Status | Study |
Not yet recruiting |
Study Name: Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III Condition: Mucopolysaccharidosis Type I Mucopolysaccharidosis Type II Date: 2017-04-24 Interventions: Other: This is an observational study This is an observational study |
Recruiting |
Study Name: Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA Condition: Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome Date: 2016-03-17 Interventions: Biological: scAAV9.U1a.hSGSH Self-complementary adeno-associated virus serotype 9 carrying the human SGS |
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) Sanfilippo B Date: 2015-10-21 Interventions: Drug: SBC-103 |
Recruiting |
Study Name: A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) Condition: Mucopolysaccharidosis Type IIIB Mucopolysaccharidosis Type 3 B Date: 2015-06-22 |
Enrolling by invitation |
Study Name: An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. Condition: Sanfilippo Syndrome Mucopolysaccharidosis (MPS) Date: 2015-01-21 Interventions: Drug: HGT-1410 HGT-1410 administered according to Patient Group assignment. |
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 Mucopolysa Date: 2014-10-23 |