171. Wilson disease Clinical trials / Disease details
Clinical trials : 68 / Drugs : 79 - (DrugBank : 17) / Drug target genes : 3 - Drug target pathways : 28
Showing 1 to 10 of 68 diseases
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2020-005266-34-ES (EUCTR) | 11/02/2022 | 06/09/2021 | A study to understand the safety and the effects of a virus that transfers a modified protein responsible for copper metabolism (copper-transporting P-type adenosine triphosphatase, ATP7B) in adults with Wilson disease. A study to understand the safety and the effects of a virus that transfers a modified protein respon ... | A Randomized, Double-blind, Placebo-controlled, Multicenter, Seamless, Adaptive, Safety, Dose-finding, and Phase 3 Clinical Study of UX701 AAV-mediated Gene Transfer for the Treatment of Wilson Disease - Cyprus2+ A Randomized, Double-blind, Placebo-controlled, Multicenter, Seamless, Adaptive, Safety, Dose-findin ... | Wilson disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Wilson disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;S ... | Product Name: UX701 (5.0 × 10^12 genome copies/kg) INN or Proposed INN: UX701 Other descriptive name: Adeno-associated viral vector serotype 9 encoding human ATP7B Product Name: UX701 (1.0 × 10^13 genome copies/kg) INN or Proposed INN: UX701 Other descriptive name: Adeno-associated viral vector serotype 9 encoding human ATP7B Product Name: UX701 (2.0 × 10^13 genome copies/kg) INN or Proposed INN: UX701 Other descriptive name: Adeno-associated viral vector serotype 9 encoding human ATP7B Product Name: UX701(5.0 × 10^12 genome copies/kg) INN or Proposed INN: UX701 Other descriptive name: A ... | Ultragenyx Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 94 | Phase 1;Phase 2;Phase 3 | United States;Portugal;Spain;Austria;Italy;United Kingdom;France;Canada;Argentina;Brazil;Poland;Germany;Japan United States;Portugal;Spain;Austria;Italy;United Kingdom;France;Canada;Argentina;Brazil;Poland;Germ ... | ||
| 2 | EUCTR2021-001015-82-DE (EUCTR) | 26/11/2021 | 10/08/2021 | Phase 3, open-label study of ALXN1840 versus standard of care in pediatric participants with Wilson disease Phase 3, open-label study of ALXN1840 versus standard of care in pediatric participants with Wilson ... | A multicenter, randomized, controlled, open-label, rater-blinded study to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease. A multicenter, randomized, controlled, open-label, rater-blinded study to evaluate efficacy, safety, ... | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;S ... | Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive n ... | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 3 | Serbia;United States;Czechia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Japan;Korea, Republic of Serbia;United States;Czechia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Hungary;C ... | ||
| 3 | EUCTR2021-001015-82-ES (EUCTR) | 22/11/2021 | 23/08/2021 | Phase 3, open-label study of ALXN1840 versus standard of care in pediatric participants with Wilson disease Phase 3, open-label study of ALXN1840 versus standard of care in pediatric participants with Wilson ... | A multicenter, randomized, controlled, open-label, rater-blinded study to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease. A multicenter, randomized, controlled, open-label, rater-blinded study to evaluate efficacy, safety, ... | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;S ... | Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive n ... | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 3 | United Kingdom;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Japan;Korea, Republic of;Serbia;United States;Czechia;Spain;Turkey;Austria;Russian Federation United Kingdom;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Japan;Korea, Repub ... | ||
| 4 | EUCTR2020-004604-33-PL (EUCTR) | 16/11/2021 | 04/06/2021 | Multicentre, open-label study to investigate the effects Cufence has, the effects the body has on Cufence and the continued safety and efficacy on patients with Wilson Disease Multicentre, open-label study to investigate the effects Cufence has, the effects the body has on Cu ... | Open label, Multicenter, Prospective Study to Characterize the Pharmacokinetics and Pharmacodynamics of Cufence (Trientine Dihydrochloride) and to Investigate the Efficacy and Safety in Wilson’s Disease Patients - TR-004 UNITED Study Open label, Multicenter, Prospective Study to Characterize the Pharmacokinetics and Pharmacodynamics ... | Wilson Disease MedDRA version: 22.1;Level: PT;Classification code 10019819;Term: Hepato-lenticular degeneration;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Wilson Disease MedDRA version: 22.1;Level: PT;Classification code 10019819;Term: Hepato-lenticular d ... | Trade Name: Cufence (trientine dihydrochloride) Product Name: Cufence (trientine dihydrochloride) INN or Proposed INN: Triethylenetetramine dihydrochloride Other descriptive name: Cufence Trade Name: Cufence(trientine dihydrochloride) Product Name: Cufence(trientine dihydrochloride) INN or ... | Univar Solutions, B.V. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 4 | Poland;Denmark;Germany | ||
| 5 | EUCTR2020-000963-22-DE (EUCTR) | 18/10/2021 | 10/03/2021 | A clinical trial: treatment of adult Wilson's Disease patients with gene therapy | A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients with Wilson’s Disease - GATEWAY clinical trial A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dos ... | Wilson's Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Wilson's Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease ... | Product Name: N/A Product Code: VTX-801 INN or Proposed INN: NA Other descriptive name: Adeno-associated viral vector serotype 3B encoding shortened human ATP7B Product Name: N/A Product Code: VTX-801 INN or Proposed INN: NA Other descriptive name: Adeno-associate ... | Vivet Therapeutics SAS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 1;Phase 2 | United States;Denmark;Germany;United Kingdom | ||
| 6 | EUCTR2020-004604-33-DK (EUCTR) | 08/10/2021 | 11/03/2021 | Multicentre, open-label study to investigate the effects Cufence has, the effects the body has on Cufence and the continued safety and efficacy on patients with Wilson Disease Multicentre, open-label study to investigate the effects Cufence has, the effects the body has on Cu ... | Open label, Multicenter, Prospective Study to Characterize the Pharmacokinetics and Pharmacodynamics of Cufence (Trientine Dihydrochloride) and to Investigate the Efficacy and Safety in Wilson’s Disease Patients - TR-004 UNITED Study Open label, Multicenter, Prospective Study to Characterize the Pharmacokinetics and Pharmacodynamics ... | Wilson Disease MedDRA version: 22.1;Level: PT;Classification code 10019819;Term: Hepato-lenticular degeneration;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] Wilson Disease MedDRA version: 22.1;Level: PT;Classification code 10019819;Term: Hepato-lenticular d ... | Trade Name: Cufence (trientine dihydrochloride) Product Name: Cufence (trientine dihydrochloride) INN or Proposed INN: Triethylenetetramine dihydrochloride Other descriptive name: Cufence Trade Name: Cufence(trientine dihydrochloride) Product Name: Cufence(trientine dihydrochloride) INN or ... | Univar Solutions, B.V. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 4 | Poland;Denmark;Germany | ||
| 7 | NCT05047523 (ClinicalTrials.gov) | October 6, 2021 | 9/9/2021 | Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease | A Multicenter, Randomized, Controlled, Open-label, Rater-blinded Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease A Multicenter, Randomized, Controlled, Open-label, Rater-blinded Study to Evaluate Efficacy, Safety, ... | Wilson Disease | Drug: ALXN1840;Drug: Standard of Care | Alexion Pharmaceuticals | NULL | Recruiting | 3 Years | 17 Years | All | 48 | Phase 3 | Australia;Japan;Korea, Republic of;Poland;Spain |
| 8 | NCT04884815 (ClinicalTrials.gov) | September 27, 2021 | 7/5/2021 | Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease | A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Seamless, Adaptive, Safety, Dose-Finding, and Phase 3 Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Seamless, Adaptive, Safety, Dose-Findin ... | Wilson Disease | Genetic: UX701;Other: Placebo;Drug: oral corticosteroids;Drug: Placebo for oral corticosteroids | Ultragenyx Pharmaceutical Inc | NULL | Recruiting | 18 Years | N/A | All | 90 | Phase 1/Phase 2 | United States |
| 9 | EUCTR2020-000963-22-DK (EUCTR) | 05/07/2021 | 07/05/2021 | A clinical trial: treatment of adult Wilson's Disease patients with gene therapy | A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in AdultPatients with Wilson's Disease - GATEWAY clinical trial A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dos ... | Wilson's Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Wilson's Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease ... | Product Name: N/A Product Code: VTX-801 INN or Proposed INN: N/A Other descriptive name: Adeno-associated viral vector serotype 3B encoding shortened human ATP7B Product Name: N/A Product Code: VTX-801 INN or Proposed INN: N/A Other descriptive name: Adeno-associat ... | Vivet Therapeutics SAS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 16 | Phase 1;Phase 2 | United States;Denmark;Germany;United Kingdom | ||
| 10 | EUCTR2020-005832-31-DK (EUCTR) | 13/04/2021 | 01/02/2021 | The effect of ALXN1840 on uptake of copper in the liver as measured by copper PET/CT scan. | Efficacy of ALXN1840 on human hepatic copper uptake quantified with 64CuCl2 PET/CT-scan. - ALXN1840-HV-Cu Absorption Efficacy of ALXN1840 on human hepatic copper uptake quantified with 64CuCl2 PET/CT-scan. - ALXN1840- ... | Healthy Volunteers (Wilson's disease) MedDRA version: 22.0;Level: SOC;Classification code 10022891;Term: Investigations;System Organ Class: 10022891 - Investigations;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Healthy Volunteers (Wilson's disease) MedDRA version: 22.0;Level: SOC;Classification code 10022891;T ... | Trade Name: Cuprior 150 mg INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE Other descriptive name: Cuprior Trade Name: Metalcaptase Product Name: Metalcaptase INN or Proposed INN: PENICILLAMINE Product Name: ALXN1840 INN or Proposed INN: ALXN1840 Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE Trade Name: Cuprior150 mg INN or Proposed INN: TRIENTINEDIHYDROCHLORIDE Other descriptive name: Cuprio ... | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 32 | Phase 2 | Denmark |