Clinical Trial Search

Primary: Change From Baseline in Liver Cu Concentration at Week 48 (Treatment Period) — 92.8 μg/g — p=0.1002

Primary: Mean Daily Copper Balance: Day 1 Through Day 8 — 0.8025; 0.3062 milligrams/day

Primary: Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of Non-ceruloplasmin-bound Copper (NCC) Or Reached A Reduction Of ≥ 25% In NCC During 6…

Primary: Serum NCC Concentration — 46.5; 58.7 µg/L

Primary: Percentage Of Participants With Normalized Concentrations Of NCC — 85.7 percentage of participants

Primary: Safety and Efficacy — 37 participants

Primary: ALT — 41.38 U/L

Primary: Gross Motor Development at 36 Mos of Age or at Death (Mos) — 13.743; 2.455; 15.667 Other - months

Primary: Number of Participants With Serious Adverse Events — 0; 0; 0 Participants

Primary: Liver Stiffness as Measured by Shear Wave Elastography — 13; 14 Participants

Primary: Number of Participants With Adverse Events — 22; 42; 12; 14 Participants

Primary: Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population) — -389.1; -610.5; -571.6 µg/L

Primary: Change From Baseline in Mean Daily Copper Balance Over 2 Weeks of Repeated Daily ALXN1840 Dosing (Over Days 4 to 15) — -0.0749; 0.6901; 0.4201 mg/day

Primary: Chloriuretic Response to a Thiaizde — 1.23 fractional chloride excretion

Primary: Number of Participants Showing Reduction or Elimination of Skin Blistering — 0; 8 participants

Primary: Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests — 0; 0; 0; 2 Participants

Primary: Number of Participants Who Show Stability, Improvement or Decline in Self Reported Hearing — 13; 18; 0 Participants

Primary: Number of Treatment-emergent Adverse Events Assessed Using CTCAE v4.0 — 1.15; 1.38; 1.14; 0.86 Events per person — p=0.50

Primary: Effect of PTG-300 on Transferrin Saturation — -6.07 percent saturation