39 studies found for:    "Mucopolysaccharidosis type I"
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Rank Status Study
1 Recruiting Mucopolysaccharidosis I (MPS I) Registry
Condition: Mucopolysaccharidosis I (MPS I)
Intervention:
2 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)
3 Terminated Clinical Trial of Growth Hormone in MPS I, II, and VI
Condition: Mucopolysaccharidosis Type I, II, and VI.
Intervention: Drug: Somatropin (DNA origin)
4 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)
5 Recruiting Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Conditions: Mucopolysaccharidosis Type I (MPS I);   Mucopolysaccharidosis Type II (MPS II);   Mucopolysaccharidosis Type III (MPS III);   Mucopolysaccharidosis Type VI (MPS VI);   Krabbe Disease
Intervention:
6 Completed
Has Results
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
Conditions: Mucopolysaccharidosis I;   Hurler's Syndrome;   Hurler-Scheie Syndrome;   Scheie Syndrome
Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
7 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
8 Completed
Has Results
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
Conditions: Mucopolysaccharidosis I;   Hurler Syndrome;   Hurler-Scheie Syndrome;   Scheie Syndrome
Intervention: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
9 Recruiting A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
Conditions: Cognitive Decline;   Mucopolysaccharidosis I;   Hurler-Scheie Syndrome;   Scheie Syndrome;   Lysosomal Storage Disease
Intervention: Drug: laronidase
10 Terminated Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
Conditions: Spinal Cord Compression;   Mucopolysaccharidosis I;   Hurler-Scheie Syndrome;   Scheie Syndrome;   Lysosomal Storage Disease
Intervention: Drug: laronidase
11 Completed
Has Results
Immune Tolerance Study With Aldurazyme® (Laronidase)
Condition: Mucopolysaccharidosis I
Interventions: Biological: Laronidase;   Drug: Cyclosporine A (CsA);   Drug: Azathioprine (Aza)
12 Recruiting Longitudinal Studies of Brain Structure and Function in MPS Disorders
Conditions: Mucopolysaccharidosis Type I;   Mucopolysaccharidosis Type II;   Mucopolysaccharidosis Type VI
Intervention:
13 Terminated Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics
Conditions: Mucopolysaccharidoses;   Mucopolysaccharidosis I;   Mucopolysaccharidosis II;   Mucopolysaccharidosis IV;   Mucopolysaccharidosis VI
Intervention: Other: Dried blood spot test for MPS
14 Completed A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
Conditions: Mucopolysaccharidosis I;   Hurler's Syndrome;   Hurler-Scheie Syndrome;   Scheie's Syndrome
Intervention: Drug: laronidase
15 Terminated Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
Conditions: Mucopolysaccharidosis I;   Lysosomal Storage Diseases;   Spinal Cord Compression
Intervention: Drug: laronidase
16 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
17 Recruiting Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)
Conditions: Mucopolysaccharidosis;   Mucopolysaccharidosis I;   Mucopolysaccharidosis II;   Mucopolysaccharidosis VI
Intervention:
18 Unknown  Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Conditions: I Cell Disease;   Fucosidosis;   Globoid Cell Leukodystrophy;   Adrenoleukodystrophy;   Mannosidosis;   Niemann-Pick Disease;   Pulmonary Complications;   Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Metachromatic Leukodystrophy;   Gaucher's Disease;   Wolman Disease
Intervention:
19 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
20 Completed
Has Results
Stem Cell Transplantation for Hurler
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Mannosidosis;   Mucolipidosis Type II (I-cell Disease)
Interventions: Procedure: Stem Cell Transplant;   Drug: Busulfan, Cyclophosphamide, ATG

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