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	EUCTR2011-003998-28-NL (EUCTR)	28/02/2012	14/11/2011	Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemia	Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE	homozygous familial hypercholesterolemia MedDRA version: 16.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Recombinant Human Apolipoprotein A-I /Phospholipids Complex Product Code: CER-001 Other descriptive name: recombinant human apolipoprotein A-I Other descriptive name: sphingomyelin Other descriptive name: 1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol)	Cerenis Therapeutics	NULL	Not Recruiting			Female: yes Male: yes	30		France;United States;Canada;Netherlands;Italy;United Kingdom
2	EUCTR2011-003998-28-IT (EUCTR)	16/02/2012	08/03/2012	Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemia	Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE	Homozygous familial hypercholesterolemia MedDRA version: 15.1;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: Recombinant Human Apolipoprotein Product Code: CER-001 Other descriptive name: Recombinant Human Apolipoprotein A-I Other descriptive name: Sfingomielina Other descriptive name: 1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol)	CERENIS THERAPEUTICS SA	NULL	Not Recruiting			Female: yes Male: yes	30		United States;Canada;Netherlands;United Kingdom;Italy
3	EUCTR2011-003998-28-GB (EUCTR)	24/01/2012	19/10/2011	Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemia	Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE	homozygous familial hypercholesterolemia MedDRA version: 15.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 10027433 - Metabolism and nutrition disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Cerenis Therapeutics	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 2	United States;Canada;Netherlands;Italy;United Kingdom
4	NCT01412034 (ClinicalTrials.gov)	November 2011	5/8/2011	Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects	Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-label Study of the Effects of CER-001 on Plaque Volume in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)	Homozygous Familial Hypercholesterolemia	Drug: CER-001	Cerenis Therapeutics, SA	NULL	Completed	12 Years	N/A	Both	23	Phase 2	United States;Canada;Italy;Netherlands;United Kingdom

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

EUCTR2011-003998-28-NL
(EUCTR)

28/02/2012

14/11/2011

Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemia

Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE

homozygous familial hypercholesterolemia
MedDRA version: 16.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: Recombinant Human Apolipoprotein A-I /Phospholipids Complex
Product Code: CER-001
Other descriptive name: recombinant human apolipoprotein A-I
Other descriptive name: sphingomyelin
Other descriptive name: 1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol)

Cerenis Therapeutics

NULL

Not Recruiting

Female: yes
Male: yes

France;United States;Canada;Netherlands;Italy;United Kingdom

EUCTR2011-003998-28-IT
(EUCTR)

16/02/2012

08/03/2012

Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemia

Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE

Homozygous familial hypercholesterolemia
MedDRA version: 15.1;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Product Name: Recombinant Human Apolipoprotein
Product Code: CER-001
Other descriptive name: Recombinant Human Apolipoprotein A-I
Other descriptive name: Sfingomielina
Other descriptive name: 1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol)

CERENIS THERAPEUTICS SA

NULL

Not Recruiting

Female: yes
Male: yes

United States;Canada;Netherlands;United Kingdom;Italy

EUCTR2011-003998-28-GB
(EUCTR)

24/01/2012

19/10/2011

Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemia

Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE

homozygous familial hypercholesterolemia
MedDRA version: 15.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 10027433 - Metabolism and nutrition disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Cerenis Therapeutics

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United States;Canada;Netherlands;Italy;United Kingdom

NCT01412034
(ClinicalTrials.gov)

November 2011

5/8/2011

Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects

Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-label Study of the Effects of CER-001 on Plaque Volume in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)

Homozygous Familial Hypercholesterolemia

Drug: CER-001

Cerenis Therapeutics, SA

NULL

Completed

12 Years

N/A

Both

Phase 2

United States;Canada;Italy;Netherlands;United Kingdom