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 : 145 / 薬物数 : 114 - (DrugBank : 26) / 標的遺伝子数 : 8 - 標的パスウェイ数 : 17
| 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 | EUCTR2005-003449-15-GB (EUCTR) | 12/07/2007 | 09/03/2007 | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects | A 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. | NULL | Not 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): no | United Kingdom | ||
| 2 | NCT00607373 (ClinicalTrials.gov) | July 2007 | 22/1/2008 | Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Mipomersen as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects | Lipid 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 Disorders | Drug: mipomersen;Drug: Placebo | Kastle Therapeutics, LLC | Ionis Pharmaceuticals, Inc. | Completed | 12 Years | N/A | All | 51 | Phase 3 | United States;Brazil;Canada;Singapore;South Africa;Taiwan;United Kingdom;Netherlands |
| 3 | NCT00280995 (ClinicalTrials.gov) | January 2006 | 20/1/2006 | Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy | A Phase 2, Open-Label, Dose Escalation Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects | Hypercholesterolemia, Familial | Drug: ISIS 301012 | Kastle Therapeutics, LLC | Ionis Pharmaceuticals, Inc. | Completed | 18 Years | N/A | Both | 12 | Phase 2 | United States;Netherlands |