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
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04681170 (ClinicalTrials.gov) | November 23, 2020 | 7/12/2020 | Safety 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 Therapy | Homozygous Familial Hypercholesterolaemia (HoFH) | Drug: Lomitapide | Amryt Pharma | NULL | Recruiting | 5 Years | 17 Years | All | 45 | Phase 3 | Germany;Israel;Italy;Saudi Arabia;Spain;Tunisia |
| 2 | EUCTR2019-002278-30-IT (EUCTR) | 11/11/2020 | 21/01/2021 | A 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 Therapy | 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 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 DAC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 45 | Phase 3 | Saudi Arabia;Spain;Turkey;Israel;Germany;Tunisia;Italy | ||
| 3 | EUCTR2019-002278-30-DE (EUCTR) | 12/08/2020 | 11/09/2019 | A 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 Therapy | 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 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 DAC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 45 | Phase 3 | Saudi Arabia;Spain;Turkey;Israel;Tunisia;Germany;Italy | ||
| 4 | EUCTR2014-003806-33-GR (EUCTR) | 30/06/2016 | 24/03/2016 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 45 | Phase 3 | Canada;Greece;Turkey;Austria;Israel;Germany;Italy | ||
| 5 | NCT02765841 (ClinicalTrials.gov) | May 2016 | 27/4/2016 | Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy | 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 | Drug: Lomitapide | Aegerion Pharmaceuticals, Inc. | NULL | Withdrawn | 5 Years | 17 Years | All | 0 | Phase 3 | NULL |
| 6 | NCT02399852 (ClinicalTrials.gov) | June 2015 | 23/3/2015 | Effects of Lomitapide on Carotid and Aortic Atherosclerosis | Effects of Lomitapide on Carotid and Aortic Atherosclerosis in Patients Treated With Lomitapide in Usual Care (CAPTURE) | Homozygous Familial Hypercholesterolemia | Drug: Lomitapide | Aegerion Pharmaceuticals, Inc. | NULL | Withdrawn | 18 Years | N/A | All | 0 | N/A | NULL |
| 7 | NCT02173158 (ClinicalTrials.gov) | April 2, 2014 | 23/6/2014 | Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy | A 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 Therapy | Familial Hypercholesterolemia - Homozygous | Drug: lomitapide | Aegerion Pharmaceuticals, Inc. | NULL | Completed | 18 Years | N/A | All | 9 | Phase 3 | Japan |
| 8 | NCT02135705 (ClinicalTrials.gov) | March 18, 2014 | 10/4/2014 | LOWER: Lomitapide Observational Worldwide Evaluation Registry | LOWER: Lomitapide Observational Worldwide Evaluation Registry | Homozygous Familial Hypercholesterolemia | Drug: Lomitapide | Amryt Pharma | NULL | Recruiting | 18 Years | N/A | All | 300 | United States | |
| 9 | EUCTR2010-023742-79-IT (EUCTR) | 25/03/2011 | 21/02/2011 | A 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 | A 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 PHARMACEUTICALS | NULL | Not Recruiting | Female: yes Male: yes | 23 | Phase 3 | Italy | ||
| 10 | NCT00943306 (ClinicalTrials.gov) | October 29, 2009 | 21/7/2009 | Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia | A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor 'Lomitapide' (LOMITAPIDE) in Patients With Homozygous Familial Hypercholesterolemia | Familial Hypercholesterolemia | Drug: lomitapide | Aegerion Pharmaceuticals, Inc. | NULL | Completed | 18 Years | N/A | All | 19 | Phase 3 | United States;Canada;Italy;South Africa |
| 11 | NCT01556906 (ClinicalTrials.gov) | June 2003 | 7/3/2012 | Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor | A 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 Hypercholeterolemia | Homozygous Familial Hypercholesterolemia | Drug: Lomitapide | Aegerion Pharmaceuticals, Inc. | University of Pennsylvania;Doris Duke Charitable Foundation | Completed | 13 Years | N/A | All | 6 | Phase 2 | United States |