79. 家族性高コレステロール血症(ホモ接合体) Homozygous familial hypercholesterolemia Clinical trials / Disease details


臨床試験数 : 142 薬物数 : 114 - (DrugBank : 29) / 標的遺伝子数 : 9 - 標的パスウェイ数 : 18

  
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PhaseCountries
1EUCTR2020-004394-49-NO
(EUCTR)
12/08/202125/05/2021Study to Evaluate the Long-Term Efficacy and Safety of the Investigational Drug LIB003 for the Reduction of Cholesterol in Patients with Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease.Open-Label Extension Phase 3 Study to Evaluate the Long-Term Efficacy and Safety of LIB003 in Patients With Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease, on Stable Lipid-Lowering Therapy Requiring Additional Low-Density Lipoprotein Cholesterol Reduction (LIBerate-OLE) - LIBerate-OLE Patients With Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease
MedDRA version: 20.0;Level: LLT;Classification code 10057079;Term: Heterozygous familial hypercholesterolemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850
MedDRA version: 20.1;Classification code 10007648;Term: Cardiovascular disease, unspecified;System Organ Class: 100000004849;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: LIB003
Product Code: lerodalcibep
INN or Proposed INN: Lerodalcibep
Other descriptive name: LIB003
LIB Therapeutics, LLCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
2350Phase 3France;United States;Canada;Spain;Turkey;South Africa;Israel;Germany;Norway;United Kingdom;New Zealand;India
2EUCTR2020-002755-38-NL
(EUCTR)
23/02/202126/12/2020Study to evaluate efficacy and safety of inclisiran in adolescents with homozygous familial hypercholesterolemiaTwo part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-13) Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: inclisiran sodium
Product Code: KJX839
INN or Proposed INN: inclisiran
Other descriptive name: Inclisiran sodium
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 3France;United States;Serbia;Slovenia;Greece;Canada;Lebanon;Turkey;Netherlands;Switzerland
3NCT04659863
(ClinicalTrials.gov)
February 16, 20212/12/2020Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial HypercholesterolemiaTwo Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Adolescents (12 to Less Than 18 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-13)Familial Hypercholesterolemia - HomozygousDrug: Inclisiran;Drug: PlaceboNovartis PharmaceuticalsNULLRecruiting12 Years17 YearsAll12Phase 3United States;Canada;France;Greece;Lebanon;Netherlands;Slovenia;Switzerland;Turkey
4EUCTR2020-002755-38-GR
(EUCTR)
02/02/202113/12/2020Two part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-13) Two part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-13) Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: inclisiran sodium
Product Code: KJX839
INN or Proposed INN: inclisiran
Other descriptive name: Inclisiran sodium
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 3France;United States;Serbia;Slovenia;Canada;Greece;Lebanon;Turkey;Netherlands;Switzerland
5EUCTR2020-002755-38-SI
(EUCTR)
30/12/202002/12/2020Study to evaluate efficacy and safety of inclisiran in adolescents with homozygous familial hypercholesterolemiaTwo part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-13) Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: inclisiran sodium
Product Code: KJX839
INN or Proposed INN: inclisiran
Other descriptive name: Inclisiran sodium
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 3Serbia;United States;Slovenia;Greece;Lebanon;Turkey;Italy;Switzerland;France;Canada;South Africa;Germany;Netherlands
6NCT04681170
(ClinicalTrials.gov)
November 23, 20207/12/2020Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)Phase III, Single Arm, Open Label, International, Multi Centre Study to Evaluate the Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) on Stable Lipid Lowering TherapyHomozygous Familial Hypercholesterolaemia (HoFH)Drug: LomitapideAmryt PharmaNULLRecruiting5 Years17 YearsAll45Phase 3Germany;Israel;Italy;Saudi Arabia;Spain;Tunisia
7EUCTR2019-002278-30-IT
(EUCTR)
11/11/202021/01/2021A clinical trial to assess the safety and effect of the drug lomitapide in the treatment of children with the condition Homozygous Familial Hypercholesterolaemia (HoFH) who are on Stable Lipid-lowering TherapyPhase III, single-arm, open-label, international, multi-centre study to evaluate the efficacy and safety of lomitapide in paediatric patients with Homozygous Familial Hypercholesterolaemia (HoFH) on stable lipid-lowering therapy - APH-19 Homozygous familial hypercholesterolaemia (HoFH). A rare and life-threatening inherited disorder of lipid metabolism with an estimated prevalence of 1 per 160,000 to 300,000 in the European population.
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Trade Name: Lojuxta 5 mg hard capsules
Product Name: Lojuxta 5 mg hard capsules
Product Code: [PRD7255830]
INN or Proposed INN: LOMITAPIDE
Trade Name: Lojuxta 10 mg hard capsules
Product Name: Lojuxta 10 mg hard capsules
Product Code: [PRD7255977]
INN or Proposed INN: LOMITAPIDE
Trade Name: Lojuxta 20 mg hard capsules
Product Name: Lojuxta 20 mg hard capsules
Product Code: [PRD7256003]
INN or Proposed INN: LOMITAPIDE
Product Name: Lomitapide 2mg hard capsules
Product Code: [Lomitapide 2mg hard capsules]
INN or Proposed INN: LOMITAPIDE
Amryt Pharmaceuticals DACNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
45Phase 3Saudi Arabia;Spain;Turkey;Israel;Germany;Tunisia;Italy
8EUCTR2019-001931-30-NL
(EUCTR)
03/11/202030/07/2020A trial to evaluate the Efficacy and Safety of Evinacumab in Paediatric Patients With Homozygous Familial HypercholesterolemiaA Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients with Homozygous Familial Hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 1;Phase 3United States;Taiwan;Ukraine;Lebanon;Austria;Italy;France;Canada;Jordan;Belgium;Australia;Netherlands;Germany;Norway
9NCT04455581
(ClinicalTrials.gov)
September 1, 202029/6/2020A Study to Determine the Safety, Tolerability, and Efficacy of SHR-1209 in Patients With Familial HypercholesterolemiaOpen-label, Single-arm, Multicentre Study to Evaluate Efficacy and Safety of SHR-1209 in Subjects With Homozygous Familial HypercholesterolemiaFamilial HypercholesterolemiaDrug: SHR-1209Jiangsu HengRui Medicine Co., Ltd.NULLNot yet recruiting18 Years80 YearsAll8Phase 2NULL
10NCT04515927
(ClinicalTrials.gov)
August 17, 202011/8/2020To Evaluate the Efficacy and Safety of JS002 in HoFH PatientsAn Open-label, Single-arm Study Evaluated the Efficacy and Safety of JS002 in Patients With Homozygous Familial HypercholesterolemiaHyperlipemiaDrug: JS002Shanghai Junshi Bioscience Co., Ltd.NULLActive, not recruiting12 Years75 YearsAll30Phase 2China
11EUCTR2019-001931-30-AT
(EUCTR)
17/08/202003/03/2020A trial to evaluate the Efficacy and Safety of Evinacumab in Paediatric Patients With Homozygous Familial HypercholesterolemiaA Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients with Homozygous Familial Hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
28Phase 1;Phase 3Australia;Germany;Netherlands;Norway;Belgium;Jordan;Canada;France;Italy;Austria;Lebanon;Ukraine;Taiwan;United States
12EUCTR2019-002278-30-DE
(EUCTR)
12/08/202011/09/2019A clinical trial to assess the safety and effect of the drug lomitapide in the treatment of children with the condition Homozygous Familial Hypercholesterolaemia (HoFH) who are on Stable Lipid-lowering TherapyPhase III, single-arm, open-label, international, multi-centre study to evaluate the efficacy and safety of lomitapide in paediatric patients with Homozygous Familial Hypercholesterolaemia (HoFH) on stable lipid-lowering therapy Homozygous familial hypercholesterolaemia (HoFH). A rare and life-threatening inherited disorder of lipid metabolism with an estimated prevalence of 1 per 160,000 to 300,000 in the European population.
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Trade Name: Lojuxta 5 mg hard capsules
INN or Proposed INN: LOMITAPIDE
Trade Name: Lojuxta 10 mg hard capsules
INN or Proposed INN: LOMITAPIDE
Trade Name: Lojuxta 20 mg hard capsules
INN or Proposed INN: LOMITAPIDE
Product Name: Lomitapide 2mg hard capsules
INN or Proposed INN: LOMITAPIDE
Amryt Pharmaceuticals DACNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
45Phase 3Saudi Arabia;Spain;Turkey;Israel;Tunisia;Germany;Italy
13EUCTR2019-004496-39-NL
(EUCTR)
12/08/202013/11/2020A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 Adults with homozygous familial hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]Product Name: AAV8.TBG.hLDLR
Product Code: AAV8.TBG.hLDLR
REGENXBIO Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 2United States;Canada;Netherlands;Italy
14NCT04233918
(ClinicalTrials.gov)
June 29, 20206/1/2020Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial HypercholesterolemiaA Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients With Homozygous Familial HypercholesterolemiaHomozygous Familial HypercholesterolemiaDrug: EvinacumabRegeneron PharmaceuticalsNULLActive, not recruiting5 Years11 YearsAll20Phase 3United States;Australia;Austria;Netherlands;Taiwan;Ukraine
15EUCTR2019-003611-62-NO
(EUCTR)
20/03/202028/11/2019Randomized, Open-Label, Phase 3 Study in which patients will receive LIB003 and Evolocumab to Evaluate the Efficacy and Safety in Homozygous Familial Hypercholesterolemia Patients on Stable Lipid-Lowering Therapy.Randomized, Open-Label, Cross-Over, Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 with Evolocumab in Homozygous Familial Hypercholesterolemia Patients on Stable Lipid-Lowering Therapy. - LIB003-003 Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Code: LIB003
INN or Proposed INN: not available
Other descriptive name: LIB003
Trade Name: Repatha (evolocumab)
INN or Proposed INN: EVOLOCUMAB
LIB Therapeutics, LLCNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
70Phase 3United States;Turkey;Israel;South Africa;Norway
16NCT04034485
(ClinicalTrials.gov)
December 7, 201922/7/2019Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFHRandomized, Open-Label, Cross-Over, Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in Homozygous Familial Hypercholesterolemia Patients on Stable Lipid-Lowering TherapyHomozygous Familial HypercholesterolemiaDrug: lerodalcibep;Drug: evolocumabLIB Therapeutics LLCNULLActive, not recruiting10 YearsN/AAll70Phase 3United States;India;Israel;Norway;South Africa;Turkey
17NCT04948008
(ClinicalTrials.gov)
November 5, 201929/6/2021Evaluate the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial HypercholesterolemiaA Seamless Phase IIb/III Clinical Study to Evaluate the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial Hypercholesterolemia in ChinaFamilial Hypercholesterolemia - Homozygous;Lipid Metabolism Disorders;Proprotein Convertase Subtilisin/Kexin 9Biological: IBI306Shenzhen People's HospitalNULLRecruiting18 Years80 YearsAll8Phase 2/Phase 3China
18NCT04080050
(ClinicalTrials.gov)
September 30, 20193/9/2019A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501Homozygous Familial Hypercholesterolemia (HoFH)Drug: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene TherapyRegenxbio Inc.NULLActive, not recruiting18 YearsN/AAll8United States;Canada;Netherlands;Italy
19NCT04031742
(ClinicalTrials.gov)
September 29, 201922/7/2019A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial HypercholesterolemiaPhase 2b/3 Study to Assess the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial Hypercholesterolemia. Part 1 - Open-label, Two-arm, Multicenter Pilot Study to Evaluate Efficacy and Safety of IBI 306 in Subjects With Homozygous Familial Hypercholesterolemia. Part 2 - Open-label, Single-arm, Multicenter Study to Evaluate Efficacy and Safety of IBI 306 in Subjects With Homozygous Familial HypercholesterolemiaHomozygous Familial HypercholesterolemiaBiological: IBI306Innovent Biologics (Suzhou) Co. Ltd.NULLRecruiting18 Years80 YearsAll30Phase 2/Phase 3China
20EUCTR2015-002276-25-PT
(EUCTR)
29/07/201915/11/2018Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Trade Name: Repatha 420 mg solution for injection in cartridge
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
115Phase 3Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;South Africa;Norway;Netherlands;Germany;New Zealand
21EUCTR2017-003170-13-AT
(EUCTR)
23/07/201911/02/2019Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab (REGN1500)
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
121Phase 3United States;Greece;Ukraine;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands;Japan
22EUCTR2015-002276-25-SI
(EUCTR)
10/06/201906/03/2019Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
Trade Name: Repatha 420 mg solution for injection in cartridge
Product Name: Evolocumab
Product Code: AMG 145
Amgen Europe B.V.NULLNot RecruitingFemale: yes
Male: yes
150Phase 3United States;Portugal;Taiwan;Greece;Spain;Ukraine;Russian Federation;Colombia;Italy;Switzerland;Malaysia;Australia;South Africa;Netherlands;Slovenia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Canada;Swaziland;Brazil;Belgium;Poland;Germany;Norway
23NCT03933293
(ClinicalTrials.gov)
May 13, 201928/4/2019A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFHA Phase 2 Study to Evaluate the Safety and Efficacy of PCSK9 Inhibitor AK102 in Patients With Homozygous Familial Hypercholesterolemia (HoFH)Homozygous Familial HypercholesterolemiaDrug: AK102;Drug: Statins;Drug: EzetimibeAkesoAD Pharmaceuticals Co., Ltd. (Guangzhou)Recruiting18 YearsN/AAll59Phase 2China
24NCT03814187
(ClinicalTrials.gov)
April 16, 201917/1/2019Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-CAn Open-label Extension Trial of the Phase III Lipid-lowering Trials to Assess the Effect of Long Term Dosing of Inclisiran Given as Subcutaneous Injections in Subjects With High Cardiovascular Risk and Elevated LDL-CASCVD;Elevated Cholesterol;Heterozygous Familial Hypercholesterolemia;Homozygous Familial HypercholesterolemiaDrug: Inclisiran SodiumNovartis PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll3275Phase 3United States;Canada;Czechia;Denmark;Germany;Hungary;Netherlands;Poland;South Africa;Spain;Sweden;Ukraine;United Kingdom
25EUCTR2017-003170-13-GB
(EUCTR)
12/03/201921/01/2019 Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial HypercholesterolemiaAn open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab (REGN1500)
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
100 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Greece;Ukraine;Turkey;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Norway;Netherlands;Germany;Japan
26EUCTR2017-003170-13-IT
(EUCTR)
18/02/201922/01/2021Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial HypercholesterolemiaAn open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia - na Homozygous familial hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850
MedDRA version: 20.0;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Trade Name: na
Product Name: Evinacumab (REGN1500)
Product Code: [REGN1500]
INN or Proposed INN: Evinacumab
Other descriptive name: Evinacumab
Trade Name: Praluent
Product Name: Praluent
Product Code: [na]
INN or Proposed INN: alirocumab
REGENERON PHARMACEUTICALS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 3United States;Czechia;Greece;Ukraine;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Norway;Germany;Netherlands;Japan
27NCT03851705
(ClinicalTrials.gov)
February 6, 20197/2/2019A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH)A Two-Part (Double-Blind Placebo Controlled/Open-Label) Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Subjects With Homozygous Familial Hypercholesterolemia (HoFH) (ORION-5)Homozygous Familial HypercholesterolemiaDrug: Inclisiran for injection;Drug: PlaceboNovartis PharmaceuticalsNULLCompleted18 Years80 YearsAll56Phase 3Hong Kong;Israel;Russian Federation;Serbia;South Africa;Taiwan;Turkey;Ukraine;Czechia
28EUCTR2017-003170-13-GR
(EUCTR)
01/02/201914/01/2019Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab (REGN1500)
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
121Phase 3United States;Czechia;Greece;Ukraine;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands;Japan
29EUCTR2015-002276-25-PL
(EUCTR)
25/01/201928/03/2019Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Trade Name: Repatha 420 mg solution for injection in cartridge
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
115Phase 3United States;Portugal;Taiwan;Greece;Spain;Ukraine;Russian Federation;Colombia;Italy;Malaysia;Australia;South Africa;Netherlands;Slovenia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Canada;Brazil;Poland;Belgium;Germany;Norway;New Zealand
30EUCTR2017-003170-13-CZ
(EUCTR)
17/12/201811/10/2018Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab (REGN1500)
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
121Phase 3United States;Greece;Ukraine;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands;Japan
31JPRN-JapicCTI-184099
30/9/201820/08/2018STUDY TO EVALUATE THE EFFICACY AND SAFETY OF EVINACUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIAA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF EVINACUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA Homozygous familial hypercholesterolemia (HoFH)Intervention name : Evinacumab
INN of the intervention : Evinacumab
Dosage And administration of the intervention : INTRAVENOUS Injection
Control intervention name : PLACEBO
INN of the control intervention : -
Dosage And administration of the control intervention : INTRAVENOUS Injection
Regeneron Pharmaceuticals, Inc.(ICCC: PAREXEL International)NULLcomplete18BOTH57NAJapan, North America, Europe, Oceania, Africa
32EUCTR2017-002297-39-SI
(EUCTR)
25/09/201820/06/2018An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial HypercholesterolemiaAn Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Sanofi-Aventis Recherche & DéveloppementNULLNot RecruitingFemale: yes
Male: yes
50Phase 3United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Norway;Netherlands
33EUCTR2017-002297-39-NO
(EUCTR)
19/09/201815/05/2018An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial HypercholesterolemiaAn Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Sanofi-Aventis Recherche & DéveloppementNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Netherlands;United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Norway
34EUCTR2017-002297-39-BG
(EUCTR)
11/09/201831/07/2018An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial HypercholesterolemiaAn Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Sanofi-Aventis Recherche & DéveloppementNULLNot RecruitingFemale: yes
Male: yes
50Phase 3United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway
35EUCTR2017-002297-39-AT
(EUCTR)
04/09/201807/06/2018An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial HypercholesterolemiaAn Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Sanofi-Aventis Recherche & DéveloppementNULLNot RecruitingFemale: yes
Male: yes
50Phase 3Austria;United States;Taiwan;Slovenia;Spain;Turkey;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway
36EUCTR2017-002297-39-DK
(EUCTR)
03/09/201804/06/2018An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial HypercholesterolemiaAn Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Trade Name: Praluent
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Trade Name: Praluent
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Sanofi-Aventis Recherche & DéveloppementNULLNot RecruitingFemale: yes
Male: yes
50Phase 3United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway
37NCT03510715
(ClinicalTrials.gov)
August 31, 201818/4/2018An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial HypercholesterolemiaAn Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial HypercholesterolemiaHypercholesterolemiaDrug: Alirocumab SAR236553 (REGN727);Drug: Rosuvastatin;Drug: Ezetimibe;Drug: Cholestyramine;Drug: Nicotinic acid;Drug: Fenofibrate;Drug: Omega-3 fatty acids;Drug: Atorvastatin;Drug: Simvastatin;Drug: Fluvastatin;Drug: Pravastatin;Drug: LovastatinSanofiRegeneron PharmaceuticalsCompleted8 Years17 YearsAll18Phase 3Brazil;Canada;Denmark;Mexico;Netherlands;Russian Federation;Slovenia;Spain;Taiwan;Turkey;Austria;France;Italy;Norway;United States
38EUCTR2017-002297-39-ES
(EUCTR)
28/08/201809/07/2018An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial HypercholesterolemiaAn Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders ;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Sanofi-Aventis Recherche & DéveloppementNULLNot Recruiting Female: yes
Male: yes
50 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Norway;Netherlands
39NCT03403374
(ClinicalTrials.gov)
August 4, 20183/1/2018Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial HypercholesterolemiaA Multicenter, Open-label, Single-arm, Study to Evaluate Safety and Tolerability of Repatha in Patients With Homozygous Familial Hypercholesterolemia (HoFH) in IndiaHomozygous Familial Hypercholesterolemia HoFHDrug: evolocumabAmgenNULLCompleted12 Years80 YearsAll30Phase 4India
40EUCTR2017-002297-39-FR
(EUCTR)
13/07/201816/05/2018An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial HypercholesterolemiaAn Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders ;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Sanofi-Aventis Recherche & DéveloppementNULLNot Recruiting Female: yes
Male: yes
50 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Norway;Netherlands
41EUCTR2017-002297-39-NL
(EUCTR)
02/07/201816/05/2018An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial HypercholesterolemiaAn Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Sanofi-Aventis Recherche & DéveloppementNULLNot RecruitingFemale: yes
Male: yes
50Phase 3United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway
42EUCTR2017-002297-39-IT
(EUCTR)
26/06/201823/05/2018An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial HypercholesterolemiaAn Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Product Name: Alirocumab
Product Code: SAR236553
INN or Proposed INN: Alirocumab
Other descriptive name: ALIROCUMAB
Sanofi-Aventis Recherche & DéveloppementNULLNot RecruitingFemale: yes
Male: yes
50Phase 3United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway
43EUCTR2017-001388-19-DE
(EUCTR)
18/06/201823/01/2018A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol levelA randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab (REGN1500)
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals Inc.NULLNot Recruiting Female: yes
Male: yes
57 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands
44EUCTR2017-001388-19-IT
(EUCTR)
11/05/201811/02/2021A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol levelA randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia - - Homozygous familial hypercholesterolemia
MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Trade Name: Praluent
Product Name: Alirocumab
Product Code: [REGN7272]
INN or Proposed INN: Alirocumab
Other descriptive name: Alirocumab
Product Name: Evinacumab
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: Evinacumab
REGENERON PHARMACEUTICALS, INC.NULLNot RecruitingFemale: yes
Male: yes
57Phase 3United States;Czechia;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands
45EUCTR2017-001388-19-GR
(EUCTR)
02/05/201802/04/2018A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol levelA randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab (REGN1500)
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals Inc.NULLNot RecruitingFemale: yes
Male: yes
65Phase 3United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Netherlands;Germany;Japan
46NCT03455777
(ClinicalTrials.gov)
April 12, 201822/2/2018Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Patients With Homozygous Familial Hypercholesterolemia (HoFH)Homozygous Familial HypercholesterolemiaDrug: AKCEA-ANGPTL3-LRXAkcea TherapeuticsIonis Pharmaceuticals, Inc.Withdrawn18 YearsN/AAll0Phase 2Canada
47EUCTR2017-001388-19-AT
(EUCTR)
23/03/201830/01/2018A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol levelA randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab (REGN1500)
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals Inc.NULLNot RecruitingFemale: yes
Male: yes
57Phase 3United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands
48EUCTR2017-003170-13-NL
(EUCTR)
15/03/201810/01/2018Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab (REGN1500)
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
121Phase 3United States;Greece;Ukraine;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Netherlands;Germany;Japan
49NCT03409744
(ClinicalTrials.gov)
March 13, 20188/1/2018Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial HypercholesterolemiaAn Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial HypercholesterolemiaHomozygous Familial HypercholesterolemiaDrug: evinacumabRegeneron PharmaceuticalsNULLActive, not recruiting12 YearsN/AAll116Phase 3United States;Australia;Austria;Canada;Czechia;France;Greece;Italy;Japan;Netherlands;South Africa;Ukraine;Norway
50NCT03399786
(ClinicalTrials.gov)
January 18, 20188/1/2018Efficacy and Safety of Evinacumab in Patients With Homozygous Familial HypercholesterolemiaA Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Evinacumab in Patients With Homozygous Familial HypercholesterolemiaHomozygous Familial HypercholesterolemiaDrug: evinacumab;Drug: PlaceboRegeneron PharmaceuticalsNULLCompleted12 YearsN/AAll65Phase 3United States;Australia;Austria;Canada;France;Greece;Italy;Japan;Netherlands;South Africa;Ukraine;Czechia;Germany;Turkey
51EUCTR2017-000351-95-DE
(EUCTR)
13/12/201717/08/2017A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol levelA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA - ODYSSEY HoFH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000012386;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
54Phase 3France;United States;Czech Republic;Greece;Canada;Ukraine;Turkey;Austria;South Africa;Germany;Italy
52EUCTR2017-000351-95-GR
(EUCTR)
08/11/201719/10/2017A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol levelA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA - ODYSSEY HoFH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
54Phase 3Germany;France;United States;Czech Republic;Canada;Greece;Ukraine;Turkey;Austria;South Africa;Italy
53EUCTR2017-000351-95-IT
(EUCTR)
12/10/201709/02/2021A study to the safety and efficacy of Alirocumab in patients with hereditary abnormal (high) cholesterol levelA Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to evaluate the efficacy and safety of Alirocumab in patients with Homozygous Familial Hypercholesterolemia - ODYSSEY HoFH Homozygous Familial Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Trade Name: Praluent
Product Name: R727
Product Code: [R727]
INN or Proposed INN: Alirocumab
Other descriptive name: Alirocumab
REGENERON PHARMACEUTICALS, INC.NULLNot RecruitingFemale: yes
Male: yes
55Phase 3United States;Czechia;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Denmark;South Africa;Germany
54NCT03156621
(ClinicalTrials.gov)
October 3, 201715/5/2017Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial HypercholesterolemiaHomozygous Familial HypercholesterolemiaDrug: Alirocumab;Drug: PlaceboRegeneron PharmaceuticalsSanofiCompleted18 YearsN/AAll69Phase 3United States;Austria;Canada;Czechia;France;Germany;Greece;Italy;Japan;South Africa;Taiwan;Turkey;Ukraine
55EUCTR2017-000351-95-AT
(EUCTR)
03/10/201722/08/2017A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol levelA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA - ODYSSEY HoFH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
54 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;Czech Republic;Greece;Canada;Ukraine;Turkey;Austria;South Africa;Germany;Italy
56EUCTR2016-001446-25-IT
(EUCTR)
02/10/201706/05/2021This is a study to investigate if gene therapy is safe in people with homozygous familial hypercholesterolemia (also called HoFH).AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) Adults with homozygous familial hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]Product Name: AAV8.TBG.hLDLR
Product Code: AAV8.TBG.hLDLR
Product Name: prednisone
Product Code: prednisone
REGENXBIO Inc.NULLNot RecruitingFemale: yes
Male: yes
12Phase 1;Phase 2United States;Canada;Netherlands;Italy
57EUCTR2016-001446-25-NL
(EUCTR)
20/09/201712/09/2016This is a study to investigate if gene therapy is safe in people with homozygous familial hypercholesterolemia (also called HoFH).AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) Adults with homozygous familial hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]Product Name: AAV8.TBG.hLDLR
Product Code: AAV8.TBG.hLDLR
University of PennsylvaniaNULLNot RecruitingFemale: yes
Male: yes
12Phase 1;Phase 2United States;Canada;Netherlands;Italy
58EUCTR2017-000351-95-FR
(EUCTR)
18/07/201723/06/2017A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol levelA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA - ODYSSEY HoFH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
54Phase 3United States;Portugal;Serbia;Greece;Spain;Ukraine;Israel;Italy;France;Denmark;South Africa;Tunisia;Netherlands;Slovenia;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Poland;Croatia;Bulgaria;Germany;Sweden
59EUCTR2016-003376-49-NL
(EUCTR)
20/06/201718/01/2017A study to evaluate the effect of ALN-PCSSC treatment in patients with Homozygous Familial HypercholesterolemiaAn Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects with Homozygous Familial Hypercholesterolemia - ORION-2 Homozygous Familial Hypercholesterolemia
MedDRA version: 19.1;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: ALN-PCSSC
Product Code: ALN-PCSSC
INN or Proposed INN: ALN-60212
Other descriptive name: ALN-60212
The Medicines CompanyNULLNot RecruitingFemale: yes
Male: yes
10Phase 2United States;South Africa;Netherlands
60EUCTR2015-002276-25-NL
(EUCTR)
11/04/201722/11/2016Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Trade Name: Repatha 420 mg solution for injection in cartridge
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
124Phase 3Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;South Africa;Norway;Netherlands;Germany;New Zealand
61EUCTR2015-002276-25-ES
(EUCTR)
27/02/201705/08/2016Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 19.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 19.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
115Phase 3Greece;Spain;Turkey;Austria;Colombia;Hungary;Czech Republic;Canada;Brazil;Australia;South Africa;Netherlands;Germany;New Zealand
62NCT02963311
(ClinicalTrials.gov)
December 13, 201610/11/2016A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)Homozygous Familial HypercholesterolemiaDrug: ALN-PCSSC;Drug: Standard of CareThe Medicines CompanyNULLCompleted12 YearsN/AAll9Phase 2United States;Netherlands;South Africa
63EUCTR2015-002276-25-DE
(EUCTR)
21/10/201605/04/2016Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) Hypercholesterolemia
MedDRA version: 18.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 18.1;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
115Phase 3Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;Italy;United Kingdom;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;South Africa;Netherlands;Germany;Norway;New Zealand
64EUCTR2015-002276-25-CZ
(EUCTR)
06/10/201604/05/2016Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Trade Name: Repatha 420 mg solution for injection in cartridge
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
115Phase 3Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;United Kingdom;Czech Republic;Hungary;Canada;Poland;Belgium;Brazil;Australia;South Africa;Netherlands;Germany;Norway;New Zealand
65EUCTR2015-004003-23-IT
(EUCTR)
28/09/201627/05/2016A controlled study to assess the safety, tolerability and activity of the study drug ISIS 703802 given subcutaneously to Healthy Volunteers with high triglycerides and patients with Familial HypercholesterolemiaA Placebo-Controlled, Dose-Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of ISIS 703802, Targeting ANGPTL3, Administered Subcutaneously to Healthy Volunteers with Elevated Triglycerides andSubjects with Familial Hypercholesterolemia Heterozygous familial hypercholesterolemia and Homozygous familial hypercholesterolemia
MedDRA version: 19.0;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850
MedDRA version: 19.0;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;Classification code 10057079;Term: Heterozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: ISIS 703802
Product Code: ISIS 703802
INN or Proposed INN: ISIS 703802
Other descriptive name: ISIS 703802
Ionis Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
61Phase 1;Phase 2Canada;Netherlands;Italy
66NCT02624869
(ClinicalTrials.gov)
September 10, 201622/10/2015Open Label Study to Evaluate Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Pediatric Subjects (10 to 17 Years of Age) With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH).Open-label, Single-Arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH)Familial HypercholesterolemiaBiological: evolocumab (AMG 145)AmgenNULLCompleted10 Years17 YearsAll163Phase 3United States;Australia;Austria;Belgium;Brazil;Canada;Colombia;Czechia;Greece;Hungary;Italy;Malaysia;Netherlands;Norway;Poland;Portugal;Russian Federation;Slovenia;South Africa;Spain;Switzerland;Turkey;United Kingdom
67EUCTR2015-002276-25-IT
(EUCTR)
25/07/201615/06/2021Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozyg Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg soluzione per iniezione in penna preriempita.
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: Evolocumab
Trade Name: Repatha 420 mg soluzione per iniezione in cartuccia
Product Name: Evolocumab
Product Code: [AMG 145]
INN or Proposed INN: EVOLOCUMAB
AMGEN INC.NULLNot RecruitingFemale: yes
Male: yes
115Phase 3Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;South Africa;Norway;Netherlands;Germany;New Zealand
68EUCTR2016-000411-32-NL
(EUCTR)
15/07/201617/05/2016A study to the safety and efficacy of REGN1500 in patients with Hereditary abnormal (high) cholesterol levelAN OPEN-LABEL, SINGLE-ARM, PROOF-OF-CONCEPT STUDY TO EVALUATE THE SAFETY AND EFFICACY OF SINGLE AND MULTIPLE DOSES OF REGN1500 IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA Homozygous Familial Hypercholesterolemia
MedDRA version: 19.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: evinacumab
Product Code: REGN1500
INN or Proposed INN: EVINACUMAB
Other descriptive name: EVINACUMAB
Product Name: Evinacumab
Product Code: REGN1500
INN or Proposed INN: EVINACUMAB
Other descriptive name: EVINACUMAB
Product Name: evinacumab
Product Code: REGN1500
INN or Proposed INN: EVINACUMAB
Other descriptive name: EVINACUMAB
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
8Phase 2United States;Canada;Netherlands
69EUCTR2014-003806-33-GR
(EUCTR)
30/06/201624/03/2016A clinical study in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy conducted to assess safety, tolerability and efficacy of Lomitapide A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy Homozygous familial hypercholesterolemia (HoFH).
MedDRA version: 18.1;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: lomitapide
Product Code: AEGR-733
INN or Proposed INN: LOMITAPIDE
Product Name: lomitapide
Product Code: AEGR-733
INN or Proposed INN: LOMITAPIDE
Product Name: lomitapide
Product Code: AEGR-733
INN or Proposed INN: LOMITAPIDE
Product Name: lomitapide
Product Code: AEGR-733
INN or Proposed INN: LOMITAPIDE
Aegerion Pharmaceuticals Inc.NULLNot RecruitingFemale: yes
Male: yes
45Phase 3Canada;Greece;Turkey;Austria;Israel;Germany;Italy
70EUCTR2015-002276-25-AT
(EUCTR)
27/06/201612/04/2016Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Trade Name: Repatha 420 mg solution for injection in cartridge
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
115Phase 3Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;Czech Republic;Hungary;Canada;Poland;Belgium;Brazil;Australia;South Africa;Germany;Netherlands;Norway;New Zealand
71JPRN-UMIN000022603
2016/06/0604/06/2016Non-randomized, prospective, non-controlled study of changes of lipid parameters after introduction of evolocumab in patients with heterozygous familial hypercholesterolemia under LDL-apheresis therapy. Homozygous familial hypercholesterolemiaSubcutaneous injection of evolocumabKanazawa University HospitalNULLComplete: follow-up complete20years-old100years-oldMale and Female2Not selectedJapan
72EUCTR2015-002276-25-HU
(EUCTR)
06/06/201612/04/2016Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 19.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 19.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
115Phase 3Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;United Kingdom;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;South Africa;Netherlands;Germany;Norway;New Zealand
73EUCTR2015-002276-25-GR
(EUCTR)
01/06/201616/05/2016Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
115Phase 3Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;Italy;United Kingdom;Czech Republic;Hungary;Canada;Poland;Belgium;Brazil;Australia;South Africa;Netherlands;Germany;Norway;New Zealand
74NCT02722408
(ClinicalTrials.gov)
June 201612/3/2016Efficacy and Safety of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid-Lowering Therapy (COBALT-1)A Phase 2 Open-Label, Dose-Finding Study to Assess the Efficacy, Safety, and Tolerability of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid Lowering Therapy (COBALT-1)HypercholesteremiaDrug: GemcabeneNeuroBo Pharmaceuticals Inc.NULLCompleted17 YearsN/AAll8Phase 2United States;Canada;Israel
75EUCTR2015-002276-25-BE
(EUCTR)
23/05/201613/04/2016Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Trade Name: Repatha 420 mg solution for injection in cartridge
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
115Phase 3Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;Czech Republic;Hungary;Canada;Poland;Belgium;Brazil;Australia;South Africa;Norway;Netherlands;Germany;New Zealand
76NCT02765841
(ClinicalTrials.gov)
May 201627/4/2016Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering TherapyA Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering TherapyHomozygous Familial HypercholesterolemiaDrug: LomitapideAegerion Pharmaceuticals, Inc.NULLWithdrawn5 Years17 YearsAll0Phase 3NULL
77NCT02651675
(ClinicalTrials.gov)
March 20164/1/2016A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)Homozygous Familial Hypercholesterolemia (HoFH)Biological: AAV directed hLDLR gene therapyRegenxbio Inc.National Heart, Lung, and Blood Institute (NHLBI)Completed18 YearsN/AAll9Phase 1/Phase 2United States;Canada;Italy;Netherlands
78NCT02434497
(ClinicalTrials.gov)
June 6, 20157/4/2015A Study to Evaluate the Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial HypercholesterolemiaAn Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (Aged 6 to <18 Years) With Homozygous Familial Hypercholesterolemia (HoFH)Homozygous Familial Hypercholesterolemia (HoFH)Drug: Rosuvastatin 20mgAstraZenecaNULLCompleted6 Years18 YearsAll9Phase 3Belgium;Canada;Denmark;Israel;Malaysia;Taiwan
79NCT02399852
(ClinicalTrials.gov)
June 201523/3/2015Effects of Lomitapide on Carotid and Aortic AtherosclerosisEffects of Lomitapide on Carotid and Aortic Atherosclerosis in Patients Treated With Lomitapide in Usual Care (CAPTURE)Homozygous Familial HypercholesterolemiaDrug: LomitapideAegerion Pharmaceuticals, Inc.NULLWithdrawn18 YearsN/AAll0N/ANULL
80EUCTR2014-004856-68-NO
(EUCTR)
29/04/201526/01/2015A 12-week, open-label , all subjects will first receive 2 weeks of placebo drug and then active medication in a dose escalating manner: each patient will first receive 50 mg, after evaluation the option exists to increase the dose to to 100 mg and after another evaluation to 200 mg active study drug, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH)A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) Homozygous Familial Hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]Product Name: MBX-8025
Product Code: MBX-8025
INN or Proposed INN: MBX-8025
CymaBay Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
8Phase 2France;Netherlands;Norway
81EUCTR2014-004856-68-NL
(EUCTR)
23/04/201528/01/2015A 12-week, open-label , all subjects will first receive 2 weeks of placebo drug and then active medication in a dose escalating manner: each patient will first receive 50 mg, after evaluation the option exists to increase the dose to to 100 mg and after another evaluation to 200 mg active study drug, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH)A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) Homozygous Familial Hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]Product Name: MBX-8025
Product Code: MBX-8025
INN or Proposed INN: MBX-8025
CymaBay Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
8Phase 2France;Norway;Netherlands
82NCT02472535
(ClinicalTrials.gov)
April 201522/5/2015Study to Evaluate the Effects of MBX-8025 in Patients With HoFHA 12-week, Open-label, Dose-escalating, Phase 2 Study to Evaluate the Effects of MBX-8025 in Patients With Homozygous Familial Hypercholesterolemia (HoFH)Homozygous Familial HypercholesterolemiaOther: Run-In Period: Placebo;Drug: MBX-8025 50 mg (Dose Escalation Period 1);Drug: MBX-8025 50 mg or 100 mg (Dose Escalation Period 2);Drug: MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3)CymaBay Therapeutics, Inc.NULLCompleted18 YearsN/ABoth13Phase 2Canada;France;Netherlands;Norway
83EUCTR2014-004746-99-DK
(EUCTR)
27/03/201527/03/2015A study to evluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia.An Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH).
MedDRA version: 19.0;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Crestor Film-Coated Tablet
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: Rosuvastatin calcium
AstraZeneca ABNULLNot RecruitingFemale: yes
Male: yes
20Phase 3United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Netherlands;Germany;Sweden
84EUCTR2014-004856-68-FR
(EUCTR)
13/03/201519/05/2015A 12-week, open-label , all subjects will first receive 2 weeks of placebo drug and then active medication in a dose escalating manner: each patient will first receive 50 mg, after evaluation the option exists to increase the dose to to 100 mg and after another evaluation to 200 mg active study drug, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH)A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) Homozygous Familial Hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]Product Name: MBX-8025
Product Code: MBX-8025
INN or Proposed INN: MBX-8025
CymaBay Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
8Phase 2France;Norway;Netherlands
85EUCTR2014-004746-99-BE
(EUCTR)
23/02/201518/12/2014A study to evluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia.An Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH).;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Trade Name: Crestor Film-Coated Tablet
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: Rosuvastatin calcium
AstraZeneca ABNULLNot RecruitingFemale: yes
Male: yes
20Phase 3United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Netherlands;Germany;Sweden
86NCT02265952
(ClinicalTrials.gov)
February 4, 201510/10/2014Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)An Open-Label, Single-Arm, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Single and Multiple Doses of REGN1500 in Patients With Homozygous Familial HypercholesterolemiaHomozygous Familial HypercholesterolemiaDrug: REGN1500 250 mg SC/15 mg/kg IV/450 mg SCRegeneron PharmaceuticalsNULLCompleted18 YearsN/AAll9Phase 2United States;Canada;Netherlands
87EUCTR2014-000972-24-DK
(EUCTR)
17/12/201425/09/2014A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial HypercholesterolemiaA Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH)
MedDRA version: 17.0;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin
AstraZeneca ABNULLNot RecruitingFemale: yes
Male: yes
25United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Germany;Netherlands;Sweden
88EUCTR2014-000972-24-DE
(EUCTR)
17/11/201426/09/2014A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial HypercholesterolemiaA Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH)
MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin
Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin
AstraZeneca ABNULLNot RecruitingFemale: yes
Male: yes
25United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Denmark;Australia;South Africa;Germany;Netherlands;Norway;Sweden
89NCT02226198
(ClinicalTrials.gov)
November 201414/8/2014A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial HypercholesterolemiaA Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (Aged 6 to <18 Years) With Homozygous Familial Hypercholesterolemia (HoFH)Homozygous Familial Hypercholesterolemia (HoFH)Drug: Rosuvastatin 20mg;Drug: PlaceboAstraZenecaNULLCompleted6 Years17 YearsAll20Phase 3Belgium;Canada;Denmark;Israel;Malaysia;Netherlands;Taiwan;Germany;Lebanon;Sweden;United States
90EUCTR2014-000972-24-BE
(EUCTR)
27/10/201411/09/2014A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial HypercholesterolemiaA Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH)
MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin
AstraZeneca ABNULLNot RecruitingFemale: yes
Male: yes
25Phase 3United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden
91EUCTR2014-000972-24-NL
(EUCTR)
20/10/201405/08/2014A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial HypercholesterolemiaA Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH)
MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin
AstraZeneca ABNULLNot RecruitingFemale: yes
Male: yes
25United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden
92EUCTR2014-000972-24-SE
(EUCTR)
20/08/201430/06/2014A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial HypercholesterolemiaA Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH)
MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin
AstraZeneca ABNULLNot RecruitingFemale: yes
Male: yes
25United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden
93NCT02173158
(ClinicalTrials.gov)
April 2, 201423/6/2014Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering TherapyA Phase 3, Single-Arm, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering TherapyFamilial Hypercholesterolemia - HomozygousDrug: lomitapideAegerion Pharmaceuticals, Inc.NULLCompleted18 YearsN/AAll9Phase 3Japan
94NCT02135705
(ClinicalTrials.gov)
March 18, 201410/4/2014LOWER: Lomitapide Observational Worldwide Evaluation RegistryLOWER: Lomitapide Observational Worldwide Evaluation RegistryHomozygous Familial HypercholesterolemiaDrug: LomitapideAmryt PharmaNULLRecruiting18 YearsN/AAll300United States
95EUCTR2011-005400-15-IT
(EUCTR)
19/12/201322/04/2013A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL Homozygous Familial Hypercholesterolemia
MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
INN or Proposed INN: NA
Other descriptive name: AMG 145
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
125Phase 2France;Czech Republic;Canada;Poland;Belgium;Brazil;Malaysia;Australia;South Africa;Netherlands;Norway;New Zealand;Japan;United States;Hong Kong;Greece;Spain;Turkey;Austria;Israel;United Kingdom;Italy
96EUCTR2011-005400-15-NL
(EUCTR)
24/09/201304/07/2013A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutationsA Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL Familial Hypercholesterolemia
MedDRA version: 18.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
Other descriptive name: AMG 145
Product Name: AMG 145
Other descriptive name: AMG145
Product Name: AMG 145
Other descriptive name: AMG145
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
300Phase 2;Phase 3United States;Hong Kong;Greece;Spain;Lebanon;Turkey;Austria;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Malaysia;Belgium;Brazil;Australia;South Africa;Netherlands;New Zealand;Japan
97EUCTR2011-005399-40-NL
(EUCTR)
24/09/201303/07/2013A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemiaA two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Homozygous familial hypercholesterolaemia
MedDRA version: 16.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
Other descriptive name: AMG 145
Amgen IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
59Phase 2/3United States;Hong Kong;Spain;Lebanon;Turkey;Italy;France;Czech Republic;Canada;Brazil;Belgium;Malaysia;South Africa;Netherlands;New Zealand
98EUCTR2012-002434-37-IT
(EUCTR)
16/07/201305/06/2013n.a.A Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-WeekStudy to Assess the Efficacy and Tolerability of Anacetrapib When Added to OngoingLipid-Lowering Therapy in Adult Patients with Homozygous FamilialHypercholesterolemia (HoFH) - n.a. Homozygous Familial Hypercholesterolemia (HoFH)
MedDRA version: 14.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Product Name: Anacetrapib
Product Code: MK-0859
INN or Proposed INN: ANACETRAPIB
Other descriptive name: ANACETRAPIB
Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., IncNULLNot RecruitingFemale: yes
Male: yes
45Phase 3United States;Czech Republic;Canada;Malaysia;Brazil;Israel;South Africa;Norway;United Kingdom;Italy
99EUCTR2011-005399-40-ES
(EUCTR)
24/06/201309/04/2013A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemiaA two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Homozygous familial hypercholesterolaemia
MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
INN or Proposed INN: n/a
Other descriptive name: AMG 145
Amgen IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
67Phase 2/3United States;Hong Kong;Spain;Turkey;Austria;France;Czech Republic;Canada;Belgium;Brazil;Malaysia;South Africa;New Zealand
100EUCTR2011-005400-15-ES
(EUCTR)
18/06/201309/04/2013A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with Severe Familial HypercholesterolemiaA Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL Homozygous Familial Hypercholesterolemia
MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
INN or Proposed INN: n/a
Other descriptive name: AMG 145
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
125Phase 2United States;Hong Kong;Greece;Spain;Turkey;Austria;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Malaysia;Poland;Belgium;Brazil;Australia;South Africa;Netherlands;Norway;Japan;New Zealand
101EUCTR2012-002434-37-CZ
(EUCTR)
13/06/201328/12/2012n/aA Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-WeekStudy to Assess the Efficacy and Tolerability of Anacetrapib When Added to OngoingLipid-Lowering Therapy in Adult Patients with Homozygous FamilialHypercholesterolemia (HoFH) with a 52-Week Open-Label Extension Homozygous Familial Hypercholesterolemia (HoFH);Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]Product Name: MK-0859
Product Code: (4S,5R)-5-[3,5-bis(trifluoromethyl)phenyl]-3-{[4’-
Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., IncNULLNot RecruitingFemale: yes
Male: yes
45United States;Czech Republic;Canada;Malaysia;Australia;Israel;South Africa;Norway;United Kingdom
102EUCTR2011-005399-40-IT
(EUCTR)
08/06/201323/04/2013A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemiaA two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Homozygous familial hypercholesterolaemia
MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
INN or Proposed INN: NA
Other descriptive name: AMG 145
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
67Phase 2/3United States;Hong Kong;Spain;Turkey;Austria;Italy;France;Czech Republic;Canada;Belgium;Brazil;Malaysia;South Africa;Netherlands;New Zealand
103NCT01841684
(ClinicalTrials.gov)
June 201324/4/2013Efficacy and Tolerability of Anacetrapib Added to Ongoing Lipid-Lowering Therapy in Adult Participants With Homozygous Familial Hypercholesterolemia (HoFH) (MK-0859-042)A Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-Week Study to Assess the Efficacy and Tolerability of Anacetrapib When Added to Ongoing Lipid-Lowering Therapy in Adult Patients With Homozygous Familial Hypercholesterolemia (HoFH)Hyperlipoproteinemia Type II;Homozygous Familial HypercholesterolemiaDrug: Anacetrapib;Drug: PlaceboMerck Sharp & Dohme Corp.NULLTerminated18 YearsN/ABoth2Phase 3United States;Canada;Germany;Israel;Italy;United Kingdom;Norway
104EUCTR2011-005400-15-CZ
(EUCTR)
30/05/201327/02/2013A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutationsA Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL Familial Hypercholesterolemia
MedDRA version: 19.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: evolocumab
Product Code: AMG 145
INN or Proposed INN: Evolocumab
Other descriptive name: AMG 145
Trade Name: Repatha 140 mg solution for injection in pre-filled pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: Evolocumab
Other descriptive name: AMG145
Product Name: Evolocumab
Product Code: AMG 145
Other descriptive name: AMG145
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
300Phase 2;Phase 3United States;Hong Kong;Greece;Spain;Lebanon;Austria;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;South Africa;Netherlands;New Zealand;Japan
105EUCTR2012-002434-37-GB
(EUCTR)
23/05/201323/11/2012n/aA Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-Week Study to Assess the Efficacy and Tolerability of Anacetrapib When Added to Ongoing Lipid-Lowering Therapy in Adult Patients with Homozygous Familial Hypercholesterolemia (HoFH) with a 52-Week Open-Label Extension - Anacetrapib in Patients with Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia (HoFH);Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]Product Name: MK-0859
Product Code: (4S,5R)-5-[3,5-bis(trifluoromethyl)phenyl]-3-{[4’-
Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., IncNULLNot Recruiting Female: yes
Male: yes
45 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Czech Republic;Canada;Malaysia;Australia;Israel;South Africa;Norway;Italy;United Kingdom
106EUCTR2011-005400-15-GB
(EUCTR)
22/05/201304/03/2013 A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutationsA Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL Familial Hypercholesterolemia
MedDRA version: 19.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
Other descriptive name: AMG 145
Product Name: AMG 145
Other descriptive name: AMG145
Product Name: AMG 145
Other descriptive name: AMG145
Amgen IncNULLNot Recruiting Female: yes
Male: yes
300 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSouth Africa;Netherlands;New Zealand;Japan;Australia;Brazil;Belgium;Malaysia;Canada;Czech Republic;France;United Kingdom;Italy;Israel;Austria;Turkey;Lebanon;Spain;Greece;Hong Kong;United States
107EUCTR2011-005400-15-AT
(EUCTR)
07/05/201320/03/2013A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutationsA Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL Homozygous Familial Hypercholesterolemia
MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
Other descriptive name: AMG 145
Amgen IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
125United States;Hong Kong;Greece;Spain;Turkey;Austria;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Brazil;Malaysia;Australia;South Africa;Norway;New Zealand;Japan
108EUCTR2011-005399-40-CZ
(EUCTR)
03/05/201326/02/2013A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemiaA two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Homozygous familial hypercholesterolaemia
MedDRA version: 16.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
Other descriptive name: AMG 145
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
59Phase 2;Phase 3United States;Hong Kong;Spain;Lebanon;Turkey;Italy;France;Czech Republic;Canada;Brazil;Belgium;Malaysia;South Africa;Netherlands;New Zealand
109NCT02286596
(ClinicalTrials.gov)
April 20133/11/2014Comparisons of Two Low-density Lipoprotein Apheresis Systems in Patients With Homozygous Familial HypercholesterolemiaComparisons of Two Low-density Lipoprotein Apheresis Systems in Patients With Homozygous Familial HypercholesterolemiaHomozygous Familial HypercholesterolemiaDevice: heparin-induced extracorporeal LDL precipitation;Device: dextran sulfate adsorptionLaval UniversityNULLCompleted18 Years60 YearsBoth9N/ACanada
110EUCTR2012-002434-37-NO
(EUCTR)
21/11/201226/11/2012n/aA Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-WeekStudy to Assess the Efficacy and Tolerability of Anacetrapib When Added to OngoingLipid-Lowering Therapy in Adult Patients with Homozygous FamilialHypercholesterolemia (HoFH) with a 52-Week Open-Label Extension Homozygous Familial Hypercholesterolemia (HoFH);Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]Product Name: MK-0859
Product Code: (4S,5R)-5-[3,5-bis(trifluoromethyl)phenyl]-3-{[4’-
Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., IncNULLNot RecruitingFemale: yes
Male: yes
45United States;Czech Republic;Canada;Malaysia;Australia;Israel;South Africa;Norway;United Kingdom
111EUCTR2011-005400-15-GR
(EUCTR)
05/09/201209/08/2012A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutationsA Multicenter, Single Arm, Open-label Study to Assess the Long-term Safety and Efficacy of AMG 145 on LDL-C in Subjects With Homozygous Familial Hypercholesterolemia or PCSK9 Mutations Homozygous Familial Hypercholesterolemia
MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
Other descriptive name: AMG 145
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
75Phase 2United States;Czech Republic;Canada;Greece;Spain;Belgium;South Africa;Netherlands;Italy;United Kingdom
112NCT01588496
(ClinicalTrials.gov)
April 5, 201227/2/2012Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities2-part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia. Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia. Part B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaHomozygous Familial HypercholesterolemiaBiological: Evolocumab;Drug: PlaceboAmgenNULLCompleted12 Years80 YearsAll58Phase 2/Phase 3United States;Belgium;Canada;Czechia;France;Hong Kong;Italy;Lebanon;Netherlands;New Zealand;South Africa;Spain;Czech Republic
113EUCTR2011-005400-15-BE
(EUCTR)
22/03/201207/02/2012A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutationsA Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL Familial Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000012386;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
Other descriptive name: AMG 145
Product Name: AMG 145
Other descriptive name: AMG145
Product Name: AMG 145
Other descriptive name: AMG145
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
300Phase 2;Phase 3United States;Hong Kong;Greece;Spain;Lebanon;Turkey;Austria;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Malaysia;Belgium;Brazil;Australia;South Africa;Netherlands;New Zealand;Japan
114EUCTR2011-005399-40-BE
(EUCTR)
09/03/201207/02/2012A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemiaA two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Homozygous familial hypercholesterolaemia
MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
Other descriptive name: AMG 145
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
59Phase 2;Phase 3United States;Hong Kong;Spain;Lebanon;Turkey;Italy;France;Czech Republic;Canada;Malaysia;Belgium;Brazil;South Africa;Netherlands;New Zealand
115EUCTR2011-003998-28-NL
(EUCTR)
28/02/201214/11/2011Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemiaModifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE homozygous familial hypercholesterolemia
MedDRA version: 16.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Recombinant Human Apolipoprotein A-I/Phospholipids Complex
Product Code: CER-001
Other descriptive name: recombinant human apolipoprotein A-I
Other descriptive name: sphingomyelin
Other descriptive name: 1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol)
Cerenis TherapeuticsNULLNot RecruitingFemale: yes
Male: yes
30France;United States;Canada;Netherlands;Italy;United Kingdom
116EUCTR2011-003998-28-IT
(EUCTR)
16/02/201208/03/2012Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemiaModifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE Homozygous familial hypercholesterolemia
MedDRA version: 15.1;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Recombinant Human Apolipoprotein
Product Code: CER-001
Other descriptive name: Recombinant Human Apolipoprotein A-I
Other descriptive name: Sfingomielina
Other descriptive name: 1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol)
CERENIS THERAPEUTICS SANULLNot RecruitingFemale: yes
Male: yes
30United States;Canada;Netherlands;United Kingdom;Italy
117EUCTR2011-003998-28-GB
(EUCTR)
24/01/201219/10/2011Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemiaModifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE homozygous familial hypercholesterolemia
MedDRA version: 15.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 10027433 - Metabolism and nutrition disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Recombinant Human Apolipoprotein A-I/Phospholipids Complex
Product Code: CER-001
INN or Proposed INN: recombinant human apolipoprotein a1
Other descriptive name: recombinant human apolipoprotein A-I
INN or Proposed INN: sphingomyelin
Other descriptive name: sphingomyelin
INN or Proposed INN: DPPG
Other descriptive name: 1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol)
Cerenis TherapeuticsNULLNot 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;Canada;Netherlands;Italy;United Kingdom
118NCT01412034
(ClinicalTrials.gov)
November 20115/8/2011Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) SubjectsModifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-label Study of the Effects of CER-001 on Plaque Volume in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)Homozygous Familial HypercholesterolemiaDrug: CER-001Cerenis Therapeutics, SANULLCompleted12 YearsN/ABoth23Phase 2United States;Canada;Italy;Netherlands;United Kingdom
119EUCTR2010-023742-79-IT
(EUCTR)
25/03/201121/02/2011A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - NDA Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - ND Homozygous Familial Hypercholesterolemia
MedDRA version: 9.1;Level: LLT;Classification code 10057080
Product Name: Lomitapide
Product Code: AEGR-733
Product Name: Lomitapide
Product Code: AEGR-733
AEGERION PHARMACEUTICALSNULLNot RecruitingFemale: yes
Male: yes
23Phase 3Italy
120NCT00943306
(ClinicalTrials.gov)
October 29, 200921/7/2009Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial HypercholesterolemiaA Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor 'Lomitapide' (LOMITAPIDE) in Patients With Homozygous Familial HypercholesterolemiaFamilial HypercholesterolemiaDrug: lomitapideAegerion Pharmaceuticals, Inc.NULLCompleted18 YearsN/AAll19Phase 3United States;Canada;Italy;South Africa
121EUCTR2008-007058-36-IT
(EUCTR)
25/06/200903/06/2009A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients with Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy - NDA Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients with Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy - ND Homozygous Familial Hypercholesterolemia
MedDRA version: 9.1;Level: LLT;Classification code 10048373;Term: Hypercholesterolaemia aggravated
Product Code: AEGR-733
Product Code: AEGR-733
Aegerion PharmaceuticalsNULLNot RecruitingFemale: yes
Male: yes
25Phase 3Italy
122NCT00730236
(ClinicalTrials.gov)
December 20076/8/2008A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients With Homozygous Familial Hypercholesterolemia on Current Lipid-lowering TherapyHomozygous Familial HypercholesterolemiaDrug: AEGR-733Aegerion Pharmaceuticals, Inc.FDA Office of Orphan Products DevelopmentCompleted18 YearsN/AAll29Phase 3United States;Canada;Italy;South Africa
123EUCTR2005-003449-15-GB
(EUCTR)
12/07/200709/03/2007A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia SubjectsA Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects - RADICHOL I Reduction of low density lipoprotein C (LDL-C) in Homozygous Familial Hypercholesterolemia (HoFH)
MedDRA version: 9.1;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia
Product Name: ISIS 301012
Product Code: ISIS 301012
Other descriptive name: ISIS 301012 phosphorothioate oligonucleotide
Genzyme Europe B.V.NULLNot Recruiting Female: yes
Male: yes
50 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited Kingdom
124NCT00607373
(ClinicalTrials.gov)
July 200722/1/2008Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial HypercholesterolemiaA Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Mipomersen as Add-on Therapy in Homozygous Familial Hypercholesterolemia SubjectsLipid Metabolism, Inborn Errors;Hypercholesterolemia, Autosomal Dominant;Hyperlipidemias;Metabolic Diseases;Hyperlipoproteinemia Type II;Metabolism, Inborn Errors;Genetic Diseases, Inborn;Infant, Newborn, Diseases;Metabolic Disorder;Congenital Abnormalities;Hypercholesterolemia;Hyperlipoproteinemias;Dyslipidemias;Lipid Metabolism DisordersDrug: mipomersen;Drug: PlaceboKastle Therapeutics, LLCIonis Pharmaceuticals, Inc.Completed12 YearsN/AAll51Phase 3United States;Brazil;Canada;Singapore;South Africa;Taiwan;United Kingdom;Netherlands
125EUCTR2005-003626-26-GB
(EUCTR)
14/06/200601/11/2005A double-blind, placebo-controlled, randomized study to evaluate the efficacy and safety of TAK-475 or placebo when co-administered with current lipid-lowering therapy in subjects with homozygous familial hypercholesterolemiaA double-blind, placebo-controlled, randomized study to evaluate the efficacy and safety of TAK-475 or placebo when co-administered with current lipid-lowering therapy in subjects with homozygous familial hypercholesterolemia Treatment of primary dyslipidemia
MedDRA version: 8;Level: PT;Classification code 10058108
Product Name: TAK-475
Product Code: TAK-475
Takeda Europe R&D Centre LtdNULLNot RecruitingFemale: yes
Male: yes
40United Kingdom
126NCT00704535
(ClinicalTrials.gov)
March 200623/6/2008Evaluation of the Safety, Tolerability and Efficacy of Ezetimibe on a Select Population of Filipinos With Hypercholesterolemia (Study P04748)(COMPLETED)Post-marketing Surveillance of the Safety, Tolerability and Efficacy of Ezetimibe Among Filipino PatientsPrimary Hypercholesterolemia;Homozygous Familial HypercholesterolemiaDrug: EzetimibeOrganon and CoNULLCompleted18 YearsN/AAll4105Philippines
127NCT00280995
(ClinicalTrials.gov)
January 200620/1/2006Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering TherapyA Phase 2, Open-Label, Dose Escalation Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia SubjectsHypercholesterolemia, FamilialDrug: ISIS 301012Kastle Therapeutics, LLCIonis Pharmaceuticals, Inc.Completed18 YearsN/ABoth12Phase 2United States;Netherlands
128NCT00263081
(ClinicalTrials.gov)
November 20056/12/2005Efficacy of Lapaquistat Acetate Co-Administered With Current Lipid-Lowering Treatment on Blood Cholesterol Levels in Subjects With Homozygous Familial HypercholesterolemiaA Double-blind, Placebo-controlled, Randomized Study to Evaluate the Efficacy and Safety of TAK-475 or Placebo When Co-administered With Current Lipid-lowering Therapy in Subjects With Homozygous Familial Hypercholesterolemia.HypercholesterolemiaDrug: Lapaquistat acetate and current lipid-lowering treatment;Drug: Current lipid-lowering treatmentTakedaNULLTerminated8 YearsN/ABoth44Phase 3United States;Canada;France;Israel;Poland;United Kingdom
129NCT00079846
(ClinicalTrials.gov)
September 200317/3/2004Implitapide in Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering TherapySafety and Efficacy Study of Implitapide Compared With Placebo in Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering TherapyFamilial HypercholesterolemiaDrug: ImplitapideMedical Research Laboratories InternationalNULLTerminated8 Years70 YearsBoth60Phase 2United States;Canada;Israel;Netherlands;Norway
130NCT01556906
(ClinicalTrials.gov)
June 20037/3/2012Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) InhibitorA Phase II Open Label, Dose-Escalation Study to Determine the Safety, Tolerability and Efficacy of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor BMS-201038 in Patients With Homozygous Familial HypercholeterolemiaHomozygous Familial HypercholesterolemiaDrug: LomitapideAegerion Pharmaceuticals, Inc.University of Pennsylvania;Doris Duke Charitable FoundationCompleted13 YearsN/AAll6Phase 2United States
131NCT00092833
(ClinicalTrials.gov)
July 200223/9/2004Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED)An Open-Label, Worldwide, Treatment Use Study to Provide Ezetimibe 10 Mg/Day to Patients With Homozygous Familial Hypercholesterolemia or Homozygous SitosterolemiaHypercholesterolemia, Familial;Lipid Metabolism, Inborn ErrorsDrug: Comparator: ezetimibeOrganon and CoNULLTerminated8 YearsN/AAll49Phase 3United States
132NCT03885921
(ClinicalTrials.gov)
October 25, 200020/3/2019Safety and Tolerability Study of Ezetimibe (SCH 058235/MK-0653) Plus Atorvastatin or Simvastatin in Homozygous Familial Hypercholesterolemia (P01417/MK-0653-019)Long-Term, Open-Label, Safety and Tolerability Study of SCH 58235 in Addition to Atorvastatin or Simvastatin in the Therapy of Homozygous Familial HypercholesterolemiaHypercholesterolemiaDrug: Ezetimibe;Drug: Atorvastatin;Drug: SimvastatinOrganon and CoNULLCompleted12 YearsN/AAll44Phase 3NULL
133NCT03884452
(ClinicalTrials.gov)
May 3, 20006/3/2019Ezetimibe (SCH 58235) Taken With Either Atorvastatin or Simvastatin in Participants With Familial Hypercholesterolemia (MK-0653-018)A Phase III Efficacy And Safety Study of Ezetimibe (SCH58235) 10 mg in Addition to Atorvastatin or Simvastatin in the Therapy of Homozygous Familial HypercholesterolemiaFamilial HypercholesterolemiaDrug: Atorvastatin;Drug: Simvastatin;Drug: Ezetimibe;Drug: Placebo for EzetimibeOrganon and CoNULLCompleted12 YearsN/AAll50Phase 3NULL
134EUCTR2017-003170-13-FR
(EUCTR)
26/10/2018 Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab (REGN1500)
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals, Inc.NULLNA Female: yes
Male: yes
100 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Greece;Ukraine;Turkey;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Norway;Netherlands;Germany;Japan
135EUCTR2014-004746-99-SE
(EUCTR)
23/12/2014A study to evluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia.An Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH).;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Trade Name: Crestor Film-Coated Tablet
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: Rosuvastatin calcium
AstraZeneca ABNULLNot RecruitingFemale: yes
Male: yes
20Phase 3United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Netherlands;Germany;Sweden
136EUCTR2015-002276-25-GB
(EUCTR)
05/04/2016Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Trade Name: Repatha 420 mg solution for injection in cartridge
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNAFemale: yes
Male: yes
115Phase 3Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Italy;Czech Republic;Hungary;Canada;Poland;Belgium;Brazil;Australia;South Africa;Norway;Germany;Netherlands;New Zealand
137EUCTR2017-001388-19-CZ
(EUCTR)
01/02/2018A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol levelA randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab (REGN1500)
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals Inc.NULLNot RecruitingFemale: yes
Male: yes
57Phase 3United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands
138EUCTR2015-002276-25-NO
(EUCTR)
11/04/2016Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial HypercholesterolemiaOpen-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE Hypercholesterolemia
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Trade Name: Repatha 420 mg solution for injection in cartridge
Product Name: Evolocumab
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNot RecruitingFemale: yes
Male: yes
115Phase 3South Africa;Netherlands;Norway;Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;Italy;United Kingdom;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;Germany;New Zealand
139EUCTR2017-001388-19-FR
(EUCTR)
24/01/2018A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol levelA randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia Homozygous familial hypercholesterolemia
MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Product Name: Evinacumab (REGN1500)
Product Code: REGN1500
INN or Proposed INN: Evinacumab
Other descriptive name: EVINACUMAB
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals Inc.NULLNA Female: yes
Male: yes
57 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands
140EUCTR2011-005399-40-Outside-EU/EEA
(EUCTR)
17/12/2014A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemiaA two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia - TESLA Homozygous familial hypercholesterolaemia
MedDRA version: 17.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: AMG 145
Product Code: AMG 145
INN or Proposed INN: EVOLOCUMAB
Amgen IncNULLNAFemale: yes
Male: yes
59Phase 2/3South Africa;Turkey;United States;Brazil;Canada;Hong Kong;Lebanon;Malaysia;New Zealand
141EUCTR2017-000351-95-CZ
(EUCTR)
28/07/2017A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol levelA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA - ODYSSEY HoFH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000012386;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Trade Name: Praluent
INN or Proposed INN: ALIROCUMAB
Other descriptive name: ALIROCUMAB
Regeneron Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
54Phase 3Serbia;Portugal;United States;Slovenia;Greece;Spain;Ukraine;Turkey;Austria;Israel;Italy;United Kingdom;Czech Republic;Canada;Poland;Belgium;Croatia;Denmark;South Africa;Bulgaria;Netherlands;Germany;Tunisia;Sweden
142EUCTR2020-004394-49-DE
(EUCTR)
27/05/2021Study to Evaluate the Long-Term Efficacy and Safety of the Investigational Drug LIB003 for the Reduction of Cholesterol in Patients with Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease.Open-Label Extension Phase 3 Study to Evaluate the Long-Term Efficacy and Safety of LIB003 in Patients With Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease, on Stable Lipid-Lowering Therapy Requiring Additional Low-Density Lipoprotein Cholesterol Reduction (LIBerate-OLE) - LIBerate-OLE Patients With Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease
MedDRA version: 20.0;Level: LLT;Classification code 10057079;Term: Heterozygous familial hypercholesterolemia;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850
MedDRA version: 20.1;Classification code 10007648;Term: Cardiovascular disease, unspecified;System Organ Class: 100000004849;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: LIB003
Product Code: lerodalcibep
INN or Proposed INN: Lerodalcibep
Other descriptive name: LIB003
LIB Therapeutics, LLCNULLNAFemale: yes
Male: yes
2000Phase 3United States;France;Canada;Spain;Turkey;South Africa;Israel;Norway;Germany;United Kingdom;New Zealand;India