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	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	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	61	Phase 1;Phase 2	Canada;Netherlands;Italy

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

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

Phase 2

Canada

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

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1;Phase 2

Canada;Netherlands;Italy