Status | Study |
Recruiting |
Study Name: Natural History Study of Patients With Hypophosphatasia (HPP) Condition: Hypophosphatasia Date: 2014-09-09 |
Completed |
Study Name: Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP) Condition: Hypophosphatasia (HPP) Date: 2014-03-25 |
Active, not recruiting |
Study Name: Safety & Efficacy Study of ALXN1101 in Pediatric Patients With MoCD Type A Currently Treated With rcPMP Condition: Molybdenum Cofactor Deficiency, Type A Date: 2014-01-09 Interventions: Drug: ALXN1101 IV infusion |
Completed |
Study Name: Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers Condition: Molybdenum Cofactor Deficiency (MoCD) Rare Autosomal Recessive Disorder Date: 2013-06-27 Interventions: Drug: ALXN1101 Randomized to r |
Recruiting |
Study Name: Apotransferrin in Atransferrinemia Condition: Congenital Atransferrinemia Date: 2013-02-20 Interventions: Drug: Human apotransferrin intravenous infusion |
Completed |
Study Name: A Natural History Study of Molybdenum Cofactor and Isolated Sulfite Oxidase Deficiencies Condition: Molybdenum Cofactor Deficiency Isolated Sulfite Oxidase Deficiency Date: 2012-11-26 |
Completed |
Study Name: A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP) Condition: Molybdenum Cofactor Deficiency Date: 2012-07-12 |
Completed |
Study Name: A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP) Condition: Hypophosphatasia (HPP) Date: 2011-08-16 |
Completed |
Study Name: Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP) Condition: Hypophosphatasia Date: 2011-07-29 Interventions: Drug: BPS804 |
Completed |
Study Name: Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP) Condition: Hypophosphatasia Date: 2010-09-17 Interventions: Biological: asfotase alfa Other Name: ENB-0040 |