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

  
1 trial found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2016-001446-25-IT
(EUCTR)
02/10/201706/05/2021This is a study to investigate if gene therapy is safe in people with homozygous familial hypercholesterolemia (also called HoFH).AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) Adults with homozygous familial hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]Product Name: AAV8.TBG.hLDLR
Product Code: AAV8.TBG.hLDLR
Product Name: prednisone
Product Code: prednisone
REGENXBIO Inc.NULLNot RecruitingFemale: yes
Male: yes
12Phase 1;Phase 2United States;Canada;Netherlands;Italy