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Trial · NCT04884815

Registered outcomes19

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

ListOutcomeTime frameDescription
Primary
Stage 1: Incidence of Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Adverse Events of Special Interest (AESIs), Treatment-Related TEAEs, and Treatment-Related TESAEs
Up to Week 52
PrimaryStage 1: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52Baseline, Week 52
PrimaryStage 1: Change in Total Copper from Baseline at Week 52Baseline, Week 52
PrimaryStage 1: Change in Ceruloplasmin-bound Copper from Baseline at Week 52Baseline, Week 52
PrimaryStage 1: Change in Ceruloplasmin from Baseline at Week 52Baseline, Week 52
PrimaryStage 1: Change in Non-Ceruloplasmin-bound Copper (NCC) from Baseline at Week 52Baseline, Week 52
PrimaryStage 1: Change in Free Copper from Baseline at Week 52Baseline, Week 52
PrimaryStage 1: Change in Ceruloplasmin Activity from Baseline at Week 52Baseline, Week 52
PrimaryStage 1: Percent Reduction in Standard of Care (SOC) Medication by Week 52Week 52
PrimaryStage 1: Number of Participants Who Discontinue SOC Medication by Week 52Week 52
PrimaryStage 1: Number of Consecutive Weeks off SOC Medication at Week 52Week 52
PrimaryStage 2: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52, Evaluated for SuperiorityBaseline, Week 52
PrimaryStage 2: Percent Reduction in SOC Medication by Week 52, Evaluated for SuperiorityWeek 52
SecondaryStage 2: Change in Ceruloplasmin Activity Levels from Baseline at Week 52, Evaluated for SuperiorityBaseline, Week 52
SecondaryStage 2: Number of Participants who Discontinue SOC Medication by Week 52Week 52
SecondaryStage 2: Change in FACIT-Fatigue Scale Score from Baseline at Week 52Baseline, Week 52
SecondaryStage 2: Change in Liver Copper Concentration Assessed by Liver Biopsy from Baseline at Week 52Baseline, Week 52
OtherStage 2: Development of Anti-ATP7B AntibodiesUp to Week 104
OtherStage 2: Incidence of TEAEs, TESAEs, AESIs, Treatment-Related TEAEs, and Treatment-Related TESAEsUp to Week 312

Source ClinicalTrials.govtrials-2026-11-12-0.5.0

Sources 3

  • ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
  • ClinicalTrials.govNo license stated
  • NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
  • ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
  • ClinicalTrials.govnone stated
  • NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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