79. Homozygous familial hypercholesterolemia
128 clinical trials,   105 drugs   (DrugBank: 29 drugs),   8 drug target genes,   15 drug target pathways

Searched query = "Homozygous familial hypercholesterolemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
9 trials found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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agemax
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PhaseCountries
1EUCTR2019-002278-30-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
2EUCTR2014-003806-33-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
3NCT02765841
(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
4NCT02399852
(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
5NCT02173158
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT02135705
(ClinicalTrials.gov)
March 18, 201410/4/2014LOWER: Lomitapide Observational Worldwide Evaluation RegistryLOWER: Lomitapide Observational Worldwide Evaluation RegistryHomozygous Familial HypercholesterolemiaDrug: LomitapideAmryt PharmaNULLRecruiting18 YearsN/AAll300United States
7EUCTR2010-023742-79-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
8NCT00943306
(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
9NCT01556906
(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