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41 studies found for:    Open Studies | "Mucopolysaccharidoses"
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Rank Status Study
21 Recruiting Biomarker for Hunter Disease
Conditions: Lysosomal Storage Diseases;   Hunter Disease;   Mucopolysaccharidoses
Intervention:
22 Recruiting The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients
Condition: Hunter Syndrome
Intervention:
23 Unknown  Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
Conditions: Mucopolysaccharidosis;   Fabry Disease
Intervention:
24 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
25 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
26 Recruiting Biomarker for Sly Disease
Conditions: Mucopolysaccharidosis VII;   Sly Disease;   Beta-Glucuronidase Deficiency;   GUSB Deficiency
Intervention:
27 Recruiting Biomarker for Patients With Hurler Disease or High-grade Suspicion for Hurler Disease
Conditions: Mucopolysaccharidosis Type I;   MPS I;   Hurler-Scheie Syndrome;   Gargoylism, Hurler 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 A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Conditions: Mucopolysaccharidosis IV Type A;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: Vimizim® (elosulfase alfa)
30 Not yet recruiting New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis
Condition: Mucopolysaccharidoses
Intervention:
31 Not yet recruiting Ascending Dose Study of Genome Editing by the Zinc Finger Protein (ZFP) Therapeutic SB-318 in Subjects With MPS I
Condition: MPS I
Intervention: Genetic: SB-318
32 Recruiting Biomarker for Maroteaux-Lamy Disease
Conditions: Maroteaux-lamy Disease;   Lysosomal Storage Diseases
Intervention:
33 Recruiting Biomarker for Morquio Disease
Conditions: Lysosomal Storage Diseases;   Morquio Disease
Intervention:
34 Not yet recruiting Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)
Condition: Hurler Syndrome, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Krabbe, Cerebral Adrenoleukodystrophy
Intervention: Drug: Umbilical cord blood transplantation with HSC835
35 Recruiting BMT Abatacept for Non-Malignant Diseases
Conditions: Hurler Syndrome;   Fanconi Anemia;   Glanzmann Thrombasthenia;   Wiskott-Aldrich Syndrome;   Chronic Granulomatous Disease;   Severe Congenital Neutropenia;   Leukocyte Adhesion Deficiency;   Shwachman-Diamond Syndrome;   Diamond-Blackfan Anemia;   Dyskeratosis-congenita;   Chediak-Higashi Syndrome;   Severe Aplastic Anemia;   Thalassemia Major;   Hemophagocytic Lymphohistiocytosis;   Sickle Cell Disease
Intervention: Drug: Abatacept
36 Recruiting Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
Condition: Hunter Syndrome
Intervention: Drug: Elaprase for intravenous (IV) infusion
37 Recruiting Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
Condition: Maroteaux-Lamy Syndrome
Intervention: Drug: Naglazyme®
38 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
39 Unknown  Gait Analysis in MPS IVA
Conditions: MPS IVA;   Morquio Syndrome
Intervention:
40 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

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