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 | EUCTR2016-003376-49-NL (EUCTR) | 20/06/2017 | 18/01/2017 | A study to evaluate the effect of ALN-PCSSC treatment in patients with Homozygous Familial Hypercholesterolemia | An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects with Homozygous Familial Hypercholesterolemia - ORION-2 | Homozygous Familial Hypercholesterolemia MedDRA version: 19.1;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: ALN-PCSSC Product Code: ALN-PCSSC INN or Proposed INN: ALN-60212 Other descriptive name: ALN-60212 | The Medicines Company | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | United States;South Africa;Netherlands | ||
| 2 | NCT02963311 (ClinicalTrials.gov) | December 13, 2016 | 10/11/2016 | A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH) | An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Drug: ALN-PCSSC;Drug: Standard of Care | The Medicines Company | NULL | Completed | 12 Years | N/A | All | 9 | Phase 2 | United States;Netherlands;South Africa |