Hepatolenticular Degeneration clinical trials
The registry lists 80 studies for Hepatolenticular Degeneration across more than 10 countries. 31 are recruiting or about to start. 71 of them are mainly about Hepatolenticular Degeneration (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-06.
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Who runs them
Alexion Pharmaceuticals, Inc. (11) · Fondation Ophtalmologique Adolphe de Rothschild (7) · Orphalan (6) · Assiut University (5) · Yale University (3) · Second Affiliated Hospital, School of Medicine, Zhejiang University (3) · Ultragenyx Pharmaceutical Inc (3) · Assistance Publique - Hôpitaux de Paris (2)
What is being tested here
Drugs under test in Hepatolenticular Degeneration studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 9
- Nov 2026Multifaceted Assessment of Patients With Wilson's Disease in a Low-Resource Setting in Upper Egypt: Service Integration, Psychosocial Burden, Dietary Practices, and the Geo-Spatial Disease Map · Assiut University
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Dec 2026A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease · Chaohui Yu
- Dec 2026Daily Versus Alternate Day Plasma Exchange in Wilson Disease With Acute Liver Failure in Children · Institute of Liver and Biliary Sciences, India
- Feb 2027Circadian Variation of Urinary Copper Excretion in Wilson Disease Patients · Hospices Civils de Lyon
- Mar 2027Description of the Copper Concentration in Breast Milk in Women Treated for Wilson's Disease · Fondation Ophtalmologique Adolphe de Rothschild
- Apr 2027Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD) · GeneCradle Inc
- Aug 2027Oral Health and Wilson's Disease: SOMAWI · Fondation Ophtalmologique Adolphe de Rothschild
- Nov 2027Endocrine Dysfunction in Pediatric Wilson's Disease · Assiut University
Who these studies take
Of the 21 Hepatolenticular Degeneration studies recruiting now, 16 (76%) set no upper age limit and 4 (19%) stop at 75 or younger. 14 (67%) take children under 18. 1 (5%) are for women only and 0 (0%) for men only; 3 (14%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 10 (48%) | 10 (48%) |
| 18 | 21 (100%) | 20 (95%) |
| 30 | 21 (100%) | 20 (95%) |
| 40 | 21 (100%) | 20 (95%) |
| 50 | 21 (100%) | 20 (95%) |
| 65 | 20 (95%) | 19 (90%) |
| 75 | 17 (81%) | 16 (76%) |
| 85 | 17 (81%) | 16 (76%) |
Across all 80 studies, recruiting or not: 33% stop at 75 or younger and 28% at 65; 53% take children; 1% are for women only and 0% for men only; 15% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 50 of 80. Browse all 80 → or search them.
- Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.RecruitingNCT048803561 country
- Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.RecruitingNCT07465718Phase 34 countries
- Oral Health and Wilson's Disease: SOMAWIRecruitingNCT054441271 country
- Description of the Copper Concentration in Breast Milk in Women Treated for Wilson's DiseaseRecruitingNCT05183165Not applicable1 country
- Cardiac Involvement in Wilson's DiseaseRecruitingNCT05493605Not applicable1 country
- Description of Renal Involvement in Wilson's DiseaseRecruitingNCT070753931 country
- A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)RecruitingNCT07748403Phase 1/22 countries
- International Wilson's Disease Patient Registry (iWilson Registry)RecruitingNCT052398587 countries
- Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson's DiseaseNot yet recruitingNCT07641140Phase 1/21 country
- Natural History of Wilson DiseaseRecruitingNCT033342923 countries
- Off Treatment Urinary Copper Excretion in Wilson Disease, Pilot StudyRecruitingNCT073012161 country
- A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson DiseaseRecruitingNCT06650319Early Phase 11 country
- Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measuring Skin ElasticityRecruitingNCT06945081Not applicable1 country
- A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)RecruitingNCT07240896Early Phase 11 country
- Circadian Variation of Urinary Copper Excretion in Wilson Disease PatientsRecruitingNCT064303591 country
- Institutional Registry of Rare DiseasesRecruitingNCT065737231 country
- Multifaceted Assessment of Patients With Wilson's Disease in a Low-Resource Setting in Upper Egypt: Service Integration, Psychosocial Burden, Dietary Practices, and the Geo-Spatial Disease MapNot yet recruitingNCT07241832
- Endocrine Dysfunction in Pediatric Wilson's DiseaseNot yet recruitingNCT07208565
- Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)Not yet recruitingNCT07173933Phase 1/21 country
- Gene Therapy for Wilson Disease Evaluated by 64Cu PET/CTEnrolling by invitationNCT071595811 country
- French Wilson Disease RegistryRecruitingNCT052318761 country
- Daily Versus Alternate Day Plasma Exchange in Wilson Disease With Acute Liver Failure in ChildrenNot yet recruitingNCT06698991Not applicable1 country
- Spanish Wilson Disease RegistryRecruitingNCT064662911 country
- Clinical Profile and Out Come of Children With Wilson's DiseaseNot yet recruitingNCT05792319
- Gandouling in the Treatment of Wilson's DiseaseRecruitingNCT05305872Phase 41 country
- Clinical Value of DWI-ADC Matching in the Short-term Prognosis of Wilson's DiseaseNot yet recruitingNCT061969311 country
- Evaluation of Cognitive Dysfunction and Psychiatric ComorbiditiesNot yet recruitingNCT06179394
- Early Detection of Cardiac Affection in Patients of Wilson's DiseaseNot yet recruitingNCT06051734
- Role for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson DiseaseRecruitingNCT049655451 country
- A Registered Cohort Study on Wilson's DiseaseRecruitingNCT040126581 country
- China Registry for Genetic / Metabolic Liver DiseasesRecruitingNCT031314271 country
- A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson DiseaseActive, not recruitingNCT04884815Phase 1/24 countries
- Study Comparing Once Daily Dose of 900mg of TETA 4HCL Against Cuprior® (450mg Trientine Base, Twice Daily).CompletedNCT06128954Phase 11 country
- Subclinical Myocardial Dysfunction in Children With Wilson's DiseaseActive, not recruitingNCT077654721 country
- An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson DiseaseActive, not recruitingNCT06663878Not applicable1 country
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical TrialActive, not recruitingNCT072266221 country
- Patient Preference Study: Standard of Care Versus Once-daily Trientine TetrahydrochlorideCompletedNCT07010575Phase 21 country
- Real World Evidence Study in Subjects With Wilson's DiseaseCompletedNCT057836871 country
- Exchangeable and Relative Exchangeable Copper as an Alternative to 24-Hour Urinary Copper in Wilson's Disease MonitoringCompletedNCT074620261 country
- A Phase I/II Study of VTX-801 in Adult Patients With Wilson's DiseaseActive, not recruitingNCT04537377Phase 1/24 countries
- Performance and Safety of MEX-CD1 Low-volume Continuous Veno-venous Haemodialysis Medical Device for Copper-extraction in Patients With Wilson's DiseaseTerminatedNCT05917327Not applicable2 countries
- WILSTIM - DBS (WILson STIMulation - Deep Brain Stimulation)CompletedNCT02552628Not applicable1 country
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's DiseaseCompletedNCT03539952Phase 39 countries
- The Exploratory Handheld Optical Coherence Tomography Study in Wilson's DiseaseCompletedNCT07046507Not applicable1 country
- ExAblate Transcranial MRgFUS for the Management of Treatment-Refractory Movement DisordersActive, not recruitingNCT02252380Not applicable1 country
- Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson's Disease in Japan.CompletedNCT00212355Phase 3
- The Clinical Study of Botulinum Toxin Type A Injection in the Treatment of Wilson DiseaseCompletedNCT067625091 country
- Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson DiseaseTerminatedNCT05047523Phase 37 countries