40 studies found for:    Open Studies | "Mucopolysaccharidoses"
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Rank Status Study
21 Recruiting Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
Conditions: Mucopolysaccharidosis;   Fabry Disease
Intervention:
22 Recruiting A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Condition: MPS IV A
Intervention:
23 Recruiting The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients
Condition: Hunter Syndrome
Intervention:
24 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
25 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
26 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
27 Recruiting Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)
Conditions: Mucopolysaccharidosis II;   MPS II;   Hunter Syndrome
Intervention:
28 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
29 Recruiting Intrathecal Enzyme Replacement for Hurler Syndrome
Condition: Hurler Syndrome
Intervention: Drug: IRT Laronidase
30 Recruiting Biomarker for Morquio Disease
Conditions: Lysosomal Storage Diseases;   Morquio Disease
Intervention:
31 Recruiting Biomarker for Maroteaux-Lamy Disease
Conditions: Maroteaux-lamy Disease;   Lysosomal Storage Diseases
Intervention:
32 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
33 Recruiting Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
Condition: Hurler Syndrome
Intervention: Drug: Laronidase
34 Recruiting Gait Analysis in MPS IVA
Conditions: MPS IVA;   Morquio Syndrome
Intervention:
35 Not yet recruiting Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
Condition: Maroteaux-Lamy Syndrome
Intervention: Drug: Naglazyme®
36 Recruiting Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
Condition: Hunter Syndrome
Interventions: Biological: idursulfase-IT;   Other: No IT treatment
37 Recruiting Neurobehavioral Phenotypes in MPS III
Conditions: Sanfilippo Syndrome Type A;   Sanfilippo Syndrome Type B;   Hurler Syndrome
Intervention:
38 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: Mps III A
Intervention: Drug: Recombinant human heparan N-sulfatase [rhHNS]
39 Not yet recruiting Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
Conditions: Primary Immune Deficiency Disorders;   Hemophagocytic Lymphohistiocytosis;   Inherited Bone Marrow Failure Syndrome;   Hemoglobinopathies;   Metabolic Disorders
Intervention: Biological: BPX-501 and AP1903
40 Recruiting Reduced Intensity Conditioning in Patients Aged ≤30 With Non-Malignant Disorders Undergoing Cord Blood Transplantation
Conditions: Primary Immunodeficiency Syndromes;   Congenital Bone Marrow Failure Syndromes;   Inherited Metabolic Disorders (IMD);   Hereditary Anemias;   Patients With Sickle Disease Presenting Specific Symptoms
Interventions: Drug: Hydroxyurea;   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Melphalan;   Drug: Thiotepa

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Indicates status has not been verified in more than two years