DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
2	筋萎縮性側索硬化症	1
169	メンケス病	5
170	オクシピタル・ホーン症候群	1
171	ウィルソン病	18

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	NCT01259050 (ClinicalTrials.gov)	October 2010	10/12/2010	Safety Study of High Doses of Zinc in ALS Patients	Phase 1 Open Label Study of Zinc Therapy in ALS Patients	Amyotrophic Lateral Sclerosis	Drug: Zinc and Copper	Phoenix Neurological Associates, LTD	NULL	Completed	18 Years	85 Years	Both	10	Phase 1;Phase 2	United States

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	NCT04074512 (ClinicalTrials.gov)	August 31, 2019	27/8/2019	Copper Histidinate Treatment for Menkes Disease	Copper Histidinate Treatment for Menkes Disease	Menkes Disease	Drug: Copper Histidinate	Cyprium Therapeutics, Inc.	NULL	Available	N/A	6 Years	All			NULL
2	JPRN-UMIN000030626	2014/11/28	28/12/2017	Investigation on the effectiveness of histidine copper treatment of Menkes disease patients		Menkes disease	Weekly,subcutaneous injection of histidine copper (750microgram once) is performed three times per week.It is continued every week until the end of the research during the research period.	Hamamatsu University School of Medicine	NULL	Complete: follow-up complete	Not applicable	Not applicable	Male and Female	2	Not selected	Japan
3	JPRN-UMIN000005259	2011/03/03	18/03/2011	Clinical trial of histidine-copper and diethyldithiocarbamate combination therapy for patients with Menkes disease		Menkes disease	give a dose of copper-histidine and diethyldithiocarbamate	Kobe Unniversitu Graduate School of Medicine	NULL	Complete: follow-up complete	Not applicable	Not applicable	Male and Female	2	Not selected	Japan
4	NCT00811785 (ClinicalTrials.gov)	February 27, 2009	18/12/2008	Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency	Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency	Menkes Disease;Occipital Horn Syndrome;Unexplained Copper Deficiency	Drug: Copper Histidine	Cyprium Therapeutics, Inc.	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);National Center for Complementary and Integrative Health (NCCIH)	Completed	N/A	80 Years	All	93	Phase 3	United States
5	NCT00001262 (ClinicalTrials.gov)	June 1990	3/11/1999	Copper Histidine Therapy for Menkes Diseases	Early Copper Histidine Therapy in Menkes Disease	Kinky Hair Syndrome	Drug: Copper Histidine	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	NULL	Completed	N/A	N/A	All	60	Phase 1;Phase 2	United States

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	NCT00811785 (ClinicalTrials.gov)	February 27, 2009	18/12/2008	Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency	Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency	Menkes Disease;Occipital Horn Syndrome;Unexplained Copper Deficiency	Drug: Copper Histidine	Cyprium Therapeutics, Inc.	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD);National Center for Complementary and Integrative Health (NCCIH)	Completed	N/A	80 Years	All	93	Phase 3	United States

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	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
2	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	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
3	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 Kingdom
4	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	United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Australia;Denmark;Germany;New Zealand;Sweden;Korea, Republic of
5	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
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
6	NCT04422431 (ClinicalTrials.gov)	June 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	Not yet recruiting	18 Years	N/A	All	28	Phase 2	United States;Australia;Austria;Belgium;Germany;Korea, Republic of;New Zealand;Poland;Russian Federation;Singapore;Spain
7	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	gmp-orphan SA	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	55	Phase 3	United States;France;Poland;Brazil;Belgium;Austria;Denmark;Germany;United Kingdom;Italy;Sweden
8	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
9	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
10	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
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
11	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	gmp-orphan SA	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	55	Phase 3	Brazil;Belgium;Denmark;Austria;Germany;United Kingdom;United States;France;Poland;Italy;Sweden
12	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	gmp-orphan SA	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	55	Phase 3	United States;France;Poland;Brazil;Belgium;Austria;Denmark;Germany;United Kingdom;Italy;Sweden
13	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]		gmp-orphan SA	NULL	Not Recruiting			Female: yes Male: yes	55	Phase 3	France;Poland;Brazil;Denmark;Austria;Germany;Italy;United Kingdom
14	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
15	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
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
16	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
17	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
18	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

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

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

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

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

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

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

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

Phase 2

United Kingdom

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

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

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

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

Phase 2

United Kingdom

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

NCT04422431
(ClinicalTrials.gov)

June 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

Not yet recruiting

18 Years

N/A

All

Phase 2

United States;Australia;Austria;Belgium;Germany;Korea, Republic of;New Zealand;Poland;Russian Federation;Singapore;Spain

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

gmp-orphan SA

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

United States;France;Poland;Brazil;Belgium;Austria;Denmark;Germany;United Kingdom;Italy;Sweden

ChiCTR1900023410

2019-06-01

2019-05-26

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

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

Phase 4

Denmark

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

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

Phase 3

France;United States;Poland;Micronesia, Federated States of;Brazil;Denmark;Austria;Germany;United Kingdom;Italy

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

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

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

gmp-orphan SA

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

Brazil;Belgium;Denmark;Austria;Germany;United Kingdom;United States;France;Poland;Italy;Sweden

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

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

gmp-orphan SA

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

United States;France;Poland;Brazil;Belgium;Austria;Denmark;Germany;United Kingdom;Italy;Sweden

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]

gmp-orphan SA

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;Poland;Brazil;Denmark;Austria;Germany;Italy;United Kingdom

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

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

Phase 3

France;United States;Poland;Brazil;Belgium;Austria;Denmark;Germany;Italy;United Kingdom;Sweden

ChiCTR-DDD-17010552

2017-01-25

2017-02-04

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

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

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

Phase 2

France;Greece;Spain;Germany;United Kingdom;Italy

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

United States;Austria;Germany;Poland;United Kingdom

EUCTR2013-003564-31-DE
(EUCTR)

27/02/2014

22/10/2013

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

Phase 4

Germany;Italy