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Metal Metabolism, Inborn Errors - 25 Studies Found

Status Study
Recruiting Study Name: Study of ALXN1101 in Neonates With Molybdenum Cofactor Deficiency (MOCD) Type A
Condition: Molybdenum Cofactor Deficiency, Type A
Date: 2015-12-03
Interventions: Drug: ALXN1101
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
  • 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 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
Withdrawn Study Name: Study of cPMP (Precusor Z) to Treat Molybdenum Cofactor Deficiency (MoCD) Type A
Condition: Molybdenum Cofactor Deficiency Type A
Date: 2009-07-10
Interventions: Drug: cPMP Intravenous solution administered daily. Dose titrated from 80 μg/kg on Days 1-12 to 120
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
Recruiting Study Name: Biomarker for Hypophosphatasia Disease
Condition:
  • HEREDITARY DISEASES
  • METABOLISM, INBORN ERRORS
  • Hypophosphat
    Date: 2015-11-10
Active, not recruiting Study Name: Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
Condition: Hypophosphatasia
Date: 2016-05-23
Interventions: Drug: Asfotase alfa Other Name: Strensiq
Not yet recruiting Study Name: Characterisation of Adult-Onset Hypophosphatasia
Condition: Hypophosphatasia
Date: 2016-05-04
Interventions: Other: no intervention - observational study
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