79. 家族性高コレステロール血症（ホモ接合体）
［臨床試験数：128，薬物数：105（DrugBank：29），標的遺伝子数：8，標的パスウェイ数：15］
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.
| 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 | 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 | ||
| 2 | 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 | ||
| 3 | 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 |
| 4 | 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 |
| 5 | 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 |
| 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 |
| 6 | 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 | |
| 7 | 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 | ||
| 8 | 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 |
| 9 | 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 |