Status | Study |
Enrolling by invitation |
Study Name: Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181 Condition: Mucopolysaccharidosis I Date: 2015-11-03 Interventions: Drug: AGT-181 intravenous infusion over 3-4 hours Ot |
Recruiting |
Study Name: Biomarker for Patients With Hurler Disease or High-grade Suspicion for Hurler Disease Condition: Mucopolysaccharidosis Type I MPS I Hurler- Date: 2014-10-23 |
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 |
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 Hurler- 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 Hurler- 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 Hurler- 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 |