171. ウィルソン病 Wilson disease Clinical trials / Disease details
臨床試験数 : 79 / 薬物数 : 77 - (DrugBank : 17) / 標的遺伝子数 : 6 - 標的パスウェイ数 : 30
| 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 | EUCTR2020-004604-33-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 | 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 | 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] | Trade Name: Cufence (trientine dihydrochloride) Product Name: Cufence (trientine dihydrochloride) INN or Proposed INN: Triethylenetetramine dihydrochloride Other descriptive name: Cufence | Univar Solutions, B.V. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 4 | Poland;Denmark;Germany | ||
| 2 | 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 | 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 | 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] | Trade Name: Cufence (trientine dihydrochloride) Product Name: Cufence (trientine dihydrochloride) INN or Proposed INN: Triethylenetetramine dihydrochloride Other descriptive name: Cufence | Univar Solutions, B.V. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 4 | Poland;Denmark;Germany | ||
| 3 | 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 | 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] | 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 | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 32 | Phase 2 | Denmark | ||
| 4 | EUCTR2020-004604-33-DE (EUCTR) | 23/02/2021 | 17/12/2020 | 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 | 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 | 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] | Trade Name: Cufence (trientine dihydrochloride) Product Name: Cufence (trientine dihydrochloride) INN or Proposed INN: Triethylenetetramine dihydrochloride Other descriptive name: Cufence | Univar Solutions, B.V. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 50 | Phase 4 | France;Poland;Denmark;Germany | ||
| 5 | EUCTR2017-004135-36-NL (EUCTR) | 30/03/2020 | 16/10/2019 | Study to Evaluate the Efficacy and Safety of ALXN1840 in pPtients with Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | 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] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Portugal;Serbia;Taiwan;Hong Kong;Greece;Spain;Israel;Russian Federation;Colombia;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Belgium;Poland;Singapore;Croatia;Germany;Japan;New Zealand;Sweden | ||
| 6 | EUCTR2017-004135-36-PT (EUCTR) | 13/01/2020 | 26/03/2019 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | 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] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Portugal;Serbia;Hong Kong;Taiwan;Spain;Israel;Russian Federation;Colombia;France;Denmark;Australia;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Canada;Brazil;Belgium;Poland;Singapore;Germany;Japan;New Zealand;Sweden | ||
| 7 | EUCTR2017-004135-36-SE (EUCTR) | 29/10/2019 | 16/04/2019 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | 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] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | Serbia;United States;Portugal;Taiwan;Hong Kong;Spain;Russian Federation;Israel;Colombia;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Belgium;Poland;Singapore;Germany;Japan;New Zealand;Sweden | ||
| 8 | EUCTR2017-004135-36-DK (EUCTR) | 16/10/2019 | 05/08/2019 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months - ALXN1840 Efficacy Safety administered for 48 Weeks Versus SOC in patients with Wilson Disease | 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] | Product Name: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE Trade Name: Penicillamine INN or Proposed INN: PENICILLAMINE Trade Name: Trientine dihydrochloride INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE Trade Name: Wilzin INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 3 | Serbia;United States;Portugal;Taiwan;Hong Kong;Spain;Israel;Russian Federation;Colombia;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Poland;Belgium;Singapore;Germany;New Zealand;Japan;Sweden | ||
| 9 | EUCTR2017-004135-36-FR (EUCTR) | 14/09/2018 | 15/01/2018 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult patients who suffer from Wilson Disease. | A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older with an Extension Phase of up to 60 Months | Wilson Disease;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: WTX101 Product Code: WTX101 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Wilson Therapeutics AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 102 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Portugal;Spain;Austria;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Belgium;Poland;Denmark;Netherlands;Germany;Sweden | ||
| 10 | EUCTR2017-004135-36-ES (EUCTR) | 27/04/2018 | 22/01/2018 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult patients who suffer from Wilson Disease. | A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older with an Extension Phase of up to 60 Months | Wilson Disease;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: WTX101 Product Code: WTX101 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Wilson Therapeutics AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 102 | Phase 3 | France;United States;Hungary;Czech Republic;Poland;Spain;Austria;Israel;Germany;Italy;United Kingdom | ||
| 11 | EUCTR2017-004135-36-GB (EUCTR) | 23/03/2018 | 19/12/2017 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult patients who suffer from Wilson Disease. | A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older with an Extension Phase of up to 60 Months | Wilson Disease;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: WTX101 Product Code: WTX101 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE Other descriptive name: ZINC ACETATE DIHYDRATE | Wilson Therapeutics AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 102 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Portugal;Spain;Austria;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Belgium;Poland;Denmark;Netherlands;Germany;Sweden | ||
| 12 | EUCTR2017-004135-36-DE (EUCTR) | 16/03/2018 | 22/12/2017 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | 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] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 3 | Serbia;United States;Portugal;Hong Kong;Taiwan;Greece;Spain;Russian Federation;Israel;Colombia;France;Australia;Denmark;Netherlands;Czechia;Korea, Democratic People's Republic of;Turkey;Austria;United Kingdom;Hungary;Canada;Belgium;Brazil;Poland;Singapore;Croatia;Germany;New Zealand;Japan;Sweden | ||
| 13 | EUCTR2017-004135-36-AT (EUCTR) | 27/02/2018 | 17/01/2018 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | 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] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 3 | Serbia;United States;Portugal;Hong Kong;Taiwan;Greece;Spain;Russian Federation;Israel;Colombia;France;Australia;Denmark;Netherlands;Czechia;Korea, Democratic People's Republic of;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Belgium;Brazil;Poland;Singapore;Croatia;Germany;New Zealand;Japan;Sweden | ||
| 14 | EUCTR2017-004135-36-HU (EUCTR) | 12/02/2018 | 07/02/2018 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult patients who suffer from Wilson Disease. | A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older with an Extension Phase of up to 60 Months | 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] | Product Name: WTX101 Product Code: WTX101 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Wilson Therapeutics AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 102 | Phase 3 | France;United States;Czech Republic;Hungary;Poland;Spain;Austria;Israel;Germany;Italy;United Kingdom | ||
| 15 | EUCTR2013-003564-31-IT (EUCTR) | 14/07/2016 | 26/04/2018 | A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months. | Multicentre, Retrospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn from Therapy With - N/A | Wilson disease, also known as hepatolenticular degeneration, a rare automsomal, recessively inherited disorder which results in chronic copper intoxication. MedDRA version: 20.0;Level: PT;Classification code 10019819;Term: Hepato-lenticular degeneration;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Trientine dihydrochloride 300mg capsules | UNIVAR LTD | NULL | Not Recruiting | Female: yes Male: yes | 90 | Phase 2 | France;Greece;Spain;Germany;United Kingdom;Italy | ||
| 16 | NCT02426905 (ClinicalTrials.gov) | January 2016 | 18/9/2014 | Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine | Multicentre, Retrospective and Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine | Wilson Disease | Drug: trientine dihydrochloride | Univar BV | NULL | Active, not recruiting | 1 Year | 90 Years | All | 90 | Phase 4 | Germany;Greece;Italy;United Kingdom |
| 17 | EUCTR2013-003564-31-DE (EUCTR) | 27/02/2014 | 22/10/2013 | A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months. | Multicentre, Retrospective and Prospective Study to Assess Long Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn from Therapy With d-Penicillamine | Wilson disease, also known as hepatolenticular degeneration, a rare automsomal, recessively inherited disorder which results in chronic copper intoxication. MedDRA version: 19.1;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Trientine dihydrochloride 300mg capsules INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE | Univar B.V. | NULL | Not Recruiting | Female: yes Male: yes | 90 | Phase 4 | Germany;Italy | ||
| 18 | NCT01874028 (ClinicalTrials.gov) | May 2013 | 6/6/2013 | A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease Patients | A Phase 1 Pharmacokinetic Profiling Study in Patients Receiving Trientine Dihydrochloride for the Treatment of Wilson's Disease. | Wilson's Disease | Drug: trientine dihydrochloride | Univar BV | Aptiv Solutions | Completed | 6 Years | 90 Years | Both | 20 | Phase 1 | Germany |
| 19 | EUCTR2017-004135-36-PL (EUCTR) | 03/04/2018 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | 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] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | NA | Female: yes Male: yes | 180 | Phase 3 | Portugal;Serbia;United States;Hong Kong;Taiwan;Greece;Spain;Russian Federation;Israel;Colombia;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Poland;Belgium;Singapore;Croatia;Germany;New Zealand;Japan;Sweden | |||
| 20 | EUCTR2017-004135-36-BE (EUCTR) | 17/06/2019 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | 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] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Portugal;Serbia;Taiwan;Hong Kong;Greece;Spain;Israel;Russian Federation;Colombia;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Belgium;Poland;Singapore;Croatia;Germany;Japan;New Zealand;Sweden | |||
| 21 | EUCTR2020-004604-33-FR (EUCTR) | 12/03/2021 | 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 | 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 | 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] | Trade Name: Cufence (trientine dihydrochloride) Product Name: Cufence (trientine dihydrochloride) INN or Proposed INN: Triethylenetetramine dihydrochloride Other descriptive name: Cufence | Univar Solutions, B.V. | NULL | NA | Female: yes Male: yes | 50 | Phase 4 | France;Poland;Denmark;Germany |