Status | Study |
Recruiting |
Study Name: A Treatment Study of Mucopolysaccharidosis Type IIIB Condition: MPS III B Mucopolysaccharidosis Type IIIB Date: 2016-02-27 Interventions: Drug: BMN 250 Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human ins |
Active, not recruiting |
Study Name: A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously Condition: Mucopolysaccharidosis III, Type B (MPS IIIB) Date: 2015-10-21 Interventions: Drug: SBC-103 |
Recruiting |
Study Name: A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) Condition: Mucopolysaccharidosis Type IIIB Mucopolys Date: 2015-06-22 |
Active, not recruiting |
Study Name: Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in MPS IIIB Condition: Mucopolysaccharidosis IIIB Date: 2014-12-15 Interventions: Drug: SBC-103 Other Name: recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) |
Completed |
Study Name: A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB Condition: MPS IIIB (Sanfilippo Syndrome) Date: 2014-11-03 |
Active, not recruiting |
Study Name: Natural History Study to Characterise the Course of Disease Progression in Patients With Mucopolysaccharidosis Type IIIB Condition: MPS IIIB (Sanfilippo B Syndrome) Date: 2014-11-03 |
Recruiting |
Study Name: Biomarker for Sanfilippo Disease Condition: Mucopolysaccharidosis Type 3 A Mucopolysaccharidosis Type 3 B Date: 2014-10-23 |
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: Natural History Studies of Mucopolysaccharidosis III Condition: Mucopolysaccharidosis Type IIIA Mucopolysaccharidosis Type IIIB Date: 2013-11-26 Interventions: Procedure: Lumbar puncture Device: Magneti |
Completed |
Study Name: Neurobehavioral Phenotypes in MPS III Condition: Sanfilippo Syndrome Type A Sanfilippo Syndrome Type B Date: 2013-05-28 |