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 79. Homozygous familial hypercholesterolemia
 [ 54 clinical trials,    69 drugs(DrugBank: 24 drugs),    6 target genes / 14 target pathways

Searched query = "Homozygous familial hypercholesterolemia"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

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No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1NCT04034485November 7, 201911 November 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: LIB003;Drug: evolocumabLIB Therapeutics LLCRecruiting10 YearsN/AAll70Phase 3United States
2NCT04080050September 30, 201911 November 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.Recruiting18 YearsN/AAll12Phase 1/Phase 2United States;Canada;Italy;Netherlands
3NCT04031742August 12, 201929 July 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.Not recruiting18 Years80 YearsAll30Phase 2/Phase 3
4NCT03814187April 16, 20194 November 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 SodiumThe Medicines CompanyRecruiting18 YearsN/AAll3700Phase 3United States;Canada;Czechia;Denmark;Germany;Hungary;Netherlands;Poland;South Africa;Spain;Sweden;Ukraine;United Kingdom
5NCT03933293April 201913 May 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: EzetimibeAkesoNot recruiting18 YearsN/AAll59Phase 2China
No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
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Inclusion_
agemin
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Inclusion_
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PhaseCountries
6NCT03851705February 6, 20194 March 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)Renal ImpairmentDrug: Inclisiran for injection;Drug: PlacebosThe Medicines CompanyRecruiting18 Years80 YearsAll45Phase 2/Phase 3Czechia;Hong Kong;Russian Federation;Serbia;Taiwan;Ukraine
7JPRN-JapicCTI-18409930/9/201810 September 2019STUDY 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 HYPERCHOLESTEROLEMIAHomozygous 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)Not Recruiting18BOTH57NAJapan, North America, Europe, Oceania, Africa
8EUCTR2017-002297-39-NO19/09/201828 February 2019An 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 HypercholesterolemiaHomozygous 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
Pharmaceutical Form: Solution for injection in pre-filled syringe
INN or Proposed INN: Alirocumab
CAS Number: 1245916-14-6
Current Sponsor code: SAR236553 (RGN727)
Other descriptive name: ALIROCUMAB
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 75-
Pharmaceutical form of the placebo: Solution for infusion in pre-filled syringe
Route of administration of the placebo: Subcutaneous use
Product Name: Alirocumab
Product Code: SAR236553
Pharmaceutical Form: Solution for injection in pre-filled syringe
INN or Proposed INN: Alirocumab
CAS Number: 1245916-14-6
Current Sponsor code: SAR236553 (RGN727)
Other descriptive name: ALIROCUMAB
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 150-
Pharmaceutical form of the placebo: Solution for infusion in pre-filled syringe
Route of administration of the placebo: Subcutaneous use
Sanofi-Aventis Recherche & DéveloppementAuthorisedFemale: yes
Male: yes
50Phase 3United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway
9NCT03510715August 31, 201814 October 2019An 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 PharmaceuticalsNot recruiting8 Years17 YearsAll18Phase 3Brazil;Canada;Denmark;Mexico;Netherlands;Russian Federation;Slovenia;Spain;Taiwan;Turkey;Austria;France;Italy;Norway;United States
10NCT03403374August 4, 201828 October 2019Safety and Tolerability of Repatha in Indian Subjects 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: Repatha® (evolocumab)AmgenNot recruiting12 Years80 YearsAll30Phase 4India
No.TrialIDDate_
enrollement
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agemin
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PhaseCountries
11NCT03455777April 12, 201810 December 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.Not recruiting18 YearsN/AAll0Phase 2Canada
12NCT03409744March 13, 201826 August 2019Evaluate 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 PharmaceuticalsRecruiting12 YearsN/AAll100Phase 3United States;Australia;Canada;France;Greece;Italy;Netherlands;Norway;Ukraine
13NCT03156621October 3, 201717 June 2019Study 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 PharmaceuticalsSanofiRecruiting18 YearsN/AAll74Phase 3Germany;Greece;Italy;Japan;South Africa;Turkey;Ukraine;Austria;Canada;Czechia;France;United States
14EUCTR2017-001388-19-FR28 February 2019A 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 hypercholesterolemiaHomozygous 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
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Evinacumab
CAS Number: 1446419-85-7
Current Sponsor code: REGN1500
Other descriptive name: EVINACUMAB
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Powder and solvent for solution for injection
Route of administration of the placebo: Intravenous use
Trade Name: Praluent
Pharmaceutical Form: Solution for injection in pre-filled pen
INN or Proposed INN: ALIROCUMAB
CAS Number: 1245916-14-6
Other descriptive name: ALIROCUMAB
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Regeneron Pharmaceuticals Inc.Not Available Female: yes
Male: yes
57Phase 3United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands
15EUCTR2017-003170-13-FR28 February 2019Evaluate 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 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
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Evinacumab
CAS Number: 1446419-85-7
Current Sponsor code: REGN1500
Other descriptive name: EVINACUMAB
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 150-
Trade Name: Praluent
Pharmaceutical Form: Solution for injection in pre-filled pen
INN or Proposed INN: ALIROCUMAB
CAS Number: 1245916-14-6
Other descriptive name: ALIROCUMAB
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Regeneron Pharmaceuticals, Inc.Not Available Female: yes
Male: yes
100Phase 3United States;Greece;Ukraine;Turkey;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Norway;Netherlands;Germany;Japan
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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Status
Inclusion_
agemin
Inclusion_
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size
PhaseCountries
16NCT02963311December 13, 201611 June 2018A 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 CompanyNot recruiting12 YearsN/AAll4Phase 2United States;Netherlands;South Africa
17EUCTR2015-004003-23-IT28/09/201612 March 2018A 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 and Subjects with Familial HypercholesterolemiaHeterozygous 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 Level: LLT Classification code 10057080 Term: Homozygous familial hypercholesterolemia System Organ Class: 100000004850
MedDRA version: 19.0 Level: LLT 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
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ISIS 703802
Current Sponsor code: ISIS 703802
Other descriptive name: ISIS 703802
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use
Ionis Pharmaceuticals, Inc.AuthorisedFemale: yes
Male: yes
61Phase 1;Phase 2Canada;Netherlands;Italy
18NCT02624869September 10, 201622 October 2019Open 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)AmgenRecruiting10 Years17 YearsAll115Phase 3United States;Australia;Austria;Belgium;Brazil;Canada;Colombia;Czechia;Greece;Hungary;Italy;Malaysia;Netherlands;Norway;Poland;Portugal;Slovenia;South Africa;Spain;Switzerland;Turkey;United Kingdom
19EUCTR2016-000411-32-NL15/07/20161 October 2018A 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 HYPERCHOLESTEROLEMIAHomozygous 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
Pharmaceutical Form: Lyophilisate for solution for infusion
INN or Proposed INN: EVINACUMAB
CAS Number: 1446419-85-7
Current Sponsor code: REGN1500
Other descriptive name: EVINACUMAB
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Product Name: Evinacumab
Product Code: REGN1500
Pharmaceutical Form: Lyophilisate for solution for injection
INN or Proposed INN: EVINACUMAB
CAS Number: 1446419-85-7
Current Sponsor code: REGN1500
Other descriptive name: EVINACUMAB
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Product Name: evinacumab
Product Code: REGN1500
Pharmaceutical Form: Lyophilisate for solution for infusion
INN or Proposed INN: EVINACUMAB
CAS Number: 1446419-85-7
Current Sponsor code: REGN1500
Other descriptive name: EVINACUMAB
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
Regeneron Pharmaceuticals, Inc.Not RecruitingFemale: yes
Male: yes
8Phase 2United States;Canada;Netherlands
20EUCTR2014-003806-33-GR30/06/20168 August 2016A clinical study in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy conducted to assess safety, tolerability and efficacy of LomitapideA 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 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
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: LOMITAPIDE
CAS Number: 202914-84-9
Current Sponsor code: AEGR-733
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2-
Product Name: lomitapide
Product Code: AEGR-733
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: LOMITAPIDE
CAS Number: 202914-84-9
Current Sponsor code: AEGR-733
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Product Name: lomitapide
Product Code: AEGR-733
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: LOMITAPIDE
CAS Number: 202914-84-9
Current Sponsor code: AEGR 733
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Product Name: lomitapide
Product Code: AEGR-733
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: LOMITAPIDE
CAS Number: 202914-84-9
Current Sponsor code: AEGR-733
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Aegerion Pharmaceuticals Inc.Not RecruitingFemale: yes
Male: yes
45Phase 3Canada;Greece;Turkey;Austria;Israel;Germany;Italy
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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size
PhaseCountries
21EUCTR2015-002276-25-AT27/06/201628 February 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-OLEHypercholesterolemia
MedDRA version: 20.0 Level: LLT Classification code 10057100 Term: Homozygous familial hypercholesterolaemia System Organ Class: 100000004850
MedDRA version: 20.0 Level: LLT 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
Pharmaceutical Form: Solution for injection in pre-filled pen
INN or Proposed INN: EVOLOCUMAB
Current Sponsor code: AMG 145
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 140-
Pharmaceutical form of the placebo: Solution for injection in pre-filled pen
Route of administration of the placebo: Subcutaneous use
Trade Name: Repatha 420 mg solution for injection in cartridge
Product Name: Evolocumab
Product Code: AMG 145
Pharmaceutical Form: Solution for injection in cartridge
INN or Proposed INN: EVOLOCUMAB
Current Sponsor code: AMG 145
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 120-
Amgen IncAuthorised Female: yes
Male: yes
115Phase 3Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;United Kingdom;Hungary;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;South Africa;Netherlands;Germany;New Zealand
22JPRN-UMIN0000226032016/06/062 April 2019Non-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 HospitalNot Recruiting20years-old100years-oldMale and Female2Not selectedJapan
23NCT02722408May 201616 December 2017Efficacy 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: Gemcabene 300 mg;Drug: Gemcabene 600 mg;Drug: Gemcabene 900 mgGemphire Therapeutics, Inc.Not recruiting17 YearsN/AAll8Phase 2United States;Canada;Israel
24NCT02765841May 20165 March 2018Evaluate 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.Not recruiting5 Years17 YearsAll0Phase 3
25NCT02651675March 201630 September 2019A 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)Recruiting18 YearsN/AAll12Phase 1/Phase 2United States;Canada;Italy;Netherlands
No.TrialIDDate_
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Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
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Inclusion_
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Inclusion_
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Inclusion_
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size
PhaseCountries
26NCT02434497June 6, 201512 March 2018A 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 20mgAstraZenecaNot recruiting6 Years18 YearsAll9Phase 3Belgium;Canada;Denmark;Israel;Malaysia;Taiwan
27NCT02399852June 20158 January 2018Effects 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.Not recruiting18 YearsN/AAll0N/A
28EUCTR2014-004856-68-NO29/04/20153 October 2016A 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
Pharmaceutical Form: Capsule
INN or Proposed INN: MBX-8025
CAS Number: 928821-40-3
Current Sponsor code: MBX-8025
Concentration unit: mg milligram(s)
Concentration type: range
Concentration number: 50-100
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use
CymaBay Therapeutics, Inc.Not RecruitingFemale: yes
Male: yes
8Phase 2France;Netherlands;Norway
29NCT02472535April 201511 April 2016Study 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.Not recruiting18 YearsN/ABoth13Phase 2Canada;France;Netherlands;Norway
30EUCTR2014-004746-99-BE23/02/20159 January 2017A 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
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Rosuvastatin calcium
CAS Number: 147098-20-2
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
AstraZeneca ABNot RecruitingFemale: yes
Male: yes
20Phase 3United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Netherlands;Germany;Sweden
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT02265952February 4, 201520 August 2018Study of REGN1500 in Patients 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: REGN1500Regeneron PharmaceuticalsNot recruiting18 YearsN/AAll9Phase 2United States;Canada;Netherlands
32NCT02226198November 201419 October 2017A 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: PlaceboAstraZenecaNot recruiting6 Years17 YearsAll20Phase 3Belgium;Canada;Denmark;Israel;Malaysia;Netherlands;Taiwan;Germany;Lebanon;Sweden;United States
33EUCTR2014-000972-24-SE20/08/201414 September 2015A 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) - HYDRAHypercholesterolemia - 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
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: calcium rosuvastatin
CAS Number: 147098-20-2
Current Sponsor code: ZD4522
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
AstraZeneca ABNot RecruitingFemale: yes
Male: yes
25United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden
34NCT02173158April 201429 June 2015Efficacy 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.Not recruiting18 YearsN/ABoth10Phase 3Japan
35NCT02135705March 20148 January 2018LOWER: Lomitapide Observational Worldwide Evaluation RegistryLOWER: Lomitapide Observational Worldwide Evaluation RegistryHomozygous Familial HypercholesterolemiaDrug: LomitapideAegerion Pharmaceuticals, Inc.Recruiting18 YearsN/AAll300N/AUnited States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36EUCTR2012-002434-37-GB23/05/201328 February 2019n/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 HypercholesterolemiaHomozygous 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’-
Pharmaceutical Form: Tablet
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., IncNot Recruiting Female: yes
Male: yes
45Phase 3United States;Czech Republic;Canada;Malaysia;Australia;Israel;South Africa;Norway;Italy;United Kingdom
37EUCTR2011-005400-15-GB22/05/201328 February 2019A 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 LDLFamilial 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
Pharmaceutical Form: Solution for injection
Current Sponsor code: AMG 145
Other descriptive name: AMG 145
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 70-
Product Name: AMG 145
Pharmaceutical Form: Solution for injection in pre-filled pen
Current Sponsor code: AMG 145
Other descriptive name: AMG145
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 140-
Product Name: AMG 145
Pharmaceutical Form: Solution for injection in cartridge
Current Sponsor code: AMG 145
Other descriptive name: AMG145
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 120-
Amgen IncNot Recruiting Female: 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
38NCT02286596April 201311 April 2016Comparisons 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 UniversityNot recruiting18 Years60 YearsBoth9N/ACanada
39EUCTR2011-005400-15-BE22/03/201228 May 2018A 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 LDLFamilial 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
Pharmaceutical Form: Solution for injection
Current Sponsor code: AMG 145
Other descriptive name: AMG 145
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 70-
Product Name: AMG 145
Pharmaceutical Form: Solution for injection in pre-filled pen
Current Sponsor code: AMG 145
Other descriptive name: AMG145
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 140-
Product Name: AMG 145
Pharmaceutical Form: Solution for injection in cartridge
Current Sponsor code: AMG 145
Other descriptive name: AMG145
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 120-
Amgen IncNot 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
40EUCTR2011-005399-40-BE09/03/201221 August 2017A 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 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 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
Pharmaceutical Form: Solution for injection in pre-filled pen
Current Sponsor code: AMG 145
Other descriptive name: AMG 145
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 140-
Pharmaceutical form of the placebo: Solution for injection in pre-filled pen
Route of administration of the placebo: Subcutaneous use
Amgen IncNot 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
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41NCT01412034November 201119 February 2015Effect 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, SANot recruiting12 YearsN/ABoth23Phase 2United States;Canada;Italy;Netherlands;United Kingdom
42EUCTR2010-023742-79-IT25/03/201112 October 2015A 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 - NDHomozygous Familial Hypercholesterolemia
MedDRA version: 9.1 Level: LLT Classification code 10057080
Product Name: Lomitapide
Product Code: AEGR-733
Pharmaceutical Form: Capsule, hard
Current Sponsor code: AEGR-733
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Product Name: Lomitapide
Product Code: AEGR-733
Pharmaceutical Form: Capsule, hard
Current Sponsor code: AEGR-733
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
AEGERION PHARMACEUTICALSNot RecruitingFemale: yes
Male: yes
23Phase 3Italy
43NCT00943306October 29, 200911 June 2018Long 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.Not recruiting18 YearsN/AAll19Phase 3United States;Canada;Italy;South Africa
44EUCTR2008-007058-36-IT25/06/200919 March 2012A 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 - NDHomozygous Familial Hypercholesterolemia
MedDRA version: 9.1 Level: LLT Classification code 10048373 Term: Hypercholesterolaemia aggravated
Product Code: AEGR-733
Pharmaceutical Form: Capsule, hard
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Product Code: AEGR-733
Pharmaceutical Form: Capsule, hard
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Aegerion PharmaceuticalsNot RecruitingFemale: yes
Male: yes
25Phase 3Italy
45NCT00730236December 200719 October 2017A 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 DevelopmentNot recruiting18 YearsN/AAll29Phase 3United States;Canada;Italy;South Africa
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
46EUCTR2005-003449-15-GB12/07/200710 July 2015A 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 IReduction 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
Pharmaceutical Form: Solution for injection
Current Sponsor code: ISIS 301012
Other descriptive name: ISIS 301012 phosphorothioate oligonucleotide
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use
Genzyme Europe B.V.AuthorisedFemale: yes
Male: yes
50United Kingdom
47EUCTR2005-003626-26-GB14/06/200618 April 2012A 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 hypercholesterolemiaTreatment of primary dyslipidemia
MedDRA version: 8 Level: PT Classification code 10058108
Product Name: TAK-475
Product Code: TAK-475
Pharmaceutical Form: Tablet
Current Sponsor code: TAK-475
Concentration unit: % percent
Concentration type: range
Concentration number: 95-105
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Takeda Europe R&D Centre LtdNot RecruitingFemale: yes
Male: yes
40United Kingdom
48NCT00704535March 200619 October 2017Evaluation 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: EzetimibeMerck Sharp & Dohme Corp.Not recruiting18 YearsN/AAll4105N/APhilippines
49NCT00280995January 200615 August 2016Dose-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.Not recruiting18 YearsN/ABoth12Phase 2United States;Netherlands
50NCT00079846September 200319 February 2015Implitapide 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 InternationalNot recruiting8 Years70 YearsBoth60Phase 2United States;Canada;Israel;Netherlands;Norway
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
51NCT01556906June 200319 October 2017Safety, 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 FoundationNot recruiting13 YearsN/AAll6Phase 2United States
52NCT00092833July 200216 December 2017Investigational 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: ezetimibeMerck Sharp & Dohme Corp.Not recruiting8 YearsN/AAll49Phase 3United States
53NCT03885921October 25, 20001 April 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: SimvastatinMerck Sharp & Dohme Corp.Not recruiting12 YearsN/AAll44Phase 3
54NCT03884452May 3, 200024 June 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 EzetimibeMerck Sharp & Dohme Corp.Not recruiting12 YearsN/AAll50Phase 3

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