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45 studies found for:    Open Studies | "Mucopolysaccharidoses"
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Rank Status Study
21 Recruiting Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
Conditions: Morquio A Syndrome;   Mucopolysaccharidosis IV A
Intervention:
22 Recruiting Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
Conditions: Mucopolysaccharidosis;   Fabry Disease
Intervention:
23 Recruiting A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Condition: MPS IV A
Intervention:
24 Recruiting Natural History Study to Characterise the Course of Disease Progression in Patients With Mucopolysaccharidosis Type IIIB
Condition: MPS IIIB (Sanfilippo B Syndrome)
Intervention:
25 Recruiting A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
Condition: MPS IIIB (Sanfilippo Syndrome)
Intervention:
26 Recruiting The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients
Condition: Hunter Syndrome
Intervention:
27 Recruiting Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Conditions: Mucopolysaccharidosis;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux-Lamy Syndrome;   Sly Syndrome;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Adrenoleukodystrophy (ALD);   Krabbe Disease;   Metachromatic Leukodystrophy (MLD);   Sphingolipidoses;   Peroxisomal Disorders
Interventions: Drug: Campath-1H;   Drug: Cyclophosphamide;   Drug: Busulfan;   Procedure: Allogeneic stem cell transplantation;   Drug: Cyclosporine A;   Drug: Mycophenolate Mofetil
28 Recruiting Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
Conditions: Severe Combined Immunodeficiency;   Malignancy, Hematologic;   Neuroblastoma;   Neoplasm;   Mucopolysaccharidosis I
Intervention: Procedure: Venipuncture
29 Recruiting Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
Conditions: Mucopolysaccharidosis II;   MPS II;   Hunter Syndrome
Intervention:
30 Recruiting UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Conditions: Adrenoleukodystrophy;   Batten Disease;   Mucopolysaccharidosis II;   Leukodystrophy, Globoid Cell;   Leukodystrophy, Metachromatic;   Neimann Pick Disease;   Pelizaeus-Merzbacher Disease;   Sandhoff Disease;   Tay-Sachs Disease;   Brain Diseases, Metabolic, Inborn
Intervention: Biological: DUOC-01
31 Recruiting Biomarker for Sly Disease
Conditions: Mucopolysaccharidosis VII;   Sly Disease;   Beta-Glucuronidase Deficiency;   GUSB Deficiency
Intervention:
32 Recruiting Biomarker for Hurler Disease
Conditions: Mucopolysaccharidosis Type I;   MPS I;   Hurler-Scheie Syndrome;   Gargoylism, Hurler Syndrome
Intervention:
33 Recruiting Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
Conditions: Mucopolysaccharidosis Type IH;   MPS I;   Hurler Syndrome
Intervention: Drug: Laronidase
34 Recruiting A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Conditions: Mucopolysaccharidosis IV Type A;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: Vimizim® (elosulfase alfa)
35 Recruiting Intrathecal Enzyme Replacement for Hurler Syndrome
Condition: Hurler Syndrome
Intervention: Drug: IRT Laronidase
36 Recruiting Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Condition: Sanfilippo Syndrome Type A (MPS IIIA)
Intervention: Drug: Recombinant human heparan N-sulfatase [rhHNS]
37 Recruiting Biomarker for Morquio Disease
Conditions: Lysosomal Storage Diseases;   Morquio Disease
Intervention:
38 Recruiting Biomarker for Maroteaux-Lamy Disease
Conditions: Maroteaux-lamy Disease;   Lysosomal Storage Diseases
Intervention:
39 Recruiting HSCT for High Risk Inherited Inborn Errors
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Tay Sachs Disease;   Sandhoffs Disease;   Wolman Disease;   I-Cell Disease;   Sanfilippo Syndrome;   GM1 Gangliosidosis
Interventions: Drug: Clofarabine;   Procedure: Total body Irradiation;   Drug: Melphalan;   Biological: Hematopoietic Stem Cell Transplantation;   Drug: Alemtuzumab;   Drug: mycophenylate mofetil;   Device: Cyclosporine A;   Drug: Hydroxyurea
40 Recruiting Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
Condition: Hurler Syndrome
Intervention: Drug: Laronidase

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