Status | Study |
Recruiting |
Study Name: A Study of JR-141 in Patients With Mucopolysaccharidosis Type II Condition: Mucopolysaccharidosis II Date: 2017-04-10 Interventions: Drug: JR-141 IV infusion (lyophilized powder), 0.01-2.0 mg/kg/week |
Enrolling by invitation |
Study Name: Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I Condition: Mucopolysaccharidosis I Date: 2017-02-27 Interventions: Drug: AGT-181 Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion |
Active, not recruiting |
Study Name: Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I Condition: Mucopolysaccharidosis I Date: 2017-02-09 Interventions: Drug: AGT-181 Human Insulin Receptor Monoclonal Antibody-Human alpha-L-iduronidase (HIRMAb-IDUA) Fusion |
Recruiting |
Study Name: Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II Condition: Mucopolysaccharidosis II Date: 2017-01-13 Interventions: Biological: SB-913 Single dose of each of the 3 components of SB-913: ZFN1, ZFN2 and hIDS Donor |
Not yet recruiting |
Study Name: Mucopolysaccharidosis (MPS) Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia Combined With Pediatric ENT Surgery or Bronchoscopy Condition: Mucopolysaccharidoses Date: 2017-01-09 |
Recruiting |
Study Name: Natural History Study of Patients With MPS IIIA Condition: Mucopolysaccharidosis IIIA Date: 2016-04-15 |
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 |
Not yet recruiting |
Study Name: Study of Idursulfase-beta (GC1111) in Hunter Syndrome Condition: Mucopolysaccharidosis II Date: 2016-01-17 Interventions: Biological: idursulfase beta I |
Recruiting |
Study Name: Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI Condition: Mucopolysaccharidosis Type I Mucopolysacc Date: 2015-05-05 Interventions: Drug: Adalimumab Subjects will |
Active, not recruiting |
Study Name: Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I Condition: Mucopolysaccharidosis I Date: 2015-02-19 Interventions: Drug: AGT-181 (HIRMAb-IDUA) intravenous infusion over 3-4 hours |