DDrare: Database of Drug Development for Rare Diseases

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.

Drug = (4S,5R)-5-[3,5-bis(trifluoromethyl)phenyl]-3-{[4’-; 1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol); AAV directed hLDLR gene therapy; AAV8.TBG.hLDLR; AEGR-733; AK102; AKCEA-ANGPTL3-LRX; ALIROCUMAB; ALN-60212; ALN-PCSSC; ALN-PCSsc; AMG 145; AMG145; ANACETRAPIB; Acetate; Alirocumab; Alirocumab SAR236553 (REGN727); Anacetrapib; Atorvastatin; CER-001; Calcium; Calcium rosuvastatin; Cholestyramine; Comparator: ezetimibe; Crestor; Crestor Film-Coated Tablet; Crestor Tablets (calcium rosuvastatin) Film-Coated tablets; Current lipid-lowering treatment; DPPG; Device: dextran sulfate adsorption; Device: heparin-induced extracorporeal LDL precipitation; Dextran; EVINACUMAB; EVOLOCUMAB; Evinacumab; Evinacumab (REGN1500); Evolocumab; Evolocumab (AMG 145); Ezetimibe; Fenofibrate; Fluvastatin; Gemcabene; Glycerol; Heparin; IBI306; ISIS 301012; ISIS 301012 phosphorothioate oligonucleotide; ISIS 703802; Implitapide; Inclisiran; Inclisiran Sodium; Inclisiran for injection; Inclisiran sodium; JS002; KJX839; LIB003; LOMITAPIDE; Lapaquistat; Lapaquistat acetate and current lipid-lowering treatment; Lojuxta; Lojuxta 10 mg hard capsules; Lojuxta 20 mg hard capsules; Lojuxta 5 mg hard capsules; Lomitapide; Lomitapide 2mg hard capsules; Lovastatin; MBX-8025; MBX-8025 50 mg (Dose Escalation Period 1); MBX-8025 50 mg or 100 mg (Dose Escalation Period 2); MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3); MK-0859; Mipomersen; Nicotinic acid; Omega-3; Omega-3 fatty acids; PLACEBO; Phospholipids; Praluent; Pravastatin; REGN1500; REGN1500 250 mg SC/15 mg/kg IV/450 mg SC; Recombinant Human Apolipoprotein; Recombinant Human Apolipoprotein A-I; Recombinant Human Apolipoprotein A-I/Phospholipids Complex; Recombinant human apolipoprotein A-I; Recombinant human apolipoprotein a1; Repatha; Repatha (evolocumab); Repatha 140 mg Solution for Injection in Pre-filled Pen; Repatha 140 mg solution for injection in pre-filled pen; Repatha 420 mg solution for injection in cartridge; Rosuvastatin; Rosuvastatin 20mg; Rosuvastatin calcium; SAR236553; SHR-1209; Sfingomielina; Simvastatin; Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy; Sphingomyelin; Standard of Care; Statins; Sulfate; TAK-475; ZD4522

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	NCT02651675 (ClinicalTrials.gov)	March 2016	4/1/2016	A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)	AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)	Homozygous Familial Hypercholesterolemia (HoFH)	Biological: AAV directed hLDLR gene therapy	Regenxbio Inc.	National Heart, Lung, and Blood Institute (NHLBI)	Active, not recruiting	18 Years	N/A	All	12	Phase 1;Phase 2	United States;Canada;Italy;Netherlands

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

NCT02651675
(ClinicalTrials.gov)

March 2016

4/1/2016

A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)

AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)

Homozygous Familial Hypercholesterolemia (HoFH)

Biological: AAV directed hLDLR gene therapy

Regenxbio Inc.

National Heart, Lung, and Blood Institute (NHLBI)

Active, not recruiting

18 Years

N/A

All

Phase 1;Phase 2

United States;Canada;Italy;Netherlands