Status | Study |
Recruiting |
Study Name: Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI Condition: Mucopolysaccharidosis Type I Mucopolysaccharidosis Type II Date: 2015-05-05 Interventions: Drug: Adalimumab Subjects will |
Recruiting |
Study Name: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Condition: Mucopolysaccharidosis Disorders Hurler Syndrome Hunter Syn Date: 2014-06-20 Interventions: Biological: Stem Cell Transplantation |
Recruiting |
Study Name: Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome Condition: Maroteaux-Lamy Syndrome Date: 2014-06-03 Interventions: Drug: Naglazyme® 1 mg per kg of body weight administered once weekly as an intravenous infusion |
Terminated |
Study Name: 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) Condition: Mucopolysaccharidosis Mucopolysaccharidosis I Mucopolysacc Date: 2014-03-20 |
Completed |
Study Name: Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome Condition: Morquio Syndrome A Maroteaux Lamy Syndrome Date: 2013-10-08 |
Recruiting |
Study Name: Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children Condition: Mucopolysaccharidosis Type I (MPS I) Mucopolysaccharidosis Type II (MPS II) Date: 2013-08-06 |
Completed |
Study Name: Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease Condition: Mucopolysaccharidosis IV A Mucopolysaccharidosis VI Date: 2012-10-12 Interventions: Other: Enzyme testing Leukocyte activity measurement of Arylsulfatase B and N acetyl galactosamine 6 sul |
Terminated |
Study Name: Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics Condition: Mucopolysaccharidoses Mucopolysaccharidosis I Mucopolysacc Date: 2012-08-23 Interventions: Other: Dried blood spot test for MPS The dried blood spot test uses a few drops of blood on filter paper |
Active, not recruiting |
Study Name: Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network Condition: MPS I MPS II MPS VI Date: 2012-04-25 |
Recruiting |
Study Name: Human Placental-Derived Stem Cell Transplantation Condition: Mucopolysaccharidosis I Mucopolysaccharidosis VI Date: 2012-04-25 Interventions: Drug: Human Placental Derived Stem Cell Infusions of thawed HPDSC to be given following UCB infusion. |