Metal Metabolism, Inborn Errors - 25 Studies Found
Recruiting |
: Study of ALXN1101 in Neonates With Molybdenum Cofactor Deficiency (MOCD) Type A : Molybdenum Cofactor Deficiency, Type A : 2015-12-03 : Drug: ALXN1101 |
Active, not recruiting |
: Safety & Efficacy Study of ALXN1101 in Pediatric Patients With MoCD Type A Currently Treated With rcPMP : Molybdenum Cofactor Deficiency, Type A : 2014-01-09 : Drug: ALXN1101 IV infusion |
Completed |
: Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers :
|
Recruiting |
: Apotransferrin in Atransferrinemia : Congenital Atransferrinemia : 2013-02-20 : Drug: Human apotransferrin intravenous infusion |
Completed |
: A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP) : Molybdenum Cofactor Deficiency : 2012-07-12 |
Withdrawn |
: Study of cPMP (Precusor Z) to Treat Molybdenum Cofactor Deficiency (MoCD) Type A : Molybdenum Cofactor Deficiency Type A : 2009-07-10 : Drug: cPMP Intravenous solution administered daily. Dose titrated from 80 μg/kg on Days 1-12 to 120 |
Completed |
: A Natural History Study of Molybdenum Cofactor and Isolated Sulfite Oxidase Deficiencies :
: 2012-11-26 |
Recruiting |
: Biomarker for Hypophosphatasia Disease :
|
Active, not recruiting |
: Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP) : Hypophosphatasia : 2016-05-23 : Drug: Asfotase alfa Other Name: Strensiq |
Not yet recruiting |
: Characterisation of Adult-Onset Hypophosphatasia : Hypophosphatasia : 2016-05-04 : Other: no intervention - observational study |