DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
79	家族性高コレステロール血症（ホモ接合体）	1

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-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

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-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