Disease · MONDO_0010200
Wilson disease
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. Wilson disease. Diseases, record MONDO_0010200. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010200
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Highest stage of its drugs
- Approval, for 6 of its 15 drugs, from DailyMed, EMA Human Drugs, PMDA and TTD
- Open Targets
Drugs15
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| TRIENTINE | CHEMBL609 | Small molecule | Approval | EMA Human Drugs | 8 | 7 | 7 | Drugs and targets |
| PENICILLAMINE | CHEMBL1430 | Small molecule | Approval | DailyMed | 5 | 3 | 3 | Drugs and targets |
| ZINC ACETATE | CHEMBL3184986 | Small molecule | Approval | EMA Human Drugs, PMDA and TTD | 6 | 2 | 1 | Drugs and targets |
| TRIENTINE TETRAHYDROCHLORIDE | CHEMBL5095420 | Small molecule | Approval | DailyMed | 4 | 2 | 2 | Drugs and targets |
| ZINC SULFATE | CHEMBL1200929 | Small molecule | Approval | EMA Human Drugs | 2 | 1 | 0 | Drugs and targets |
| TRIENTINE HYDROCHLORIDE | CHEMBL3989777 | Small molecule | Approval | EMA Human Drugs and TTD | 2 | 0 | 0 | Drugs and targets |
| TIOMOLIBDATE CHOLINE | CHEMBL4594259 | Small molecule | Phase 3 | ClinicalTrials.gov | 8 | 6 | 6 | Drugs and targets |
| TIOMOLIBDATE DIAMMONIUM | CHEMBL3775741 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| ZINC ACETATE ANHYDROUS | CHEMBL1200928 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| NIMATPAGENE PARIPARVOVEC | CHEMBL5314853 | Gene | Phase 2 | TTD | 1 | 0 | 0 | Drugs and targets |
1 to 10 of 15
See all 15 drugsTargets1
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes |
|---|---|---|---|---|---|
| SNAP25 | Synaptosomal-associated protein 25 | 1 | Synaptosomal nerve-associated protein 25 (SNAP-25) hydrolytic enzyme | Unknown | https://conductscience.org/library/genes/6616 |
Source Drugs and targets
Trials20
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT03403205 | Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease | Phase 3 | 214 | 2023-10-10 | Wilson Disease | TIOMOLIBDATE CHOLINE | Its record names the disease | ClinicalTrials.gov |
| NCT03539952 | Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease | Phase 3 | 77 | 2023-04-20 | Wilson Disease | TRIENTINE TETRAHYDROCHLORIDE | Its record names the disease | ClinicalTrials.gov |
| NCT02763215 | The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment | 64 | 2020-10-26 | Wilson Disease | COPPER, PENICILLAMINE, TRIENTINE | Its record names the disease | ClinicalTrials.gov | |
| NCT05047523 | Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease | Phase 3 | 40 | 2024-10-15 | Wilson Disease | TIOMOLIBDATE CHOLINE | Its record names the disease | ClinicalTrials.gov |
| NCT00212355 | Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson's Disease in Japan. | Phase 3 | 37 | 2014-05-14 | Wilson's Disease | ZINC ACETATE | Its record names the disease | ClinicalTrials.gov |
| NCT04422431 | Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840 | Phase 2 | 31 | 2023-07-03 | Wilson Disease | TIOMOLIBDATE CHOLINE | Its record names the disease | ClinicalTrials.gov |
| NCT02273596 | Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients | Phase 2 | 29 | 2021-09-29 | Wilson Disease | TIOMOLIBDATE CHOLINE | Its record names the disease | ClinicalTrials.gov |
| NCT04573309 | Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 | Phase 2 | 9 | 2023-10-27 | Wilson Disease | TIOMOLIBDATE CHOLINE | Its record names the disease | ClinicalTrials.gov |
| NCT01472874 | Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease | Not applicable | 8 | 2014-05-19 | Wilson Disease | TRIENTINE | Its record names the disease | ClinicalTrials.gov |
| NCT03957720 | The Individual Therapy for Patients With Wilson's Disease | Early phase 1 | 400 | Wilson's Disease | PENICILLAMINE, ZINC GLUCONATE | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 20
See all 20 trialsComparisons in its trials4
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Treatment Period: ALXN1840 vs Extension Period: ALXN1840: deaths from any cause | 0 of 31 | 0 of 25 | 56 | NCT04422431 | Trial comparisons |
| Treatment Period: ALXN1840 vs Extension Period: ALXN1840: serious adverse events | 3 of 31 | 2 of 25 | 56 | NCT04422431 | Trial comparisons |
| Cohort 1 vs Cohort 2: deaths from any cause | 0 of 8 | 0 of 1 | 9 | NCT04573309 | Trial comparisons |
| Cohort 1 vs Cohort 2: serious adverse events | 0 of 8 | 0 of 1 | 9 | NCT04573309 | Trial comparisons |
Papers from its trials8
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
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
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0010200
GET https://conductscience.org/api/diseases/v1/MONDO_0010200/drugs