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	NCT02472535 (ClinicalTrials.gov)	April 2015	22/5/2015	Study to Evaluate the Effects of MBX-8025 in Patients With HoFH	A 12-week, Open-label, Dose-escalating, Phase 2 Study to Evaluate the Effects of MBX-8025 in Patients With Homozygous Familial Hypercholesterolemia (HoFH)	Homozygous Familial Hypercholesterolemia	Other: Run-In Period: Placebo;Drug: MBX-8025 50 mg (Dose Escalation Period 1);Drug: MBX-8025 50 mg or 100 mg (Dose Escalation Period 2);Drug: MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3)	CymaBay Therapeutics, Inc.	NULL	Completed	18 Years	N/A	Both	13	Phase 2	Canada;France;Netherlands;Norway

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

NCT02472535
(ClinicalTrials.gov)

April 2015

22/5/2015

Study to Evaluate the Effects of MBX-8025 in Patients With HoFH

A 12-week, Open-label, Dose-escalating, Phase 2 Study to Evaluate the Effects of MBX-8025 in Patients With Homozygous Familial Hypercholesterolemia (HoFH)

Homozygous Familial Hypercholesterolemia

Other: Run-In Period: Placebo;Drug: MBX-8025 50 mg (Dose Escalation Period 1);Drug: MBX-8025 50 mg or 100 mg (Dose Escalation Period 2);Drug: MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3)

CymaBay Therapeutics, Inc.

NULL

Completed

18 Years

N/A

Both

Phase 2

Canada;France;Netherlands;Norway