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.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-005266-34-ES
(EUCTR)
11/02/202206/09/2021A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
94Phase 1;Phase 2;Phase 3United States;Portugal;Spain;Austria;Italy;United Kingdom;France;Canada;Argentina;Brazil;Poland;Germany;Japan
2EUCTR2021-001015-82-DE
(EUCTR)
26/11/202110/08/2021Phase 3, open-label study of ALXN1840 versus standard of care in pediatric participants with Wilson diseaseA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
48Phase 3Serbia;United States;Czechia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Japan;Korea, Republic of
3EUCTR2021-001015-82-ES
(EUCTR)
22/11/202123/08/2021Phase 3, open-label study of ALXN1840 versus standard of care in pediatric participants with Wilson diseaseA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
48Phase 3United Kingdom;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Japan;Korea, Republic of;Serbia;United States;Czechia;Spain;Turkey;Austria;Russian Federation
4EUCTR2020-004604-33-PL
(EUCTR)
16/11/202104/06/2021Multicentre, 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 DiseaseOpen 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Phase 4Poland;Denmark;Germany
5EUCTR2020-000963-22-DE
(EUCTR)
18/10/202110/03/2021A clinical trial: treatment of adult Wilson's Disease patients with gene therapyA 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 SASNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 1;Phase 2United States;Denmark;Germany;United Kingdom
6EUCTR2020-004604-33-DK
(EUCTR)
08/10/202111/03/2021Multicentre, 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 DiseaseOpen 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Phase 4Poland;Denmark;Germany
7NCT05047523
(ClinicalTrials.gov)
October 6, 20219/9/2021Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson DiseaseA 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 DiseaseWilson DiseaseDrug: ALXN1840;Drug: Standard of CareAlexion PharmaceuticalsNULLRecruiting3 Years17 YearsAll48Phase 3Australia;Japan;Korea, Republic of;Poland;Spain
8NCT04884815
(ClinicalTrials.gov)
September 27, 20217/5/2021Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson DiseaseA 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 DiseaseWilson DiseaseGenetic: UX701;Other: Placebo;Drug: oral corticosteroids;Drug: Placebo for oral corticosteroidsUltragenyx Pharmaceutical IncNULLRecruiting18 YearsN/AAll90Phase 1/Phase 2United States
9EUCTR2020-000963-22-DK
(EUCTR)
05/07/202107/05/2021A clinical trial: treatment of adult Wilson's Disease patients with gene therapyA 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 SASNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 1;Phase 2United States;Denmark;Germany;United Kingdom
10EUCTR2020-005832-31-DK
(EUCTR)
13/04/202101/02/2021The 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
32Phase 2Denmark
11EUCTR2021-000102-25-DK
(EUCTR)
09/04/202101/02/2021The 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 HospitalNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 2Denmark
12EUCTR2020-004604-33-DE
(EUCTR)
23/02/202117/12/2020Multicentre, 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 DiseaseOpen 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Phase 4France;Poland;Denmark;Germany
13EUCTR2019-003711-60-DE
(EUCTR)
15/01/202102/06/2020A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840A 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 2United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Australia;Denmark;Germany;New Zealand;Sweden;Korea, Republic of
14NCT04422431
(ClinicalTrials.gov)
December 2, 202022/5/2020Copper Concentration and Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840A 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 WeeksWilson DiseaseDrug: Bis-Choline TetrathiomolybdateAlexion PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll31Phase 2United States;Denmark;New Zealand;Russian Federation;Singapore;Spain;United Kingdom;Australia;Austria;Belgium;Canada;Germany;Korea, Republic of;Poland
15EUCTR2019-003711-60-FR
(EUCTR)
22/10/202020/08/2020A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840A 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 2United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Australia;Denmark;Germany;New Zealand;Sweden;Korea, Republic of
16NCT04526210
(ClinicalTrials.gov)
October 21, 202019/8/2020Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy ParticipantsA 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 ParticipantsWilson DiseaseDrug: ALXN1840;Drug: Bupropion HydrochlorideAlexion PharmaceuticalsNULLCompleted18 Years50 YearsAll54Phase 1United States
17EUCTR2019-003711-60-AT
(EUCTR)
23/09/202001/10/2020A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840A 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, IncNULLNot RecruitingFemale: yes
Male: yes
28Phase 2United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Australia;Denmark;Germany;New Zealand;Sweden;Korea, Republic of
18NCT04573309
(ClinicalTrials.gov)
September 7, 202028/9/2020Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840Wilson DiseaseDrug: ALXN1840Alexion PharmaceuticalsNULLRecruiting18 YearsN/AAll10Phase 2United States;New Zealand;United Kingdom
19EUCTR2019-003711-60-DK
(EUCTR)
27/08/202019/06/2020A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840A 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 2United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Denmark;Australia;Germany;New Zealand;Sweden;Korea, Republic of
20EUCTR2019-003711-60-GB
(EUCTR)
26/08/202009/07/2020A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840A 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 2Korea, Republic of;Sweden;New Zealand;Germany;Denmark;Australia;Singapore;Poland;Belgium;Canada;France;United Kingdom;Russian Federation;Austria;Turkey;Spain;Serbia;United States
21EUCTR2020-001104-41-GB
(EUCTR)
22/07/202011/06/2020Copper Balance in Participants with Wilson Disease Treated with ALXN1840A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
10Phase 2United Kingdom
22NCT04526197
(ClinicalTrials.gov)
July 8, 202019/8/2020Phase 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 DiseaseDrug: ALXN1840;Drug: CelecoxibAlexion PharmaceuticalsNULLCompleted18 Years50 YearsAll36Phase 1United States
23EUCTR2017-004135-36-NL
(EUCTR)
30/03/202016/10/2019Study 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, IncNULLNot RecruitingFemale: yes
Male: yes
180Phase 3United 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
24EUCTR2017-004135-36-PT
(EUCTR)
13/01/202026/03/2019A 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, IncNULLNot RecruitingFemale: yes
Male: yes
180Phase 3United 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
25EUCTR2017-004135-36-SE
(EUCTR)
29/10/201916/04/2019A 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, IncNULLNot RecruitingFemale: yes
Male: yes
180Phase 3Serbia;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
26EUCTR2017-004135-36-DK
(EUCTR)
16/10/201905/08/2019A 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
180Phase 3Serbia;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
27EUCTR2016-003876-29-BE
(EUCTR)
31/07/201913/05/2019Chelate Study: Trientine for the treatment of Wilson's diseaseCHELATE 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
OrphalanNULLNot RecruitingFemale: yes
Male: yes
55Phase 3France;United States;Poland;Brazil;Belgium;Austria;Denmark;Germany;United Kingdom;Italy;Sweden
28ChiCTR1900023410
2019-06-012019-05-26The application of quantitative susceptibility mapping and blood and urine cell culture in Wilson's diseaseThe application of quantitative susceptibility mapping and blood and urine cell culture in Wilson's disease Wilson’s diseaseGold 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 HospitalNULLRecruitingBothTarget condition:40;Difficult condition:20China
29EUCTR2019-000905-57-DK
(EUCTR)
17/04/201914/03/2019Efficacy of zinc on copper uptake in the body depending on zinc type and dose regimen measured with 64CuCl2 PET/CT-scanEfficacy 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 HospitalNULLNot RecruitingFemale: yes
Male: yes
30Phase 4Denmark
30NCT03957720
(ClinicalTrials.gov)
March 15, 20198/4/2019The Individual Therapy for Patients With Wilson's DiseaseThe Individual Therapy for Patients With Wilson's DiseaseWilson's DiseaseDrug: DMPS;Drug: Penicillamine;Drug: DMSA;Drug: Zinc gluconateSecond Affiliated Hospital, School of Medicine, Zhejiang UniversityNULLRecruitingN/AN/AAll400Early Phase 1China
31EUCTR2017-004135-36-FR
(EUCTR)
14/09/201815/01/2018A 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 ABNULLAuthorised-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): noUnited States;Portugal;Spain;Austria;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Belgium;Poland;Denmark;Netherlands;Germany;Sweden
32NCT03659331
(ClinicalTrials.gov)
September 201828/8/2018A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson DiseaseA Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson DiseaseWilson DiseaseDietary Supplement: ZincProf. Elon PrasNULLUnknown status18 YearsN/AAll50N/ANULL
33NCT03539952
(ClinicalTrials.gov)
June 4, 20184/5/2018Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's DiseaseCHELATE STUDY: Trientine Tetrahydrochloride (TETA 4HCL) for the Treatment of Wilson's DiseaseWilson DiseaseDrug: TETA 4HCL;Drug: PenicillamineGMP-Orphan SASNULLActive, not recruiting18 Years75 YearsAll55Phase 3United States;Belgium;Brazil;Denmark;France;Germany;Italy;Poland;United Kingdom;Sweden
34EUCTR2017-004135-36-ES
(EUCTR)
27/04/201822/01/2018A 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 ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
102Phase 3France;United States;Hungary;Czech Republic;Poland;Spain;Austria;Israel;Germany;Italy;United Kingdom
35EUCTR2017-004135-36-GB
(EUCTR)
23/03/201819/12/2017A 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 ABNULLAuthorised-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): noUnited States;Portugal;Spain;Austria;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Belgium;Poland;Denmark;Netherlands;Germany;Sweden
36EUCTR2017-004135-36-DE
(EUCTR)
16/03/201822/12/2017A 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
180Phase 3Serbia;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
37EUCTR2017-004135-36-AT
(EUCTR)
27/02/201817/01/2018A 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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
180Phase 3Poland;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
38NCT03403205
(ClinicalTrials.gov)
February 15, 201819/12/2017Efficacy 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 MonthsA 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 MonthsWilson DiseaseDrug: ALXN1840;Drug: SoC TherapyAlexion PharmaceuticalsNULLActive, not recruiting12 YearsN/AAll215Phase 3United 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
39EUCTR2017-004135-36-HU
(EUCTR)
12/02/201807/02/2018A 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 ABNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
102Phase 3France;United States;Czech Republic;Hungary;Poland;Spain;Austria;Israel;Germany;Italy;United Kingdom
40NCT03299829
(ClinicalTrials.gov)
January 10, 201825/9/2017A Retrospective Study to Assess the Clinical Efficacy and Safety of Trientine in Wilson's Disease PatientsA Retrospective Study to Assess the Clinical Efficacy and Safety of Trientine in Wilson's Disease PatientsTrientine Treatment for Wilson's DiseaseDrug: TrientineExcelsiorNULLCompleted3 Years75 YearsAll48Taiwan
41EUCTR2016-003876-29-IT
(EUCTR)
13/12/201706/02/2018Chelate Study: Trientine for the treatment of Wilson's diseaseCHELATE 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 SANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
55Phase 3France;United States;Poland;Micronesia, Federated States of;Brazil;Denmark;Austria;Germany;United Kingdom;Italy
42EUCTR2016-003876-29-DE
(EUCTR)
15/09/201703/04/2017Chelate Study: Trientine for the treatment of Wilson's diseaseCHELATE 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
OrphalanNULLNot RecruitingFemale: yes
Male: yes
55Phase 3France;United States;Poland;Belgium;Brazil;Denmark;Austria;Germany;United Kingdom;Italy;Sweden
43EUCTR2016-003876-29-DK
(EUCTR)
16/05/201729/03/2017Chelate Study: Trientine for the treatment of Wilson's diseaseCHELATE 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
OrphalanNULLNot RecruitingFemale: yes
Male: yes
55Phase 3France;United States;Poland;Belgium;Brazil;Austria;Denmark;Germany;United Kingdom;Italy;Sweden
44EUCTR2016-003876-29-AT
(EUCTR)
12/04/201710/04/2017Chelate Study: Trientine for the treatment of Wilson's diseaseCHELATE 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 SANULLNot 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): noFrance;Poland;Brazil;Denmark;Austria;Germany;Italy;United Kingdom
45EUCTR2016-003876-29-GB
(EUCTR)
10/04/201704/04/2017Chelate Study: Trientine for the treatment of Wilson's diseaseCHELATE 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 SANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
55Phase 3France;United States;Poland;Brazil;Belgium;Austria;Denmark;Germany;Italy;United Kingdom;Sweden
46ChiCTR-DDD-17010552
2017-01-252017-02-04Genetic diagnosis of Wilson's diseaseGenetic diagnosis of Wilson's disease Wilson DiseaseGold 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 HospitalNULLRecruitingBothTarget condition:20;Difficult condition:0China
47EUCTR2013-003564-31-IT
(EUCTR)
14/07/201626/04/2018A 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 capsulesUNIVAR LTDNULLNot RecruitingFemale: yes
Male: yes
90Phase 2France;Greece;Spain;Germany;United Kingdom;Italy
48NCT02763215
(ClinicalTrials.gov)
May 19, 20163/5/2016The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care TreatmentMulti-Center Study for the Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care TreatmentWilson DiseaseDrug: Standard of Care MedicationsAlexion PharmaceuticalsNULLCompleted18 YearsN/AAll64United States;Austria;Germany;Poland;United Kingdom
49NCT02702765
(ClinicalTrials.gov)
February 201626/2/2016sCD163 and sMR in Wilsons Disease - Associations With Disease Severity and FibrosisMacrophages and the Macrophage Activation Markers sCD163 and Mannose Receptor (sMR) in Patients With Wilsons Disease - Associations With Liver Disease Severity and FibrosisWilsons DiseaseProcedure: Fibroscan;Procedure: Ultrasound;Drug: Galactose;Procedure: Liver biopsy;Procedure: Functional hepatic nitrogen clearanceUniversity of AarhusAarhus University HospitalActive, not recruiting18 Years100 YearsAll33N/ADenmark
50NCT02426905
(ClinicalTrials.gov)
January 201618/9/2014Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-PenicillamineMulticentre, Retrospective and Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine in Wilson Disease Patients Withdrawn From Therapy With d-PenicillamineWilson DiseaseDrug: trientine dihydrochlorideUnivar BVNULLActive, not recruiting1 Year90 YearsAll90Phase 4Germany;Greece;Italy;United Kingdom
51EUCTR2014-001703-41-GB
(EUCTR)
11/05/201529/01/2015A 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 MonthsA 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 ABNULLNot 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): noUnited States;Poland;Austria;Germany;United Kingdom
52EUCTR2014-001703-41-PL
(EUCTR)
22/12/201417/10/2014A 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 ABNULLNot RecruitingFemale: yes
Male: yes
30Phase 2United States;Poland;Austria;Germany;United Kingdom
53NCT02273596
(ClinicalTrials.gov)
November 24, 201420/10/2014Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease PatientsA 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 MonthsWilson DiseaseDrug: ALXN1840Alexion PharmaceuticalsNULLCompleted18 YearsN/AAll29Phase 2United States;Austria;Germany;Poland;United Kingdom
54EUCTR2014-001703-41-AT
(EUCTR)
21/08/201424/06/2014A 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.NULLNot 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): noUnited States;Poland;Austria;Germany;United Kingdom
55EUCTR2014-001703-41-DE
(EUCTR)
18/08/201405/06/2014A 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.NULLNot 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): noUnited States;Poland;Austria;Germany;United Kingdom
56EUCTR2013-003564-31-DE
(EUCTR)
27/02/201422/10/2013A 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.NULLNot RecruitingFemale: yes
Male: yes
90Phase 4Germany;Italy
57NCT01874028
(ClinicalTrials.gov)
May 20136/6/2013A Phase 1 Study to Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease PatientsA Phase 1 Pharmacokinetic Profiling Study in Patients Receiving Trientine Dihydrochloride for the Treatment of Wilson's Disease.Wilson's DiseaseDrug: trientine dihydrochlorideUnivar BVAptiv SolutionsCompleted6 Years90 YearsBoth20Phase 1Germany
58NCT01472874
(ClinicalTrials.gov)
January 201011/11/2011Single Daily Dosage of Trientine for Maintenance Treatment for Wilson DiseaseSingle Daily Dosage of Trientine for Maintenance Treatment for Wilson DiseaseWilson DiseaseDrug: Once a day TrientineYale UniversityBausch Health Americas, Inc.Completed18 YearsN/AAll8N/AUnited States
59NCT00212355
(ClinicalTrials.gov)
March 200513/9/2005Efficacy 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 DiseaseDrug: NPC-02NobelpharmaNULLCompleted1 YearN/AAll37Phase 3NULL
60NCT00212368
(ClinicalTrials.gov)
October 200413/9/2005Efficacy 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 DiseaseDrug: Zinc acetateNobelpharmaNULLCompleted1 YearN/ABothPhase 3NULL
61NCT04965545
(ClinicalTrials.gov)
January 1, 20045/7/2021Role for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson DiseaseRole for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson DiseaseWilson DiseaseDietary Supplement: low copper dietSecond Affiliated Hospital, School of Medicine, Zhejiang UniversityNULLRecruiting6 Years65 YearsAll1000China
62NCT00004339
(ClinicalTrials.gov)
January 199418/10/1999Study of Tetrathiomolybdate in Patients With Wilson DiseaseWilson DiseaseDrug: tetrathiomolybdate;Drug: trientineNational Center for Research Resources (NCRR)University of MichiganCompletedN/AN/ABoth90Phase 3United States
63NCT00004338
(ClinicalTrials.gov)
October 199318/10/1999Study of Zinc for Wilson DiseaseWilson DiseaseDrug: zinc acetateNational Center for Research Resources (NCRR)University of MichiganCompletedN/AN/ABoth300Phase 4United States
64EUCTR2020-004604-33-FR
(EUCTR)
12/03/2021Multicentre, 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 DiseaseOpen 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.NULLNAFemale: yes
Male: yes
50Phase 4France;Poland;Denmark;Germany
65EUCTR2017-004135-36-BE
(EUCTR)
17/06/2019A 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, IncNULLNot RecruitingFemale: yes
Male: yes
180Phase 3United 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
66EUCTR2017-004135-36-PL
(EUCTR)
03/04/2018A 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, IncNULLNAFemale: yes
Male: yes
180Phase 3Portugal;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
67EUCTR2021-001015-82-PL
(EUCTR)
15/09/2021Phase 3, open-label study of ALXN1840 versus standard of care in pediatric participants with Wilson diseaseA 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.NULLNAFemale: yes
Male: yes
48Phase 3Serbia;United States;Czechia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Japan;Korea, Republic of
68EUCTR2017-004135-36-CZ
(EUCTR)
09/01/2018A 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.NULLNAFemale: yes
Male: yes
180Phase 3Serbia;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