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
37 studies found for:    "Mucopolysaccharidosis type I"
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Rank Status Study
1 Terminated
Has Results
Clinical Trial of Growth Hormone in MPS I, II, and VI
Conditions: Mucopolysaccharidosis I;   Mucopolysaccharidosis II;   Mucopolysaccharidosis VI
Intervention: Drug: Somatropin (DNA origin)
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 Not yet recruiting Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
Condition: Mucopolysaccharidosis I
Intervention: Drug: AGT-181 (HIRMAb-IDUA)
4 Recruiting Mucopolysaccharidosis I (MPS I) Registry
Condition: Mucopolysaccharidosis I (MPS I)
Intervention:
5 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
6 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
7 Recruiting Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
Conditions: Mucopolysaccharidosis I;   Cognitive Decline
Intervention: Drug: Intrathecal recombinant human alpha iduronidase
8 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
9 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)
10 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)
11 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)
12 Recruiting Longitudinal Studies of Brain Structure and Function in MPS Disorders
Conditions: Mucopolysaccharidosis Type I;   Mucopolysaccharidosis Type II;   Mucopolysaccharidosis Type VI
Intervention:
13 Recruiting Biomarker for Hurler Disease
Conditions: Mucopolysaccharidosis Type I;   MPS I;   Hurler-Scheie Syndrome;   Gargoylism, Hurler Syndrome
Intervention:
14 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:
15 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
16 Completed
Has Results
Immune Tolerance Study With Aldurazyme® (Laronidase)
Condition: Mucopolysaccharidosis I
Interventions: Biological: Laronidase;   Drug: Cyclosporine A (CsA);   Drug: Azathioprine (Aza)
17 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
18 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
19 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
20 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:

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