Choline ( DrugBank: Choline )
4 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 93 | 原発性胆汁性胆管炎 | 7 |
| 171 | ウィルソン病 | 29 |
| 193 | プラダー・ウィリ症候群 | 8 |
| 299 | 嚢胞性線維症 | 4 |
93. 原発性胆汁性胆管炎
臨床試験数 : 298 / 薬物数 : 252 - (DrugBank : 59) / 標的遺伝子数 : 35 - 標的パスウェイ数 : 115
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2020-001961-34-PL (EUCTR) | 24/02/2021 | 18/11/2020 | Clinical trial comparing the efficacy and safety of Rhudex granules with placebo in the treatment of the liver disease Primary Biliary Cholangitis | Double-blind, randomised, placebo-controlled, phase II dose-finding study comparing different doses of RhuDex granules with placebo in the treatment of primary biliary cholangitis - RhuDex vs placebo in PBC | Primary biliary cholangitis MedDRA version: 21.0;Level: PT;Classification code 10080429;Term: Primary biliary cholangitis;System Organ Class: 10019805 - Hepatobiliary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: RhuDex 25 mg (31.75 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 50 mg (63.5 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 100 mg (127 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt | Dr. Falk Pharma GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 136 | Phase 2 | Hungary;Slovakia;Belgium;Poland;Netherlands;Germany;United Kingdom | ||
| 2 | EUCTR2020-001961-34-SK (EUCTR) | 24/02/2021 | 02/10/2020 | Clinical trial comparing the efficacy and safety of Rhudex granules with placebo in the treatment of the liver disease Primary Biliary Cholangitis | Double-blind, randomised, placebo-controlled, phase II dose-finding study comparing different doses of RhuDex granules with placebo in the treatment of primary biliary cholangitis - RhuDex vs placebo in PBC | Primary biliary cholangitis MedDRA version: 21.0;Level: PT;Classification code 10080429;Term: Primary biliary cholangitis;System Organ Class: 10019805 - Hepatobiliary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: RhuDex 25 mg (31.75 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 50 mg (63.5 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 100 mg (127 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt | Dr. Falk Pharma GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 136 | Phase 2 | Hungary;Slovakia;Poland;Belgium;Netherlands;Germany;United Kingdom | ||
| 3 | EUCTR2020-001961-34-DE (EUCTR) | 02/02/2021 | 14/09/2020 | Clinical trial comparing the efficacy and safety of Rhudex granules with placebo in the treatment of the liver disease Primary Biliary Cholangitis | Double-blind, randomised, placebo-controlled, phase II dose-finding study comparing different doses of RhuDex granules with placebo in the treatment of primary biliary cholangitis - RhuDex vs placebo in PBC | Primary biliary cholangitis MedDRA version: 21.0;Level: PT;Classification code 10080429;Term: Primary biliary cholangitis;System Organ Class: 10019805 - Hepatobiliary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: RhuDex 25 mg (31.75 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 50 mg (63.5 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 100 mg (127 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt | Dr. Falk Pharma GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 136 | Phase 2 | Hungary;Slovakia;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom | ||
| 4 | EUCTR2020-001961-34-IT (EUCTR) | 25/01/2021 | 24/05/2021 | Clinical trial comparing the efficacy and safety of Rhudex granules with placebo in the treatment of the liver disease Primary Biliary Cholangitis | Double-blind, randomised, placebo-controlled, phase II dose-finding study comparing different doses of RhuDex granules with placebo in the treatment of primary biliary cholangitis - RhuDex vs placebo in PBC | Primary biliary cholangitis MedDRA version: 20.0;Level: SOC;Classification code 10019805;Term: Hepatobiliary disorders;System Organ Class: 10019805 - Hepatobiliary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: RhuDex 100 mg (127 mg RhuDex choline salt) granules Product Code: [RhuDex choline salt] INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 25 mg (31.75 mg RhuDex choline salt) granules Product Code: [RhuDex choline salt] INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 50 mg (63.5 mg RhuDex choline salt) granules Product Code: [RhuDex choline salt] INN or Proposed INN: RhuDex choline salt | DR. FALK PHARMA GMBH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 136 | Phase 2 | Czechia;Hungary;Slovakia;Spain;Poland;Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 5 | EUCTR2020-001961-34-NL (EUCTR) | 15/12/2020 | 08/10/2020 | Clinical trial comparing the efficacy and safety of Rhudex granules with placebo in the treatment of the liver disease Primary Biliary Cholangitis | Double-blind, randomised, placebo-controlled, phase II dose-finding study comparing different doses of RhuDex granules with placebo in the treatment of primary biliary cholangitis - RhuDex vs placebo in PBC | Primary biliary cholangitis MedDRA version: 21.0;Level: PT;Classification code 10080429;Term: Primary biliary cholangitis;System Organ Class: 10019805 - Hepatobiliary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: RhuDex 25 mg (31.75 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 50 mg (63.5 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 100 mg (127 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt | Dr. Falk Pharma GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 136 | Phase 2 | Hungary;Slovakia;Belgium;Germany;Netherlands;United Kingdom | ||
| 6 | EUCTR2020-001961-34-BE (EUCTR) | 11/12/2020 | 23/10/2020 | Clinical trial comparing the efficacy and safety of Rhudex granules with placebo in the treatment of the liver disease Primary Biliary Cholangitis | Double-blind, randomised, placebo-controlled, phase II dose-finding study comparing different doses of RhuDex granules with placebo in the treatment of primary biliary cholangitis - RhuDex vs placebo in PBC | Primary biliary cholangitis MedDRA version: 21.0;Level: PT;Classification code 10080429;Term: Primary biliary cholangitis;System Organ Class: 10019805 - Hepatobiliary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: RhuDex 25 mg (31.75 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 50 mg (63.5 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 100 mg (127 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt | Dr. Falk Pharma GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 136 | Phase 2 | Hungary;Slovakia;Belgium;Netherlands;Germany;United Kingdom | ||
| 7 | EUCTR2020-001961-34-HU (EUCTR) | 16/11/2020 | 17/09/2020 | Clinical trial comparing the efficacy and safety of Rhudex granules with placebo in the treatment of the liver disease Primary Biliary Cholangitis | Double-blind, randomised, placebo-controlled, phase II dose-finding study comparing different doses of RhuDex granules with placebo in the treatment of primary biliary cholangitis - RhuDex vs placebo in PBC | Primary biliary cholangitis MedDRA version: 21.0;Level: PT;Classification code 10080429;Term: Primary biliary cholangitis;System Organ Class: 10019805 - Hepatobiliary disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: RhuDex 25 mg (31.75 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 50 mg (63.5 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt Product Name: RhuDex 100 mg (127 mg RhuDex choline salt) granules INN or Proposed INN: RhuDex choline salt | Dr. Falk Pharma GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 136 | Phase 2 | Hungary |
171. ウィルソン病
臨床試験数 : 79 / 薬物数 : 77 - (DrugBank : 17) / 標的遺伝子数 : 6 - 標的パスウェイ数 : 30
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2021-001015-82-DE (EUCTR) | 26/11/2021 | 10/08/2021 | Phase 3, open-label study of ALXN1840 versus standard of care in pediatric participants with Wilson disease | A multicenter, randomized, controlled, open-label, rater-blinded study to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease. | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 3 | France;United States;Canada;Spain;Poland;Australia;Germany;United Kingdom;Japan;Korea, Republic of | ||
| 2 | EUCTR2021-001015-82-ES (EUCTR) | 22/11/2021 | 23/08/2021 | Phase 3, open-label study of ALXN1840 versus standard of care in pediatric participants with Wilson disease | A multicenter, randomized, controlled, open-label, rater-blinded study to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease. | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate | Alexion Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 48 | Phase 3 | United Kingdom;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Japan;Korea, Republic of;Serbia;United States;Czechia;Spain;Turkey;Austria;Russian Federation | ||
| 3 | EUCTR2020-005832-31-DK (EUCTR) | 13/04/2021 | 01/02/2021 | The effect of ALXN1840 on uptake of copper in the liver as measured by copper PET/CT scan. | Efficacy of ALXN1840 on human hepatic copper uptake quantified with 64CuCl2 PET/CT-scan. - ALXN1840-HV-Cu Absorption | Healthy Volunteers (Wilson's disease) MedDRA version: 22.0;Level: SOC;Classification code 10022891;Term: Investigations;System Organ Class: 10022891 - Investigations;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Cuprior 150 mg INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE Other descriptive name: Cuprior Trade Name: Metalcaptase Product Name: Metalcaptase INN or Proposed INN: PENICILLAMINE Product Name: ALXN1840 INN or Proposed INN: ALXN1840 Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE | Alexion Pharmaceuticals Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 32 | Phase 2 | Denmark | ||
| 4 | EUCTR2021-000102-25-DK (EUCTR) | 09/04/2021 | 01/02/2021 | The effect of ALXN1840 on excretion of copper in gall as measured by copper PET/MR-scan. | Efficacy of ALXN1840 on human biliary copper excretion quantified with 64CuCl2 PET/MR-scan - ALXN1840-WD-Cu Excretion | Wilson's Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: ALXN1840 INN or Proposed INN: ALXN1840 Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE Product Name: 64CuCl2 INN or Proposed INN: [64Cu]Cl2 Other descriptive name: COPPER (64CU) CHLORIDE | Aarhus University Hospital | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 2 | Denmark | ||
| 5 | EUCTR2019-003711-60-DE (EUCTR) | 15/01/2021 | 02/06/2020 | A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840 | A Phase 2, single-arm, pathologist-blinded study using liver biopsy specimens to assess copper concentration and histopathologic changes in patients with Wilson disease who are treated with ALXN1840 for 48 weeks followed by an extension treatment period with ALXN1840 for up to an additional 48 weeks | Wilson's Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 2 | United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Australia;Denmark;Germany;New Zealand;Sweden;Korea, Republic of | ||
| 6 | NCT04422431 (ClinicalTrials.gov) | December 2, 2020 | 22/5/2020 | Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840 | A Phase 2, Single-arm Pathologist-blinded 48-week Study Using Liver Biopsy Specimens to Assess Copper Concentration and Histopathologic Changes in ALXN1840-treated Patients With Wilson Disease Followed by an up to 48-weeks Extension Period | Wilson Disease | Drug: Bis-Choline Tetrathiomolybdate | Alexion Pharmaceuticals | NULL | Active, not recruiting | 18 Years | N/A | All | 31 | Phase 2 | United States;Denmark;New Zealand;Russian Federation;Singapore;Spain;United Kingdom;Australia;Austria;Belgium;Canada;Germany;Korea, Republic of;Poland |
| 7 | 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 | ||
| 8 | EUCTR2019-003711-60-AT (EUCTR) | 23/09/2020 | 01/10/2020 | A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840 | A Phase 2, single-arm, pathologist-blinded study using liver biopsy specimens to assess copper concentration and histopathologic changes in patients with Wilson disease who are treated with ALXN1840 for 48 weeks followed by an extension treatment period with ALXN1840 for up to an additional 48 weeks | Wilson's Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 28 | Phase 2 | United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Australia;Denmark;Germany;New Zealand;Sweden;Korea, Republic of | ||
| 9 | EUCTR2019-003711-60-DK (EUCTR) | 27/08/2020 | 19/06/2020 | A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840 | A Phase 2, single-arm, pathologist-blinded study using liver biopsy specimens to assess copper concentration and histopathologic changes in patients with Wilson disease who are treated with ALXN1840 for 48 weeks followed by an extension treatment period with ALXN1840 for up to an additional 48 weeks | Wilson's Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 2 | United States;Serbia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Canada;Belgium;Poland;Singapore;Denmark;Australia;Germany;New Zealand;Sweden;Korea, Republic of | ||
| 10 | 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 | ||
| 11 | 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 | ||
| 12 | EUCTR2017-004135-36-NL (EUCTR) | 30/03/2020 | 16/10/2019 | Study to Evaluate the Efficacy and Safety of ALXN1840 in pPtients with Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Portugal;Serbia;Taiwan;Hong Kong;Greece;Spain;Israel;Russian Federation;Colombia;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Belgium;Poland;Singapore;Croatia;Germany;Japan;New Zealand;Sweden | ||
| 13 | EUCTR2017-004135-36-PT (EUCTR) | 13/01/2020 | 26/03/2019 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Portugal;Serbia;Hong Kong;Taiwan;Spain;Israel;Russian Federation;Colombia;France;Denmark;Australia;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Canada;Brazil;Belgium;Poland;Singapore;Germany;Japan;New Zealand;Sweden | ||
| 14 | EUCTR2017-004135-36-SE (EUCTR) | 29/10/2019 | 16/04/2019 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | Serbia;United States;Portugal;Taiwan;Hong Kong;Spain;Russian Federation;Israel;Colombia;France;Australia;Denmark;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Belgium;Poland;Singapore;Germany;Japan;New Zealand;Sweden | ||
| 15 | EUCTR2017-004135-36-DK (EUCTR) | 16/10/2019 | 05/08/2019 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months - ALXN1840 Efficacy Safety administered for 48 Weeks Versus SOC in patients with Wilson Disease | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE Trade Name: Penicillamine INN or Proposed INN: PENICILLAMINE Trade Name: Trientine dihydrochloride INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE Trade Name: Wilzin INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 3 | Serbia;United States;Portugal;Taiwan;Hong Kong;Spain;Israel;Russian Federation;Colombia;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Poland;Belgium;Singapore;Germany;New Zealand;Japan;Sweden | ||
| 16 | EUCTR2017-004135-36-FR (EUCTR) | 14/09/2018 | 15/01/2018 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult patients who suffer from Wilson Disease. | A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older with an Extension Phase of up to 60 Months | Wilson Disease;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: WTX101 Product Code: WTX101 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Wilson Therapeutics AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 102 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Portugal;Spain;Austria;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Belgium;Poland;Denmark;Netherlands;Germany;Sweden | ||
| 17 | EUCTR2017-004135-36-ES (EUCTR) | 27/04/2018 | 22/01/2018 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult patients who suffer from Wilson Disease. | A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older with an Extension Phase of up to 60 Months | Wilson Disease;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: WTX101 Product Code: WTX101 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Wilson Therapeutics AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 102 | Phase 3 | France;United States;Hungary;Czech Republic;Poland;Spain;Austria;Israel;Germany;Italy;United Kingdom | ||
| 18 | EUCTR2017-004135-36-GB (EUCTR) | 23/03/2018 | 19/12/2017 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult patients who suffer from Wilson Disease. | A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older with an Extension Phase of up to 60 Months | Wilson Disease;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: WTX101 Product Code: WTX101 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE Other descriptive name: ZINC ACETATE DIHYDRATE | Wilson Therapeutics AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 102 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Portugal;Spain;Austria;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Belgium;Poland;Denmark;Netherlands;Germany;Sweden | ||
| 19 | EUCTR2017-004135-36-DE (EUCTR) | 16/03/2018 | 22/12/2017 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 3 | Serbia;United States;Portugal;Hong Kong;Taiwan;Greece;Spain;Russian Federation;Israel;Colombia;France;Australia;Denmark;Netherlands;Czechia;Korea, Democratic People's Republic of;Turkey;Austria;United Kingdom;Hungary;Canada;Belgium;Brazil;Poland;Singapore;Croatia;Germany;New Zealand;Japan;Sweden | ||
| 20 | EUCTR2017-004135-36-AT (EUCTR) | 27/02/2018 | 17/01/2018 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 180 | Phase 3 | Serbia;United States;Portugal;Hong Kong;Taiwan;Greece;Spain;Russian Federation;Israel;Colombia;France;Australia;Denmark;Netherlands;Czechia;Korea, Democratic People's Republic of;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Belgium;Brazil;Poland;Singapore;Croatia;Germany;New Zealand;Japan;Sweden | ||
| 21 | EUCTR2017-004135-36-HU (EUCTR) | 12/02/2018 | 07/02/2018 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of WTX101 in adult patients who suffer from Wilson Disease. | A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older with an Extension Phase of up to 60 Months | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: WTX101 Product Code: WTX101 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Wilson Therapeutics AB | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 102 | Phase 3 | France;United States;Czech Republic;Hungary;Poland;Spain;Austria;Israel;Germany;Italy;United Kingdom | ||
| 22 | EUCTR2014-001703-41-GB (EUCTR) | 11/05/2015 | 29/01/2015 | A clinical trial in adult Wilson Disease Patients to evaluate efficacy and safety of WTX101 following administration for 24 weeks with an Extension Phase of 12 Months | A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older with an Extension Phase of 12 Months - Wilson Disease Patients to evaluate efficacy and safety of WTX101 | Wilson Disease MedDRA version: 18.1;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850 | Product Code: WTX101 INN or Proposed INN: none Other descriptive name: bis-choline TETRATHIOMOLYBDATE | Wilson Therapeutics AB | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Poland;Austria;Germany;United Kingdom | ||
| 23 | EUCTR2014-001703-41-PL (EUCTR) | 22/12/2014 | 17/10/2014 | A clinical trial in adult Wilson Disease Patients to evaluate efficacy and safety of WTX101 following administration for 24 weeks and an extension phase of 36 months. | A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older with an Extension Phase of 36 Months | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: WTX101 INN or Proposed INN: none Other descriptive name: bis-choline TETRATHIOMOLYBDATE | Wilson Therapeutics AB | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | United States;Poland;Austria;Germany;United Kingdom | ||
| 24 | EUCTR2014-001703-41-AT (EUCTR) | 21/08/2014 | 24/06/2014 | A clinical trial in adult Wilson Disease Patients to evaluate efficacy and safety of WTX101 following administration for 24 weeks and an extension phase of 36 months. | A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older with an Extension Phase of 36 Months | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: WTX101 INN or Proposed INN: none Other descriptive name: bis-choline TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Poland;Austria;Germany;United Kingdom | ||
| 25 | EUCTR2014-001703-41-DE (EUCTR) | 18/08/2014 | 05/06/2014 | A clinical trial in adult Wilson Disease Patients to evaluate efficacy and safety of WTX101 following administration for 24 weeks and an extension phase of 36 months. | A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older with an Extension Phase of 36 Months | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Code: WTX101 INN or Proposed INN: none Other descriptive name: bis-choline TETRATHIOMOLYBDATE | Alexion Pharmaceuticals, INC. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Poland;Austria;Germany;United Kingdom | ||
| 26 | EUCTR2017-004135-36-BE (EUCTR) | 17/06/2019 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Portugal;Serbia;Taiwan;Hong Kong;Greece;Spain;Israel;Russian Federation;Colombia;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Belgium;Poland;Singapore;Croatia;Germany;Japan;New Zealand;Sweden | |||
| 27 | EUCTR2017-004135-36-PL (EUCTR) | 03/04/2018 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: TRIENTINE DIHYDROCHLORIDE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc | NULL | NA | Female: yes Male: yes | 180 | Phase 3 | Portugal;Serbia;United States;Hong Kong;Taiwan;Greece;Spain;Russian Federation;Israel;Colombia;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Poland;Belgium;Singapore;Croatia;Germany;New Zealand;Japan;Sweden | |||
| 28 | EUCTR2017-004135-36-CZ (EUCTR) | 09/01/2018 | A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease. | A Phase 3, Randomized, Rater- Blinder, Multi-Centre Study to Evaluate the Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care In Patients with Wilson Disease Aged 12 Years and Older With an Extension Period of Up to 60 months | Wilson Disease;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 INN or Proposed INN: Not applied Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE INN or Proposed INN: PENICILLAMINE INN or Proposed INN: ZINC ACETATE DIHYDRATE | Alexion Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 180 | Phase 3 | Serbia;United States;Portugal;Hong Kong;Taiwan;Spain;Russian Federation;Israel;Colombia;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Canada;Brazil;Belgium;Poland;Singapore;Germany;New Zealand;Japan;Sweden | |||
| 29 | EUCTR2021-001015-82-PL (EUCTR) | 15/09/2021 | Phase 3, open-label study of ALXN1840 versus standard of care in pediatric participants with Wilson disease | A multicenter, randomized, controlled, open-label, rater-blinded study to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease. | Wilson Disease MedDRA version: 20.0;Level: LLT;Classification code 10047988;Term: Wilson's disease;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate Product Name: ALXN1840 Product Code: ALXN1840 INN or Proposed INN: tiomolibdic acid Other descriptive name: BIS-CHOLINE TETRATHIOMOLYBDATE, bis[(2-Hydroxyethyl)trimethylammonium] tetrathiomolybdate | Alexion Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 48 | Phase 3 | Serbia;United States;Czechia;Spain;Turkey;Austria;Russian Federation;United Kingdom;France;Hungary;Canada;Poland;Australia;Denmark;Netherlands;Germany;Japan;Korea, Republic of |
193. プラダー・ウィリ症候群
臨床試験数 : 113 / 薬物数 : 111 - (DrugBank : 26) / 標的遺伝子数 : 48 - 標的パスウェイ数 : 102
| 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 | NCT05701774 (ClinicalTrials.gov) | January 31, 2023 | 18/1/2023 | Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome | An Open-Label Study of DCCR (Diazoxide Choline) Extended-Release Tablets in Patients With Prader-Willi Syndrome | Prader-Willi Syndrome | Drug: DCCR | Soleno Therapeutics, Inc. | NULL | Not yet recruiting | 4 Years | N/A | All | 83 | Phase 3 | NULL |
| 2 | NCT04086810 (ClinicalTrials.gov) | October 2019 | 10/9/2019 | An Open-Label Study of DCCR Tablet in Patients With PWS | An Open-Label Study of Diazoxide Choline Controlled-Release Tablet in Patients With Prader-Willi Syndrome | Prader-Willi Syndrome | Drug: DCCR | Soleno Therapeutics, Inc. | NULL | Withdrawn | 4 Years | N/A | All | 0 | Phase 3 | NULL |
| 3 | EUCTR2018-004216-22-GB (EUCTR) | 25/09/2019 | 27/06/2019 | A study to determine the safety of the study drug Diazoxide Choline Controlled-Release Tablet after being given for a long time to patients with the genetic disorder Prader-Willi Syndrome. | An Open-Label, Long-Term Safety and Efficacy Evaluation of Diazoxide Choline Controlled-Release Tablet in Patients with Prader-Willi Syndrome | Hyperphagia associated with Prader-Willi Syndrome (PWS) MedDRA version: 20.0;Level: PT;Classification code 10020710;Term: Hyperphagia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: diazoxide choline Product Code: DCCR INN or Proposed INN: Diazoxide choline Other descriptive name: DIAZOXIDE CHOLINE | Soleno Therapeutics UK Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Phase 3 | United States;United Kingdom | ||
| 4 | EUCTR2018-004215-50-GB (EUCTR) | 23/04/2019 | 12/08/2019 | A study to evaluate the effects of a medicine named diazoxide choline in patients with the genetic disorder Prader-Willi syndrome. | A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients with Prader-Willi Syndrome | Hyperphagia associated with Prader-Willi Syndrome. MedDRA version: 20.0;Level: PT;Classification code 10020710;Term: Hyperphagia;System Organ Class: 10027433 - Metabolism and nutrition disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: diazoxide choline Product Code: DCCR INN or Proposed INN: Diazoxide choline Other descriptive name: DIAZOXIDE CHOLINE | Soleno Therapeutics UK Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 105 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;United Kingdom | ||
| 5 | NCT03714373 (ClinicalTrials.gov) | October 1, 2018 | 27/9/2018 | Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period | An Open-Label, Long-Term Safety and Efficacy Evaluation of Diazoxide Choline Extended-Release Tablets in Participants With Prader-Willi Syndrome With a Double-Blind, Placebo-Controlled, Randomized Withdrawal Period | Prader-Willi Syndrome | Drug: DCCR;Drug: Placebo for DCCR | Soleno Therapeutics, Inc. | NULL | Active, not recruiting | 4 Years | N/A | All | 115 | Phase 3 | United States;United Kingdom |
| 6 | NCT03440814 (ClinicalTrials.gov) | May 9, 2018 | 13/2/2018 | A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome | A Randomized, Double-Blind, Placebo-Controlled Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome | Prader-Willi Syndrome | Drug: DCCR;Drug: Placebo for DCCR | Soleno Therapeutics, Inc. | NULL | Completed | 4 Years | N/A | All | 127 | Phase 3 | United States;United Kingdom |
| 7 | NCT02893618 (ClinicalTrials.gov) | July 2017 | 30/8/2016 | A 5 Treatment Period Pharmacokinetic Study Evaluating Dose Proportionality and Food Effects of Diazoxide Choline Controlled-Release Tablet (DCCR) | A 5 Treatment Period Crossover Pharmacokinetic Study Evaluating Dose Proportionality and Food Effects of Diazoxide Choline Controlled-Release Tablet (DCCR) | Prader-Willi Syndrome | Drug: Diazoxide choline controlled-release tablet | Essentialis, Inc. | NULL | Not yet recruiting | 18 Years | 65 Years | Both | 32 | Phase 2 | NULL |
| 8 | NCT02034071 (ClinicalTrials.gov) | April 2014 | 8/1/2014 | Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome | A Dose Titration Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome With a Double-Blind, Placebo-Controlled, Randomized Withdrawal Extension | Prader-Willi Syndrome | Drug: DCCR;Drug: Placebo | Essentialis, Inc. | NULL | Completed | 10 Years | 22 Years | Both | 13 | Phase 1/Phase 2 | United States |
299. 嚢胞性線維症
臨床試験数 : 1,695 / 薬物数 : 1,527 - (DrugBank : 268) / 標的遺伝子数 : 111 - 標的パスウェイ数 : 174
| 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 | NCT03312140 (ClinicalTrials.gov) | November 6, 2014 | 4/10/2017 | Examination of the Lipid Metabolism of the Liver After Choline Substitution in Cystic Fibrosis | Examination of the Lipid Metabolism of the Liver After Choline Substitution in Cystic Fibrosis | Cystic Fibrosis Liver Disease | Drug: Choline Chloride | University Hospital Tuebingen | NULL | Completed | 18 Years | N/A | Male | 10 | N/A | NULL |
| 2 | NCT01070446 (ClinicalTrials.gov) | October 2007 | 10/2/2010 | Choline Nutrition in Children With Cystic Fibrosis | Choline Nutrition in Children With Cystic Fibrosis | Cystic Fibrosis | Dietary Supplement: Vitamin: Choline Bitartrate (2-hydroxyethyl) trimethylammonium salt 1:1 | University of British Columbia | Cystic Fibrosis Foundation (US) | Completed | 5 Years | 17 Years | Both | 34 | N/A | Canada |
| 3 | NCT00686361 (ClinicalTrials.gov) | October 2007 | 26/5/2008 | Choline Nutrition in Children With Cystic Fibrosis (CF) | To Investigate Whether Choline Supplementation in Children With CF Will Correct Biochemical Markers of Choline Deficiency and Improve Plasma Indices of Methylation Capacity and Redox Status and Result in Decreased Pro-inflammatory Cytokines | Cystic Fibrosis | Drug: Choline supplementation | University of British Columbia | Cystic Fibrosis Foundation Therapeutics | Completed | 5 Years | 17 Years | Both | 34 | N/A | Canada |
| 4 | NCT00406536 (ClinicalTrials.gov) | January 2007 | 29/11/2006 | Study of LYM-X-SORB™ to Improve Fatty Acid and Choline Status in Children With CF and PI | Phase II Study: LYM-X-SORB™, an Organized Lipid Matrix: Fatty Acids and Choline in CF | Cystic Fibrosis;Pancreatic Insufficiency | Dietary Supplement: Lym-X-Sorb powder;Dietary Supplement: Placebo powder | Avanti Polar Lipids, Inc. | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Completed | 6 Years | 17 Years | Both | 110 | Phase 2 | United States |