DDrare: Database of Drug Development for Rare Diseases

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_ gender	Target_ size	Phase	Countries
1	EUCTR2014-000972-24-DK (EUCTR)	17/12/2014	25/09/2014	A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia	A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA	Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.0;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin	AstraZeneca AB	NULL	Not Recruiting	Female: yes Male: yes	25		United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Germany;Netherlands;Sweden
2	EUCTR2014-000972-24-DE (EUCTR)	17/11/2014	26/09/2014	A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia	A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA	Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin	AstraZeneca AB	NULL	Not Recruiting	Female: yes Male: yes	25		United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Denmark;Australia;South Africa;Germany;Netherlands;Norway;Sweden
3	EUCTR2014-000972-24-BE (EUCTR)	27/10/2014	11/09/2014	A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia	A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA	Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin	AstraZeneca AB	NULL	Not Recruiting	Female: yes Male: yes	25	Phase 3	United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden
4	EUCTR2014-000972-24-NL (EUCTR)	20/10/2014	05/08/2014	A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia	A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA	Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin	AstraZeneca AB	NULL	Not Recruiting	Female: yes Male: yes	25		United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden
5	EUCTR2014-000972-24-SE (EUCTR)	20/08/2014	30/06/2014	A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia	A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA	Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin	AstraZeneca AB	NULL	Not Recruiting	Female: yes Male: yes	25		United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden

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

EUCTR2014-000972-24-DK
(EUCTR)

17/12/2014

25/09/2014

A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia

A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA

Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH)
MedDRA version: 17.0;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin

AstraZeneca AB

NULL

Not Recruiting

Female: yes
Male: yes

United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Germany;Netherlands;Sweden

EUCTR2014-000972-24-DE
(EUCTR)

17/11/2014

26/09/2014

A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia

Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH)
MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin
Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin

AstraZeneca AB

NULL

Not Recruiting

Female: yes
Male: yes

United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Denmark;Australia;South Africa;Germany;Netherlands;Norway;Sweden

EUCTR2014-000972-24-BE
(EUCTR)

27/10/2014

11/09/2014

A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia

Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin

AstraZeneca AB

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden

EUCTR2014-000972-24-NL
(EUCTR)

20/10/2014

05/08/2014

A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia

Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin

AstraZeneca AB

NULL

Not Recruiting

Female: yes
Male: yes

United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden

EUCTR2014-000972-24-SE
(EUCTR)

20/08/2014

30/06/2014

A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia

Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets
Product Name: Crestor
Product Code: ZD4522
INN or Proposed INN: calcium rosuvastatin

AstraZeneca AB

NULL

Not Recruiting

Female: yes
Male: yes

United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden