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_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2014-003806-33-GR (EUCTR)	30/06/2016	24/03/2016	A clinical study in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy conducted to assess safety, tolerability and efficacy of Lomitapide	A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy	Homozygous familial hypercholesterolemia (HoFH). MedDRA version: 18.1;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: lomitapide Product Code: AEGR-733 INN or Proposed INN: LOMITAPIDE Product Name: lomitapide Product Code: AEGR-733 INN or Proposed INN: LOMITAPIDE Product Name: lomitapide Product Code: AEGR-733 INN or Proposed INN: LOMITAPIDE Product Name: lomitapide Product Code: AEGR-733 INN or Proposed INN: LOMITAPIDE	Aegerion Pharmaceuticals Inc.	NULL	Not Recruiting			Female: yes Male: yes	45	Phase 3	Canada;Greece;Turkey;Austria;Israel;Germany;Italy
2	EUCTR2010-023742-79-IT (EUCTR)	25/03/2011	21/02/2011	A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - ND	A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - ND	Homozygous Familial Hypercholesterolemia MedDRA version: 9.1;Level: LLT;Classification code 10057080	Product Name: Lomitapide Product Code: AEGR-733 Product Name: Lomitapide Product Code: AEGR-733	AEGERION PHARMACEUTICALS	NULL	Not Recruiting			Female: yes Male: yes	23	Phase 3	Italy
3	EUCTR2008-007058-36-IT (EUCTR)	25/06/2009	03/06/2009	A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients with Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy - ND	A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients with Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy - ND	Homozygous Familial Hypercholesterolemia MedDRA version: 9.1;Level: LLT;Classification code 10048373;Term: Hypercholesterolaemia aggravated	Product Code: AEGR-733 Product Code: AEGR-733	Aegerion Pharmaceuticals	NULL	Not Recruiting			Female: yes Male: yes	25	Phase 3	Italy
4	NCT00730236 (ClinicalTrials.gov)	December 2007	6/8/2008	A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)	A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients With Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy	Homozygous Familial Hypercholesterolemia	Drug: AEGR-733	Aegerion Pharmaceuticals, Inc.	FDA Office of Orphan Products Development	Completed	18 Years	N/A	All	29	Phase 3	United States;Canada;Italy;South Africa

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

EUCTR2014-003806-33-GR
(EUCTR)

30/06/2016

24/03/2016

A clinical study in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy conducted to assess safety, tolerability and efficacy of Lomitapide

A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy

Homozygous familial hypercholesterolemia (HoFH).
MedDRA version: 18.1;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: lomitapide
Product Code: AEGR-733
INN or Proposed INN: LOMITAPIDE
Product Name: lomitapide
Product Code: AEGR-733
INN or Proposed INN: LOMITAPIDE
Product Name: lomitapide
Product Code: AEGR-733
INN or Proposed INN: LOMITAPIDE
Product Name: lomitapide
Product Code: AEGR-733
INN or Proposed INN: LOMITAPIDE

Aegerion Pharmaceuticals Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Canada;Greece;Turkey;Austria;Israel;Germany;Italy

EUCTR2010-023742-79-IT
(EUCTR)

25/03/2011

21/02/2011

A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - ND

Homozygous Familial Hypercholesterolemia
MedDRA version: 9.1;Level: LLT;Classification code 10057080

Product Name: Lomitapide
Product Code: AEGR-733
Product Name: Lomitapide
Product Code: AEGR-733

AEGERION PHARMACEUTICALS

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Italy

EUCTR2008-007058-36-IT
(EUCTR)

25/06/2009

03/06/2009

A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients with Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy - ND

Homozygous Familial Hypercholesterolemia
MedDRA version: 9.1;Level: LLT;Classification code 10048373;Term: Hypercholesterolaemia aggravated

Product Code: AEGR-733
Product Code: AEGR-733

Aegerion Pharmaceuticals

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Italy

NCT00730236
(ClinicalTrials.gov)

December 2007

6/8/2008

A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)

A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients With Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy

Homozygous Familial Hypercholesterolemia

Drug: AEGR-733

Aegerion Pharmaceuticals, Inc.

FDA Office of Orphan Products Development

Completed

18 Years

N/A

All

Phase 3

United States;Canada;Italy;South Africa