Status | Study |
Recruiting |
Study Name: Biomarker for Patients With Hurler Disease or High-grade Suspicion for Hurler Disease Condition: Mucopolysaccharidosis Type I MPS I H Date: 2014-10-23 |
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 |
Recruiting |
Study Name: Longitudinal Studies of Brain Structure and Function in MPS Disorders Condition: Mucopolysaccharidosis Type I Mucopolysaccharidosis Type II Date: 2011-08-02 |
Active, not recruiting |
Study Name: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders Condition: Hurler Syndrome (MPS I) Hurler-Scheie Syndrome With Early Neuro Date: 2011-06-10 Interventions: Biological: FCRx infusion Enriched hematopoetic stem cell infusion |
Completed |
Study Name: Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I Condition: Mucopolysaccharidosis I Hurlers Syndrome Date: 2009-06-02 Interventions: Biological: rhIDU (recombinant human-Alpha-L-Iduronidase) |
Completed |
Study Name: A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I Condition: Cognitive Decline Mucopolysaccharidosis I Date: 2009-02-25 Interventions: Drug: laronidase For the treatment group, intrathecal rhIDU injections will consist of 3 cc of Aldurazym |
Terminated |
Study Name: Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I Condition: Spinal Cord Compression Mucopolysaccharidosis I Date: 2008-06-17 Interventions: Drug: laronidase 1.74 mg intrathecally every 1-3 months for 1 year |
Terminated |
Study Name: ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases Condition: Inherited Metabolic Diseases Lysosomal Storage Disorders P Date: 2008-04-03 Interventions: Biological: ALD-101 A subpopulation of cord blood cells composed of cells that express a high level of t |
Recruiting |
Study Name: A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants Condition: Mucopolysaccharidosis I Hurler's Syndrome Date: 2007-01-03 Interventions: Biological: Aldurazyme (laronidase) dose of 0.58mg/kg body weight intravenously (IV) every week |
Completed |
Study Name: Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease Condition: Mucopolysaccharidosis I Hurler Syndrome Date: 2005-11-22 Interventions: Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) 0.58 mg/kg every week |