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
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106 studies found for:    Mucopolysaccharidoses

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1 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
2 Terminated Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
Condition: Mucopolysaccharidosis Type IVA
Intervention:
3 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:
4 Terminated A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Conditions: MPS IV A;   Mucopolysaccharidosis IVA;   Morquio A Syndrome
Intervention: Drug: BMN 110
5 Unknown † Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis
Condition: Mucopolysaccharidosis
Interventions: Drug: Conditioning regimen;   Drug: Graft-versus-host disease (GVHD) prophylaxis;   Procedure: Stem Cell Transplantation
6 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
7 Active, not recruiting Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Conditions: Mucopolysaccharidosis IV A;   Morquio A Syndrome;   MPS IVA
Interventions: Drug: BMN 110 - Weekly;   Drug: BMN 110 - Every Other Week
8 Terminated A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Conditions: MPS IV A;   Mucopolysaccharidosis IVA;   Morquio A Syndrome
Intervention:
9 Recruiting Natural History Studies of Mucopolysaccharidosis III
Conditions: Mucopolysaccharidosis Type IIIA;   Mucopolysaccharidosis Type IIIB
Interventions: Procedure: Lumbar puncture;   Device: Magnetic Resonance Imaging (MRI) of the brain
10 Recruiting Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
Conditions: Mucopolysaccharidosis IV A;   Mucopolysaccharidosis VI
Intervention: Other: Enzyme testing
11 Suspended Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa
Conditions: Mucopolysaccharidosis IV;   Morquio A Disease
Intervention:
12 Active, not recruiting Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Conditions: Mucopolysaccharidosis IVA;   Morquio A Syndrome;   MPS IVA
Intervention: Drug: BMN 110
13 Recruiting Mucopolysaccharidosis I (MPS I) Registry
Condition: Mucopolysaccharidosis I (MPS I)
Intervention:
14 Completed
Has Results 		Study of rhASB in Patients With Mucopolysaccharidosis VI
Condition: Mucopolysaccharidosis VI
Interventions: Drug: N-acetylgalactosamine 4-sulfatase;   Drug: Placebo/rhASB
15 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)
16 Completed Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
Condition: Mucopolysaccharidosis II
Intervention: Genetic: lymphocyte gene therapy
17 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
18 Completed
Has Results 		A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
Condition: MPS IV A
Intervention: Drug: BMN 110
19 Completed
Has Results 		A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Condition: MPS IV A
Interventions: Drug: BMN 110 Weekly;   Drug: Placebo;   Drug: BMN 110 Every Other Week
20 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
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† Indicates status has not been verified in more than two years