Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
106 studies found for:    Mucopolysaccharidoses
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Rank Status Study
21 Recruiting The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients
Condition: Hunter Syndrome
Intervention:
22 Completed
Has Results
Immune Tolerance Study With Aldurazyme® (Laronidase)
Condition: Mucopolysaccharidosis I
Interventions: Biological: Laronidase;   Drug: Cyclosporine A (CsA);   Drug: Azathioprine (Aza)
23 Temporarily not available An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Condition: Mucopolysaccharidosis Type 7
Intervention: Drug: UX003
24 Recruiting Longitudinal Studies of Brain Structure and Function in MPS Disorders
Conditions: Mucopolysaccharidosis Type I;   Mucopolysaccharidosis Type II;   Mucopolysaccharidosis Type VI
Intervention:
25 Recruiting Longitudinal Study of Bone Disease in Children With Mucopolysaccharidoses (MPS) I, II, and VI
Condition: Mucopolysaccharidoses
Intervention:
26 Recruiting Intrathecal Enzyme Replacement for Hurler Syndrome
Condition: Hurler Syndrome
Intervention: Drug: IRT Laronidase
27 Completed
Has Results
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Conditions: Mucopolysaccharidosis I;   Hurler Syndrome;   Hurler-Scheie Syndrome;   Scheie Syndrome
Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
28 Completed
Has Results
Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
Conditions: Mucopolysaccharidosis I;   Hurlers Syndrome;   Hurler Scheie Syndrome;   Scheie
Interventions: Biological: rhIDU (recombinant human-Alpha-L-Iduronidase);   Biological: Placebo
29 Completed
Has Results
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
Conditions: Mucopolysaccharidosis I;   Hurler's Syndrome;   Hurler-Scheie Syndrome;   Scheie Syndrome
Interventions: Biological: Aldurazyme;   Biological: placebo
30 Recruiting A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
Conditions: Mucopolysaccharidosis I;   Hurler's Syndrome;   Hurler-Scheie Syndrome;   Scheie
Intervention: Biological: Aldurazyme (laronidase)
31 Terminated Safety and Exercise Study of BMN 110 for Morquio A Syndrome
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
32 Terminated Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
33 Recruiting Natural History Study to Characterise the Course of Disease Progression in Patients With Mucopolysaccharidosis Type IIIB
Condition: MPS IIIB (Sanfilippo B Syndrome)
Intervention:
34 Completed Iduronate-2-Sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
Condition: Mucopolysaccharidosis II
Intervention: Drug: Iduronate-2-sulfatase enzyme replacement therapy
35 Recruiting MT2013-31:Allo BMT for Metabolic Disorders, Osteopetrosis and Males With Rett Syndrome
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis II;   Mucopolysaccharidosis VI;   Mucopolysaccharidosis VII;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux Lamy Syndrome;   Sly Syndrome;   Glycoprotein Metabolic Disorders;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Adrenoleukodystrophy;   Peroxisomal Disorders;   Osteopetrosis;   Rett Syndrome;   Sphingolipidosis;   Gangliosidosis;   Globoid Cell Leukodystrophy;   Metachromatic Leukodystrophy;   Niemann Pick B;   Niemann Pick C Subtype 2;   I-cell Disease
Interventions: Procedure: blood stem cell transplant;   Drug: Rabbit Anti-Thymocyte Globulin (ATG);   Drug: Fludarabine;   Drug: Busulfan;   Drug: Cyclophosphamide;   Drug: Cyclosporine A (CSA);   Drug: Methylprednisolone;   Drug: Mycophenolate Mofetil (MMF);   Drug: Granulocyte-Colony Stimulating Factor (G-CSF);   Drug: Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF);   Drug: N-acetylcysteine;   Drug: Celecoxib;   Drug: Vitamin E;   Drug: Alpha Lipoic Acid
36 Unknown  Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
Conditions: Mucopolysaccharidosis;   Fabry Disease
Intervention:
37 Recruiting A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
Condition: MPS IIIB (Sanfilippo Syndrome)
Intervention:
38 Recruiting Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in MPS IIIB
Condition: Mucopolysaccharidosis IIIB
Intervention: Drug: SBC-103
39 Recruiting Biomarker for Hunter Disease
Conditions: Lysosomal Storage Diseases;   Hunter Disease;   Mucopolysaccharidoses
Intervention:
40 Completed To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
Condition: Mucopolysaccharidosis II
Interventions: Drug: GC1111_0.5mg/kg;   Drug: GC1111_1.0mg/kg;   Drug: Elaprase_0.5mg/kg

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Indicates status has not been verified in more than two years