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Home › Conditions › Wilson Disease

Wilson Disease

Part of Movement Disorders

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for Wilson Disease

9 trials tracked for Wilson Disease: 1 in phase 3 or 4 and 1 with published results. The most-cited published study has 73 citations.

9Trials tracked
1Phase 3 & 4
0Recruiting
1With published results
Phase distribution
Phase 3 1 Phase 2 3 Phase 1 3 Other / NA 2
  1. Phase 3 Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease Completed · 73 cited
  2. Phase 2 Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840 Completed
  3. Phase 2 Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 Completed
  4. Phase 2 Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients Completed
  5. Phase 1 Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants Completed
  6. Phase 1 Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants Completed
Show 3 more trials
  1. Phase 1 Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants. Completed
  2. N/A Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease Completed
  3. N/A The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment Completed

Showing the 9 most-cited and recently-updated of 9 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Wilson Disease: what the trials found

Clinical evidence confirms the efficacy of Penicillamine in managing Wilson Disease over a 12-week period 1. Additionally, ALXN1840 has been evaluated across multiple Phase 1 and Phase 2 trials, demonstrating safety and tolerability profiles in various study cohorts [2, 5, 6, 7].

In clinical evaluations of ALXN1840, 85.7% of participants achieved normalized concentrations of non-ceruloplasmin-bound copper (NCC) 4. Furthermore, treatment with ALXN1840 was associated with improvements in neurological status as measured by the UWDRS (subscores and total score) and psychiatric status dimensions using the M.I.N.I. at week 24 4.

Recent results — preliminary, needs further review

  • Bis-Choline Tetrathiomolybdate showed a change from baseline in liver copper concentration at week 48, though the result did not reach statistical significance (p=0.1002) 3.
  • Standard of care medications were evaluated for NCC normalization or reduction; 51.6% of participants achieved these targets at 6 months, increasing to 62.5% through the last assessment 8.
  • Once-a-day Trientine was assessed in a small cohort (N=8) 9.

For the clinician treating this condition

  • Penicillamine is an established intervention for Wilson Disease 1.
  • ALXN1840 demonstrates significant efficacy in normalizing non-ceruloplasmin-bound copper (NCC) and improving neurological and psychiatric scores 4.
  • Multiple trials confirm the safety profile of ALXN1840 across various phases of clinical investigation [2, 5, 6, 7].

AI synthesis of 5 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
NODDI MRI shows phenotype-specific white matter alterations in Wilson disease patients
Neurology
NODDI MRI shows phenotype-specific white matter alterations in Wilson disease patients What does Wilson disease do to the brain's wiring? A new scan shows specific damage
An observational study of 30 Wilson disease patients and 30 matched controls found phenotype-specific microstructural white matter alteratio…
A new brain scan reveals specific wiring damage in Wilson disease patients, linking reduced nerve fiber density directly to slower processin…
medRxiv Apr 5, 2026

Research across Movement Disorders

Related studies from across the Movement Disorders family.

Quantitative susceptibility mapping shows higher deep gray matter alterations in Wilson's disease compared to healthy controls
Gastroenterology Meta-analysis
Quantitative susceptibility mapping shows higher deep gray matter alterations in Wilson's disease compared to healthy controls New scan findings show brain changes in Wilson's disease patients compared to healthy people
This meta-analysis evaluated quantitative susceptibility mapping (QSM) alterations in deep gray matter among 325 patients with Wilson's dise…
A new scan technique reveals specific brain changes in Wilson's disease patients, showing higher metal buildup in key areas compared to heal…
Apr 14, 2026