Treatment Trials

3 Clinical Trials for Various Conditions

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      COMPLETED
      Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
      Description

      To evaluate the safety and efficacy of ORGN001(formerly ALXN1101) in neonate patients with MoCD Type A

      Conditions
      Molybdenum Cofactor Deficiency, Type A
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      COMPLETED
      Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers
      Description

      Phase 1 single dose study of ALXN1101 in healthy volunteers.

      Conditions
      Molybdenum Cofactor Deficiency (MoCD)Rare Autosomal Recessive DisorderDeficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
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      COMPLETED
      Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
      Description

      This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.

      Conditions
      Molybdenum Cofactor Deficiency, Type A
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