Status | Study |
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 |
Not yet recruiting |
Study Name: Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD) Condition: Inherited Metabolic Disorders IMD Date: 2016-03-16 Interventions: Drug: Umbilical cord blood transplantation with HSC835 Hematopoietic Stem cell transplantation will be d |
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 |
Recruiting |
Study Name: Biomarker for Patients With Hurler Disease or High-grade Suspicion for Hurler Disease Condition: Mucopolysaccharidosis Type I MPS I Hurler-Scheie Syndrome Date: 2014-10-23 |
Recruiting |
Study Name: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Condition: Mucopolysaccharidosis Disorders Hurler Syndrome Date: 2014-06-20 Interventions: Biological: Stem Cell Transplantation |
Recruiting |
Study Name: BMT Abatacept for Non-Malignant Diseases Condition: Hurler Syndrome Fanconi Anemia Date: 2013-07-24 Interventions: Drug: Abatacept All patients will receive 4 doses of abatacept in addition to standard GVHD prophylaxis |
Completed |
Study Name: Neurobehavioral Phenotypes in MPS III Condition: Sanfilippo Syndrome Type A Sanfilippo Syndrome Type B Date: 2013-05-28 |
Recruiting |
Study Name: Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome Condition: Mucopolysaccharidosis Type IH MPS I Date: 2012-04-04 Interventions: Drug: Laronidase Administered 0.58 mg/kg/dose intravenously (IV) once a week beginning 12 weeks before p |
Active, not recruiting |
Study Name: Administration of IV Laronidase Post Bone Marrow Transplant in Hurler Condition: Hurler Syndrome Date: 2010-07-28 Interventions: Drug: Laronidase Laronidase 0.58 mg/kg intravenously (IV) once a week for a maximum of 2 years |