Status | Study |
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: Neurobehavioral Phenotypes in MPS III Condition: Sanfilippo Syndrome Type A Sanfilippo Syndrome Type B Date: 2013-05-28 |
Completed |
Study Name: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome Condition: Mucopolysaccharidosis Type III A Sanfilippo Disease Type A Date: 2011-11-10 Interventions: Genetic: SAF-301 The treatment plan consists on a direct injection of the investigational medicinal prod |
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 Neurologic Involvement and/or Sens Date: 2011-06-10 Interventions: Biological: FCRx infusion Enriched hematopoetic stem cell infusion |
Active, not recruiting |
Study Name: Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) Condition: Sanfilippo Syndrome Date: 2011-02-16 Interventions: Biological: rhHNS-10 mg Once p |
Completed |
Study Name: Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients Condition: Sanfilippo Syndrome Type A (MPS IIIA) Date: 2010-06-24 Interventions: Biological: Recombinant human heparan N-sulfatase |
Completed |
Study Name: A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA) Condition: Sanfilippo Syndrome Type A Date: 2010-01-11 Interventions: Other: assessment Physical, developmental, neurological, behavioral, and neurocognitive assessments |
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 |
Completed |
Study Name: HSCT for High Risk Inherited Inborn Errors Condition: Adrenoleukodystrophy Metachromatic Leukodystrophy Globoid Date: 2006-09-29 Interventions: Drug: Clofarabine days -7 thro |