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	EUCTR2019-002278-30-DE (EUCTR)	12/08/2020	11/09/2019	A clinical trial to assess the safety and effect of the drug lomitapide in the treatment of children with the condition Homozygous Familial Hypercholesterolaemia (HoFH) who are on Stable Lipid-lowering Therapy	Phase III, single-arm, open-label, international, multi-centre study to evaluate the efficacy and safety of lomitapide in paediatric patients with Homozygous Familial Hypercholesterolaemia (HoFH) on stable lipid-lowering therapy	Homozygous familial hypercholesterolaemia (HoFH). A rare and life-threatening inherited disorder of lipid metabolism with an estimated prevalence of 1 per 160,000 to 300,000 in the European population. MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]	Trade Name: Lojuxta 5 mg hard capsules INN or Proposed INN: LOMITAPIDE Trade Name: Lojuxta 10 mg hard capsules INN or Proposed INN: LOMITAPIDE Trade Name: Lojuxta 20 mg hard capsules INN or Proposed INN: LOMITAPIDE Product Name: Lomitapide 2mg hard capsules INN or Proposed INN: LOMITAPIDE	Amryt Pharmaceuticals DAC	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	45	Phase 3	Saudi Arabia;Spain;Turkey;Israel;Tunisia;Germany;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

EUCTR2019-002278-30-DE
(EUCTR)

12/08/2020

11/09/2019

A clinical trial to assess the safety and effect of the drug lomitapide in the treatment of children with the condition Homozygous Familial Hypercholesterolaemia (HoFH) who are on Stable Lipid-lowering Therapy

Phase III, single-arm, open-label, international, multi-centre study to evaluate the efficacy and safety of lomitapide in paediatric patients with Homozygous Familial Hypercholesterolaemia (HoFH) on stable lipid-lowering therapy

Homozygous familial hypercholesterolaemia (HoFH). A rare and life-threatening inherited disorder of lipid metabolism with an estimated prevalence of 1 per 160,000 to 300,000 in the European population.
MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Trade Name: Lojuxta 5 mg hard capsules
INN or Proposed INN: LOMITAPIDE
Trade Name: Lojuxta 10 mg hard capsules
INN or Proposed INN: LOMITAPIDE
Trade Name: Lojuxta 20 mg hard capsules
INN or Proposed INN: LOMITAPIDE
Product Name: Lomitapide 2mg hard capsules
INN or Proposed INN: LOMITAPIDE

Amryt Pharmaceuticals DAC

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

Saudi Arabia;Spain;Turkey;Israel;Tunisia;Germany;Italy