171. ウィルソン病 Wilson disease Clinical trials / Disease details
臨床試験数 : 68 / 薬物数 : 79 - (DrugBank : 17) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28
| 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 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+ | 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] | 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 | 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 | ||
| 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 | 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. | 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 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 | 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 | ||
| 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 | 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. | 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 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 | 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 | ||
| 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 | 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 | ||
| 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 | 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] | 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 | 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 | 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 | ||
| 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 | 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 | 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 | 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] | 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 | 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 | 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 | ||
| 11 | EUCTR2021-000102-25-DK (EUCTR) | 09/04/2021 | 01/02/2021 | The effect of ALXN1840 on excretion of copper in gall as measured by copper PET/MR-scan. | Efficacy of ALXN1840 on human biliary copper excretion quantified with 64CuCl2 PET/MR-scan - ALXN1840-WD-Cu Excretion | Wilson's 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] | Product Name: ALXN1840 INN or Proposed INN: ALXN1840 Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE Product Name: 64CuCl2 INN or Proposed INN: [64Cu]Cl2 Other descriptive name: COPPER (64CU) CHLORIDE | Aarhus University Hospital | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 2 | Denmark | ||
| 12 | 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 | ||
| 13 | EUCTR2019-003711-60-DE (EUCTR) | 15/01/2021 | 02/06/2020 | A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840 | A Phase 2, single-arm, pathologist-blinded study using liver biopsy specimens to assess copper concentration and histopathologic changes in patients with Wilson disease who are treated with ALXN1840 for 48 weeks followed by an extension treatment period with ALXN1840 for up to an additional 48 weeks | Wilson's 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 Product Code: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 2 | United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Australia;Denmark;Germany;New Zealand;Sweden;Korea, Republic of | ||
| 14 | NCT04422431 (ClinicalTrials.gov) | December 2, 2020 | 22/5/2020 | Copper Concentration and Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840 | A Phase 2, Single-arm, Pathologist-blinded Study Using Liver Biopsy Specimens to Assess Copper Concentration and Histopathologic Changes in Patients With Wilson Disease Who Are Treated With ALXN1840 for 48 Weeks Followed by an Extension Treatment Period With ALXN1840 for up to an Additional 48 Weeks | Wilson Disease | Drug: Bis-Choline Tetrathiomolybdate | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 31 | Phase 2 | United States;Denmark;New Zealand;Russian Federation;Singapore;Spain;United Kingdom;Australia;Austria;Belgium;Canada;Germany;Korea, Republic of;Poland |
| 15 | EUCTR2019-003711-60-FR (EUCTR) | 22/10/2020 | 20/08/2020 | A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840 | A Phase 2, single-arm, pathologist-blinded study using liver biopsy specimens to assess copper concentration and histopathologic changes in patients with Wilson disease who are treated with ALXN1840 for 48 weeks followed by an extension treatment period with ALXN1840 for up to an additional 48 weeks | Wilson's 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 Product Code: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 2 | United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Australia;Denmark;Germany;New Zealand;Sweden;Korea, Republic of | ||
| 16 | NCT04526210 (ClinicalTrials.gov) | October 21, 2020 | 19/8/2020 | Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants | A Phase 1, Randomized, 2-Period, 2-Sequence, Cross-over Study to Determine the Effect of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants | Wilson Disease | Drug: ALXN1840;Drug: Bupropion Hydrochloride | Alexion Pharmaceuticals | NULL | Completed | 18 Years | 50 Years | All | 54 | Phase 1 | United States |
| 17 | EUCTR2019-003711-60-AT (EUCTR) | 23/09/2020 | 01/10/2020 | A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840 | A Phase 2, single-arm, pathologist-blinded study using liver biopsy specimens to assess copper concentration and histopathologic changes in patients with Wilson disease who are treated with ALXN1840 for 48 weeks followed by an extension treatment period with ALXN1840 for up to an additional 48 weeks | Wilson's 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 Product Code: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 2 | United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Australia;Denmark;Germany;New Zealand;Sweden;Korea, Republic of | ||
| 18 | NCT04573309 (ClinicalTrials.gov) | September 7, 2020 | 28/9/2020 | Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 | A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 | Wilson Disease | Drug: ALXN1840 | Alexion Pharmaceuticals | NULL | Recruiting | 18 Years | N/A | All | 10 | Phase 2 | United States;New Zealand;United Kingdom |
| 19 | EUCTR2019-003711-60-DK (EUCTR) | 27/08/2020 | 19/06/2020 | A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840 | A Phase 2, single-arm, pathologist-blinded study using liver biopsy specimens to assess copper concentration and histopathologic changes in patients with Wilson disease who are treated with ALXN1840 for 48 weeks followed by an extension treatment period with ALXN1840 for up to an additional 48 weeks | Wilson's 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 Product Code: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 2 | United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Denmark;Australia;Germany;New Zealand;Sweden;Korea, Republic of | ||
| 20 | EUCTR2019-003711-60-GB (EUCTR) | 26/08/2020 | 09/07/2020 | A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840 | A Phase 2, single-arm, pathologist-blinded study using liver biopsy specimens to assess copper concentration and histopathologic changes in patients with Wilson disease who are treated with ALXN1840 for 48 weeks followed by an extension treatment period with ALXN1840 for up to an additional 48 weeks | Wilson's 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 Product Code: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 2 | Korea, Republic of;Sweden;New Zealand;Germany;Denmark;Australia;Singapore;Poland;Belgium;Canada;France;United Kingdom;Russian Federation;Austria;Turkey;Spain;Serbia;United States | ||
| 21 | EUCTR2020-001104-41-GB (EUCTR) | 22/07/2020 | 11/06/2020 | Copper Balance in Participants with Wilson Disease Treated with ALXN1840 | A Phase 2, open-label study to assess copper and molybdenum balance in participants with Wilson disease treated with ALXN1840 - Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 | Wilson Disease (WD) 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 Code: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 10 | Phase 2 | United Kingdom | ||
| 22 | NCT04526197 (ClinicalTrials.gov) | July 8, 2020 | 19/8/2020 | Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants. | A Phase 1, Randomized, 2-Period, 2-Sequence, Cross-over Study to Determine the Effect of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants. | Wilson Disease | Drug: ALXN1840;Drug: Celecoxib | Alexion Pharmaceuticals | NULL | Completed | 18 Years | 50 Years | All | 36 | Phase 1 | United States |
| 23 | 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 | ||
| 24 | 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 | ||
| 25 | 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;Sweden;New Zealand;Japan;Germany;Singapore;Poland;Belgium;Brazil;Canada;Hungary;Czech Republic | ||
| 26 | 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 | 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;Spain;Russian Federation;Israel;Colombia;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Belgium;Poland;Singapore;Germany;New Zealand;Japan;Sweden | ||
| 27 | EUCTR2016-003876-29-BE (EUCTR) | 31/07/2019 | 13/05/2019 | Chelate Study: Trientine for the treatment of Wilson's disease | CHELATE STUDY: Trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s disease | Wilson's disease (WD) is a life-threatening inborn error of copper metabolism leading to an excessive copper accumulation, mainly in the liver or brain, causing hepatic and neurologic severe symptoms. 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] | Trade Name: Cuprior 150 mg film-coated tablets Product Name: TETA 4HCl Product Code: TETA 4HCl INN or Proposed INN: Trientine tetrahydrochloride Other descriptive name: TETA 4HCl Trade Name: Artamin 150 mg Product Name: D-penicillamine 150mg INN or Proposed INN: penicillamine Other descriptive name: penicillamine Trade Name: Artamin 250 mg Product Name: D-penicillamine 250 mg INN or Proposed INN: penicillamine Other descriptive name: penicillamine | Orphalan | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | France;United States;Poland;Brazil;Belgium;Austria;Denmark;Germany;United Kingdom;Italy;Sweden | ||
| 28 | ChiCTR1900023410 | 2019-06-01 | 2019-05-26 | The application of quantitative susceptibility mapping and blood and urine cell culture in Wilson's disease | The application of quantitative susceptibility mapping and blood and urine cell culture in Wilson's disease | Wilson’s disease | Gold Standard:The main clinical diagnostic criteria: 1) liver disease history, liver disease symptoms or extravertebral system symptoms; 2) serum ceruloplasmin was significantly decreased and/or liver copper increased; 3) corneal Kayser-Fleischer ring; 4) positive family history.;Index test:Quantitative susceptibility mapping; | Shanghai Tongren Hospital | NULL | Recruiting | Both | Target condition:40;Difficult condition:20 | China | |||
| 29 | EUCTR2019-000905-57-DK (EUCTR) | 17/04/2019 | 14/03/2019 | Efficacy of zinc on copper uptake in the body depending on zinc type and dose regimen measured with 64CuCl2 PET/CT-scan | Efficacy of zinc on human gut copper uptake depending on zinc type and dose regimen quantified with 64CuCl2 PET/CT-scan | Healthy volunteers (Wilson's 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] | Trade Name: Wilzin INN or Proposed INN: Zinc acetate Trade Name: Zink Natur-Drogeriet INN or Proposed INN: Zinc gluconate Other descriptive name: ZINC GLUCONATE Product Name: 64CuCl2 INN or Proposed INN: 64CuCl2 Other descriptive name: COPPER (64CU) CHLORIDE | The Department of Hepatology and Gastroenterology, Aarhus University Hospital | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 4 | Denmark | ||
| 30 | NCT03957720 (ClinicalTrials.gov) | March 15, 2019 | 8/4/2019 | The Individual Therapy for Patients With Wilson's Disease | The Individual Therapy for Patients With Wilson's Disease | Wilson's Disease | Drug: DMPS;Drug: Penicillamine;Drug: DMSA;Drug: Zinc gluconate | Second Affiliated Hospital, School of Medicine, Zhejiang University | NULL | Recruiting | N/A | N/A | All | 400 | Early Phase 1 | China |
| 31 | 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 | ||
| 32 | NCT03659331 (ClinicalTrials.gov) | September 2018 | 28/8/2018 | A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease | A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease | Wilson Disease | Dietary Supplement: Zinc | Prof. Elon Pras | NULL | Unknown status | 18 Years | N/A | All | 50 | N/A | NULL |
| 33 | NCT03539952 (ClinicalTrials.gov) | June 4, 2018 | 4/5/2018 | Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease | CHELATE STUDY: Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's Disease | Wilson Disease | Drug: TETA 4HCL;Drug: Penicillamine | GMP-Orphan SAS | NULL | Active, not recruiting | 18 Years | 75 Years | All | 55 | Phase 3 | United States;Belgium;Brazil;Denmark;France;Germany;Italy;Poland;United Kingdom;Sweden |
| 34 | 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 | ||
| 35 | 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 | ||
| 36 | 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 | ||
| 37 | 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 | Poland;Singapore;Croatia;Germany;New Zealand;Japan;Sweden;Brazil;Belgium;Canada;Hungary;Czech Republic;United Kingdom;Austria;Turkey;Korea, Democratic People's Republic of;Czechia;Netherlands;Denmark;Australia;France;Colombia;Israel;Russian Federation;Spain;Greece;Taiwan;Hong Kong;Portugal;United States;Serbia | ||
| 38 | NCT03403205 (ClinicalTrials.gov) | February 15, 2018 | 19/12/2017 | Efficacy and Safety of ALXN1840 (Formerly Named WTX101) Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease With an Extension Period of up to 60 Months | 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 | Drug: ALXN1840;Drug: SoC Therapy | Alexion Pharmaceuticals | NULL | Active, not recruiting | 12 Years | N/A | All | 215 | Phase 3 | United States;Australia;Austria;Belgium;Canada;Czechia;Denmark;France;Germany;Hong Kong;Hungary;Israel;Japan;Korea, Republic of;New Zealand;Poland;Russian Federation;Serbia;Singapore;Spain;Taiwan;Turkey;United Kingdom;Italy |
| 39 | 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 | ||
| 40 | NCT03299829 (ClinicalTrials.gov) | January 10, 2018 | 25/9/2017 | A Retrospective Study to Assess the Clinical Efficacy and Safety of Trientine in Wilson's Disease Patients | A Retrospective Study to Assess the Clinical Efficacy and Safety of Trientine in Wilson's Disease Patients | Trientine Treatment for Wilson's Disease | Drug: Trientine | Excelsior | NULL | Completed | 3 Years | 75 Years | All | 48 | Taiwan | |
| 41 | EUCTR2016-003876-29-IT (EUCTR) | 13/12/2017 | 06/02/2018 | Chelate Study: Trientine for the treatment of Wilson's disease | CHELATE STUDY: Trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s disease - Chelate Study: Trientine for the treatment of Wilson's disease | Wilson's disease (WD) is a life-threatening inborn error of copper metabolism leading to an excessive copper accumulation, mainly in the liver or brain, causing hepatic and neurologic severe symptoms. 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] | Product Name: TETA.4HCI Product Code: TETA.4HCI INN or Proposed INN: Trientine tetrahydrochloride Trade Name: PEMINE - 150 MG CAPSULE RIGIDE 50 CAPSULE INN or Proposed INN: D-penicillamine | GMP ORPHAN SA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 3 | France;United States;Poland;Micronesia, Federated States of;Brazil;Denmark;Austria;Germany;United Kingdom;Italy | ||
| 42 | EUCTR2016-003876-29-DE (EUCTR) | 15/09/2017 | 03/04/2017 | Chelate Study: Trientine for the treatment of Wilson's disease | CHELATE STUDY: Trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s disease | Wilson's disease (WD) is a life-threatening inborn error of copper metabolism leading to an excessive copper accumulation, mainly in the liver or brain, causing hepatic and neurologic severe symptoms. 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] | Trade Name: Cuprior 150 mg film-coated tablets Product Name: TETA 4HCl Product Code: TETA 4HCl INN or Proposed INN: Trientine tetrahydrochloride Other descriptive name: TETA 4HCl Trade Name: Metalcaptase® 150 mg gastro-resistant tablet INN or Proposed INN: penicillamine Other descriptive name: penicillamine Trade Name: Metalcaptase® 300 mg gastro-resistant tablet INN or Proposed INN: penicillamine Other descriptive name: penicillamine | Orphalan | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | France;United States;Poland;Belgium;Brazil;Denmark;Austria;Germany;United Kingdom;Italy;Sweden | ||
| 43 | EUCTR2016-003876-29-DK (EUCTR) | 16/05/2017 | 29/03/2017 | Chelate Study: Trientine for the treatment of Wilson's disease | CHELATE STUDY: Trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s disease | Wilson's disease (WD) is a life-threatening inborn error of copper metabolism leading to an excessive copper accumulation, mainly in the liver or brain, causing hepatic and neurologic severe symptoms. 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] | Trade Name: Cuprior 150 mg film-coated tablets Product Name: TETA 4HCl Product Code: TETA 4HCl INN or Proposed INN: Trientine tetrahydrochloride Other descriptive name: TETA 4HCl Product Name: D-penicillamine 150mg INN or Proposed INN: penicillamine Other descriptive name: penicillamine Product Name: D-penicillamine 250 mg INN or Proposed INN: penicillamine Other descriptive name: penicillamine | Orphalan | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | France;United States;Poland;Belgium;Brazil;Austria;Denmark;Germany;United Kingdom;Italy;Sweden | ||
| 44 | EUCTR2016-003876-29-AT (EUCTR) | 12/04/2017 | 10/04/2017 | Chelate Study: Trientine for the treatment of Wilson's disease | CHELATE STUDY: Trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s disease | Wilson's disease (WD) is a life-threatening inborn error of copper metabolism leading to an excessive copper accumulation, mainly in the liver or brain, causing hepatic and neurologic severe symptoms. 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] | Product Name: TETA 4HCl Product Code: TETA 4HCl INN or Proposed INN: Trientine tetrahydrochloride Other descriptive name: TETA 4HCl INN or Proposed INN: penicillamine Other descriptive name: penicillamine INN or Proposed INN: penicillamine Other descriptive name: penicillamine | gmp-orphan SA | NULL | Not Recruiting | Female: yes Male: yes | 55 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Poland;Brazil;Denmark;Austria;Germany;Italy;United Kingdom | ||
| 45 | EUCTR2016-003876-29-GB (EUCTR) | 10/04/2017 | 04/04/2017 | Chelate Study: Trientine for the treatment of Wilson's disease | CHELATE STUDY: Trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s disease - CHELATE STUDY GMPO-131-002 | Wilson's disease (WD) is a life-threatening inborn error of copper metabolism leading to an excessive copper accumulation, mainly in the liver or brain, causing hepatic and neurologic severe symptoms. 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] | Trade Name: Cuprior 150mg film-coated tablets Product Name: Cuprior 150mg film-coated tablets Product Code: TETA.4HCl INN or Proposed INN: Trientine tetrahydrochloride Other descriptive name: TETA 4HCl INN or Proposed INN: D-penicillamine Other descriptive name: D-penicillamine INN or Proposed INN: D-penicillamine Other descriptive name: D-penicillamine | gmp-orphan SA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 55 | Phase 3 | France;United States;Poland;Brazil;Belgium;Austria;Denmark;Germany;Italy;United Kingdom;Sweden | ||
| 46 | ChiCTR-DDD-17010552 | 2017-01-25 | 2017-02-04 | Genetic diagnosis of Wilson's disease | Genetic diagnosis of Wilson's disease | Wilson Disease | Gold Standard:Clinical symptoms; Family history of genetic; Plasma ceruloplasmin levels;Urine copper levels of 24 hours; The nervous system of assessment and head imaging examination;Index test:Determination of gene sequence of the gene of ATP7B; | Anhui Provincial Hospital | NULL | Recruiting | Both | Target condition:20;Difficult condition:0 | China | |||
| 47 | 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 | ||
| 48 | NCT02763215 (ClinicalTrials.gov) | May 19, 2016 | 3/5/2016 | The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment | Multi-Center Study for the Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care Treatment | Wilson Disease | Drug: Standard of Care Medications | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 64 | United States;Austria;Germany;Poland;United Kingdom | |
| 49 | NCT02702765 (ClinicalTrials.gov) | February 2016 | 26/2/2016 | sCD163 and sMR in Wilsons Disease - Associations With Disease Severity and Fibrosis | Macrophages and the Macrophage Activation Markers sCD163 and Mannose Receptor (sMR) in Patients With Wilsons Disease - Associations With Liver Disease Severity and Fibrosis | Wilsons Disease | Procedure: Fibroscan;Procedure: Ultrasound;Drug: Galactose;Procedure: Liver biopsy;Procedure: Functional hepatic nitrogen clearance | University of Aarhus | Aarhus University Hospital | Active, not recruiting | 18 Years | 100 Years | All | 33 | N/A | Denmark |
| 50 | 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 |
| 51 | EUCTR2014-001703-41-GB (EUCTR) | 11/05/2015 | 29/01/2015 | A clinical trial in adult Wilson Disease Patients to evaluate efficacy and safety of WTX101 following administration for 24 weeks with an Extension Phase of 12 Months | A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older with an Extension Phase of 12 Months - Wilson Disease Patients to evaluate efficacy and safety of WTX101 | Wilson Disease MedDRA version: 18.1;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850 | Product Code: WTX101 INN or Proposed INN: none Other descriptive name: bis-choline TETRATHIOMOLYBDATE | Wilson Therapeutics AB | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Poland;Austria;Germany;United Kingdom | ||
| 52 | EUCTR2014-001703-41-PL (EUCTR) | 22/12/2014 | 17/10/2014 | A clinical trial in adult Wilson Disease Patients to evaluate efficacy and safety of WTX101 following administration for 24 weeks and an extension phase of 36 months. | A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older with an Extension Phase of 36 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 Code: WTX101 INN or Proposed INN: none Other descriptive name: bis-choline TETRATHIOMOLYBDATE | Wilson Therapeutics AB | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | United States;Poland;Austria;Germany;United Kingdom | ||
| 53 | NCT02273596 (ClinicalTrials.gov) | November 24, 2014 | 20/10/2014 | Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients | A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older With an Extension Phase of 36 Months | Wilson Disease | Drug: ALXN1840 | Alexion Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 29 | Phase 2 | United States;Austria;Germany;Poland;United Kingdom |
| 54 | EUCTR2014-001703-41-AT (EUCTR) | 21/08/2014 | 24/06/2014 | A clinical trial in adult Wilson Disease Patients to evaluate efficacy and safety of WTX101 following administration for 24 weeks and an extension phase of 36 months. | A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older with an Extension Phase of 36 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 Code: WTX101 INN or Proposed INN: none Other descriptive name: bis-choline TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Poland;Austria;Germany;United Kingdom | ||
| 55 | EUCTR2014-001703-41-DE (EUCTR) | 18/08/2014 | 05/06/2014 | A clinical trial in adult Wilson Disease Patients to evaluate efficacy and safety of WTX101 following administration for 24 weeks and an extension phase of 36 months. | A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older with an Extension Phase of 36 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 Code: WTX101 INN or Proposed INN: none Other descriptive name: bis-choline TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, INC. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Poland;Austria;Germany;United Kingdom | ||
| 56 | 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 | ||
| 57 | 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 |
| 58 | NCT01472874 (ClinicalTrials.gov) | January 2010 | 11/11/2011 | Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease | Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease | Wilson Disease | Drug: Once a day Trientine | Yale University | Bausch Health Americas, Inc. | Completed | 18 Years | N/A | All | 8 | N/A | United States |
| 59 | NCT00212355 (ClinicalTrials.gov) | March 2005 | 13/9/2005 | Efficacy and Safety, Long-term Study of Zinc Acetate to Treat Wilson's Disease in Japan. | Phase3, Open-Label, Clinical Trial of Zinc Acetate for Treatment of Wilson's Disease in Japan. | Wilson's Disease | Drug: NPC-02 | Nobelpharma | NULL | Completed | 1 Year | N/A | All | 37 | Phase 3 | NULL |
| 60 | NCT00212368 (ClinicalTrials.gov) | October 2004 | 13/9/2005 | Efficacy and Safety Study of Zinc Acetate to Treat Wilson's Disease in Japan. | Phase3,Open-label,Clinical Trial of Zinc Acetate for Treatment of Wilson's Disease in Japan. | Wison's Disease | Drug: Zinc acetate | Nobelpharma | NULL | Completed | 1 Year | N/A | Both | Phase 3 | NULL | |
| 61 | NCT04965545 (ClinicalTrials.gov) | January 1, 2004 | 5/7/2021 | Role for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson Disease | Role for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson Disease | Wilson Disease | Dietary Supplement: low copper diet | Second Affiliated Hospital, School of Medicine, Zhejiang University | NULL | Recruiting | 6 Years | 65 Years | All | 1000 | China | |
| 62 | NCT00004339 (ClinicalTrials.gov) | January 1994 | 18/10/1999 | Study of Tetrathiomolybdate in Patients With Wilson Disease | Wilson Disease | Drug: tetrathiomolybdate;Drug: trientine | National Center for Research Resources (NCRR) | University of Michigan | Completed | N/A | N/A | Both | 90 | Phase 3 | United States | |
| 63 | NCT00004338 (ClinicalTrials.gov) | October 1993 | 18/10/1999 | Study of Zinc for Wilson Disease | Wilson Disease | Drug: zinc acetate | National Center for Research Resources (NCRR) | University of Michigan | Completed | N/A | N/A | Both | 300 | Phase 4 | United States | |
| 64 | EUCTR2021-001015-82-PL (EUCTR) | 15/09/2021 | Phase 3, open-label 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. | 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 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 | Alexion Pharmaceuticals, Inc. | NULL | NA | 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 | |||
| 65 | 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 | |||
| 66 | 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 | |||
| 67 | EUCTR2017-004135-36-CZ (EUCTR) | 09/01/2018 | 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- Blinder, Multi-Centre 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;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: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 180 | Phase 3 | Serbia;United States;Portugal;Hong Kong;Taiwan;Spain;Russian Federation;Israel;Colombia;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Belgium;Poland;Singapore;Germany;New Zealand;Japan;Sweden | |||
| 68 | 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 |