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10 studies found for:    "Mucopolysaccharidosis type VII"
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Rank Status Study
1 Completed An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With MPS 7
Condition: Mucopolysaccharidosis Type 7
Intervention: Drug: UX003
2 No longer 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
3 Recruiting Biomarker for Sly Disease
Conditions: Mucopolysaccharidosis VII;   Sly Disease;   Beta-Glucuronidase Deficiency;   GUSB Deficiency
Intervention:
4 Completed A Phase 3 Study of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7
Conditions: MPS 7;   Sly Syndrome;   Mucopolysaccharidosis;   MPS VII
Intervention: Drug: UX003
5 Recruiting A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7
Conditions: Sly Syndrome;   MPS VII;   Mucopolysaccharidosis;   Mucopolysaccharidosis VII
Intervention: Drug: UX003
6 Active, not recruiting An Open-Label Study of UX003-rhGUS Enzyme Replacement Treatment in MPS 7 Patients Less Than 5 Years of Age
Conditions: Sly Syndrome;   MPS VII;   Mucopolysaccharidosis;   Mucopolysaccharidosis VII
Intervention: Drug: UX003
7 Recruiting Longitudinal Studies of Brain Structure and Function in MPS Disorders
Conditions: Mucopolysaccharidosis Type I;   Mucopolysaccharidosis Type II;   Mucopolysaccharidosis Type VI;   Mucopolysaccharidosis Type IV;   Mucopolysaccharidosis Type VII
Intervention:
8 Terminated
Has Results
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: Cyclophosphamide;   Drug: Campath-1H;   Drug: Busulfan
9 Recruiting MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Conditions: Mucopolysaccharidosis Disorders;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux Lamy Syndrome;   Sly Syndrome;   Alpha-Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Glycoprotein Metabolic Disorders;   Sphingolipidoses;   Recessive Leukodystrophies;   Globoid Cell Leukodystrophy;   Metachromatic Leukodystrophy;   Niemann-Pick B;   Niemann-Pick C Subtype 2;   Sphingomyelin Deficiency;   Peroxisomal Disorders;   Adrenoleukodystrophy With Cerebral Involvement;   Zellweger Syndrome;   Neonatal Adrenoleukodystrophy;   Infantile Refsum Disease;   Acyl-CoA Oxidase Deficiency;   D-Bifunctional Enzyme Deficiency;   Multifunctional Enzyme Deficiency;   Alpha-methylacyl-CoA Racmase Deficiency;   Mitochondrial Neurogastrointestingal Encephalopathy;   Severe Osteopetrosis;   Hereditary Leukoencephalopathy;   Inherited Metabolic Disorders
Interventions: Biological: Stem Cell Transplantation;   Drug: IMD Preparative Regimen;   Drug: Osteopetrosis Only Preparative Regimen;   Drug: Osteopetrosis Haploidentical Only Preparative Regimen;   Drug: cALD SR-A (Standard-Risk, Regimen A);   Drug: cALD SR-B (Standard-Risk, Regimen B);   Drug: cALD HR-D (High-Risk, Regimen C);   Drug: cALD HR-D (High-Risk, Regimen D)
10 Active, not 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

Study has passed its completion date and status has not been verified in more than two years.