Status | Study |
Not yet recruiting |
Study Name: Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I and II Condition: Mucopolysaccharidosis I Mucopolysaccharidosis II Date: 2017-05-11 Interventions: Drug: Adalimumab Injection [Humira] |
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 |
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-318 in Subjects With MPS I Condition: MPS I Date: 2016-02-29 Interventions: Biological: SB-318 A single dose of each of the three components of SB-318 [zinc finger nucleases (ZFN1, |
Enrolling by invitation |
Study Name: Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181 Condition: Mucopolysaccharidosis I Date: 2015-11-03 Interventions: Drug: AGT-181 intravenous infusion over 3-4 hours Ot |
Recruiting |
Study Name: Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI Condition: Mucopolysaccharidosis Type I Mucopolysaccharidosis Type II 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 |
Enrolling by invitation |
Study Name: Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I Condition: Mucopolysaccharidosis I Cognitive Decline Date: 2014-09-03 Interventions: Drug: Intrathecal recombinant human alpha iduronidase Intrathecal recombinant human alpha iduronidase ev |
Terminated |
Study Name: Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics Condition: Mucopolysaccharidoses Mucopolysaccharidosis I Date: 2012-08-23 Interventions: Other: Dried blood spot test for MPS The dried blood spot test uses a few drops of blood on filter paper |
Completed |
Study Name: Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I Condition: Mucopolysaccharidosis I Hurlers Syndrome Date: 2009-06-02 Interventions: Biological: rhIDU (recombinant human-Alpha-L-Iduronidase) |