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Found 23 studies with search of: "Mucopolysaccharidosis type I"
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Rank Status Study
1 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)
2 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-Schei Syndrome;   Scheie Syndrome
Intervention: Drug: Aldurazyme (laronidase)
3 Completed 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: Drug: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
4 Completed 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
Intervention: Drug: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
5 Recruiting A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG Levels in Aldurazyme® Treated Patients
Conditions: Mucopolysaccharidosis I;   Hurler's Syndrome;   Hurler-Scheie Syndrome;   Scheie's Syndrome
Intervention: Drug: Aldurazyme® (laronidase)
6 Recruiting Stem Cell Transplant w/Laronidase for Hurler
Conditions: Mucopolysaccharidosis I;   Hurler Syndrome
Interventions: Procedure: Stem Cell Transplant;   Drug: Laronidase ERT
7 Recruiting Clinical Trial of Growth Hormone in MPS I, II, and VI
Condition: Mucopolysaccharidosis Type I, II, and VI.
Intervention: Drug: Somatropin (DNA origin)
8 Recruiting Intrathecal Enzyme Replacement for Hurler Syndrome
Condition: MPS IH (Hurler Syndrome)
Intervention: Drug: IRT Laronidase
9 Completed A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
Conditions: Confirmed Diagnosis of Mucopolysaccharidosis I;   Less Than 5 Years of Age
Intervention: Drug: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
10 Recruiting Mucopolysaccharidosis I (MPS I) Registry
Condition: Mucopolysaccharidosis I
Intervention:
11 Recruiting Immune Tolerance Study With Aldurazyme®
Condition: Mucopolysaccharidosis I
Interventions: Biological: Aldurazyme® (laronidase);   Drug: Neoral® (CsA);   Drug: Imuran® (Aza)
12 Not yet 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
13 Recruiting 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
14 Recruiting Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
Conditions: Mucopolysaccharidosis I;   Lysosomal Storage Diseases;   Spinal Cord Compression
Intervention: Drug: laronidase
15 Completed 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
16 Active, not recruiting 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:
17 Recruiting Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
Conditions: Red Blood Cell Defects;   Sickle Cell Disease;   Thalassemia;   Blackfan-Diamond Anemia;   Leukocyte Defects and Immune Deficiencies;   Hereditary Lymphohistiocytosis;   Chronic Granulomatous Disease;   Wiskott-Aldrich Syndrome;   Chediak Higashi Syndrome;   CD40 Ligand Deficiency;   Hyper IgM Syndrome;   Leucocytes Adhesion Defect Type 1;   Osteopetrosis;   Congenital Neutropenia;   X-Linked Lymphoproliferative Disease;   Platelets Defects;   Congenital Amegakaryocytic Thrombocytopenia;   Metabolic and Storage Disorders;   Hurler Disease;   Leukodystrophies;   Niemann-Pick Disease;   Fucosidosis;   Stem Cell Defects;   Reticular Agenesis
Intervention: Procedure: Hematopoietic stem cell transplantation
18 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
19 Recruiting 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: Campath, Busulfan, Cyclophosphamide
20 Active, not recruiting Effects of Growth Hormone in Chronically Ill Children
Conditions: - Hurler Syndrome (MPS-1) With Short Stature and Muscle Wasting;   - Cerebral Palsy With Muscle Wasting;   - Juvenile Rheumatoid Arthritis With Muscle Wasting and Short Stature;   - Crohn’s Disease;   - HIV Infection.
Interventions: Drug: Growth Hormone;   Procedure: Whole body Protein turnover;   Procedure: DEXA scan

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