Mucopolysaccharidoses - 25 Studies Found
Completed |
: Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome) : Mucopolysaccharidosis II : 1999-10-18 : Genetic: lymphocyte gene therapy |
Recruiting |
: Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI :
|
Recruiting |
: A Treatment Study of Mucopolysaccharidosis Type IIIB :
: 2016-02-27 : Drug: BMN 250 Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human ins |
Active, not recruiting |
: Longitudinal Study of Bone Disease in Children With Mucopolysaccharidoses (MPS) I, II, and VI : Mucopolysaccharidoses : 2011-11-11 |
Completed |
: Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) : Mucopolysaccharidosis II : 2003-09-29 :
|
Recruiting |
: Natural History Study of Patients With MPS IIIA : Mucopolysaccharidosis IIIA : 2016-04-15 |
No longer available |
: An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient : Mucopolysaccharidosis Type 7 : 2014-03-24 : Drug: UX003 Open Label Other Names: |
Enrolling by invitation |
: Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I : Mucopolysaccharidosis I : 2017-02-27 : Drug: AGT-181 Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion |
Active, not recruiting |
: Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I : Mucopolysaccharidosis I : 2017-02-09 : Drug: AGT-181 Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion |
Recruiting |
: Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II : Mucopolysaccharidosis II : 2017-01-13 : Biological: SB-913 Single dose of each of the 3 components of SB-913: ZFN1, ZFN2 and hIDS Donor |