Intervention: Drug: AT1001 (migalastat hydrochloride)
9 Completed Stem Cell Transplant for Inborn Errors of Metabolism
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Gaucher's Disease;   Fucosidosis;   Wolman Disease;   Niemann-Pick Disease;   Batten Disease;   GM1 Gangliosidosis;   Tay Sachs Disease;   Sandhoff Disease
Interventions: Procedure: Stem Cell Transplant;   Drug: Busulfan, Cyclophosphamide, ATG
10 Recruiting Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) Study
Condition: Fabry Disease
Interventions: Drug: agalsidase alfa (Replagal®) and agalsidase beta (Fabrazyme®);   Drug: Enzyme Replacement Therapy (ERT)
11 Completed Alpha-Galactosidase A Replacement Therapy for Fabry Disease
Condition: Fabry Disease
Intervention: Drug: DRX005B
12 Completed Dosing Study of Replagal in Patients With Fabry Disease
Condition: Fabry Disease
Intervention: Drug: Replagal
13 Recruiting A Study of the Effects of Fabrazyme on Mother's Lactation and on the Growth, Development and Immunologic Response of Their Infants
Condition: Fabry Disease
Intervention: Drug: Fabrazyme (agalsidase beta)
14 Completed PEG-Glucocerebrosidase for the Treatment of Gaucher Disease
Condition: Gaucher's Disease
Intervention: Drug: Lysodase
15 Completed PET Scans in Normal Volunteers and Patients With Fabry Disease
Conditions: Fabry Disease;   Healthy
Intervention:
16 Enrolling by invitation Compassionate Use of Metazym in Patients With Late Infantile Metachromatic Leukodystrophy
Condition: Late Infantile Metachromatic Leukodystrophy
Intervention: Drug: rhASA
17 Completed Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity
Condition: Fabry Disease
Intervention:
18 Completed A 12-Week Safety and Pharmacodynamic Study of AT1001 in Female Patients With Fabry Disease
Condition: Fabry Disease
Intervention: Drug: AT1001 (migalastat hydrochloride)
19 Recruiting Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
Conditions: Red Blood Cell Defects;   Sickle Cell Disease;   Thalassemia;   Blackfan-Diamond Anemia;   Leukocyte Defects and Immune Deficiencies;   Hereditary Lymphohistiocytosis;   Chronic Granulomatous Disease;   Wiskott-Aldrich Syndrome;   Chediak Higashi Syndrome;   CD40 Ligand Deficiency;   Hyper IgM Syndrome;   Leucocytes Adhesion Defect Type 1;   Osteopetrosis;   Congenital Neutropenia;   X-Linked Lymphoproliferative Disease;   Platelets Defects;   Congenital Amegakaryocytic Thrombocytopenia;   Metabolic and Storage Disorders;   Hurler Disease;   Leukodystrophies;   Niemann-Pick Disease;   Fucosidosis;   Stem Cell Defects;   Reticular Agenesis
Intervention: Procedure: Hematopoietic stem cell transplantation
20 Recruiting Genetic Studies of Lysosomal Storage Disorders
Conditions: Gaucher's Disease;   Lysosomal Storage Disease
Intervention:

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