Status | Study |
Completed |
Study Name: Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging Condition: MPS IIIB (Sanfilippo B Syndrome) Date: 2014-03-12 |
Completed |
Study Name: Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease Condition: Sanfilippo Syndrome Date: 2014-02-10 Interventions: Drug: Recombinant human heparan N-sulfatase [rhHNS] Recombinant human heparan N-sulfatase [rhHNS] |
Active, not recruiting |
Study Name: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy Condition: Mucopolysaccharidosis Type III A Sanfilip Date: 2014-01-16 Interventions: Genetic: SAF-301 |
Completed |
Study Name: Natural History Studies of Mucopolysaccharidosis III Condition: Mucopolysaccharidosis Type IIIA Mucopolysaccharidosis Type IIIB Date: 2013-11-26 Interventions: Procedure: Lumbar puncture Device: Magneti |
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 Syn Date: 2013-05-28 |
Completed |
Study Name: Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders Condition: Lysosomal Storage Disease Peroxisomal Disorder Date: 2012-06-20 Interventions: Drug: Campath-1H A daily dose |
Completed |
Study Name: Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Condition: Sanfilippo Syndrome Type B Date: 2012-01-10 |
Completed |
Study Name: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome Condition: Mucopolysaccharidosis Type III A Sanfilip 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 |