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 | NCT03455777 (ClinicalTrials.gov) | April 12, 2018 | 22/2/2018 | Study 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Homozygous Familial Hypercholesterolemia | Drug: AKCEA-ANGPTL3-LRX | Akcea Therapeutics | Ionis Pharmaceuticals, Inc. | Withdrawn | 18 Years | N/A | All | 0 | Phase 2 | Canada |
| 2 | EUCTR2015-004003-23-IT (EUCTR) | 28/09/2016 | 27/05/2016 | A 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 Hypercholesterolemia | A 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 andSubjects with Familial Hypercholesterolemia | Heterozygous 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;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;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 INN or Proposed INN: ISIS 703802 Other descriptive name: ISIS 703802 | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 61 | Phase 1;Phase 2 | Canada;Netherlands;Italy |