UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Non-randomized, prospective, non-controlled study of changes of lipid parameters after introduction of evolocumab in patients with heterozygous familial hypercholesterolemia under LDL-apheresis therapy.
Title of the study (Brief title)	Impact of evelocumab in homozygous familial hypercholesterolemia under LDL-apheresis.
Region	Japan

Condition
Condition	Homozygous familial hypercholesterolemia
Classification by specialty	Cardiology Endocrinology and Metabolism
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To investigate the safety and the changes of lipid parameters after the introduction of evolocumab in the patients with homozygous familial hypercholesterolemia under LDL-apheresis.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Comparison of the time constant average levels of LDL-C and apoB before and after evolocumab thereapy.
Key secondary outcomes

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Subcutaneous injection of evolocumab
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	20 years-old <
Age-upper limit	100 years-old >
Gender	Male and Female
Key inclusion criteria	Homozygous familial hypercholesterolemic patients under LDL-apheresis therapy and stable lipid-lowering therapy including statins, ezetimibe, fibrates, resin, probucol, EPA, niacin, and vitamin E
Key exclusion criteria	Myocardial infarction or unstable angina within 8 weeks Severe anemia (Hb <10g/dL) Allergy against evolocumab Immunosupressive therapy Severe hepatic disorder (ASTorALT>100IU/L) Severe renal disorder (BUN>40mg/dL or Cre>2.0mg/dL) (Possible of) pregnancy
Target sample size	2

Research contact person
Name of lead principal investigator	Masa-aki Kawashiri
Organization	Kanazawa University Hospital
Division name	Department of Cardiology
Address	13-1 Takara-machi, Kanazawa
TEL	076-265-2251
Email	mk@med.kanazawa-u.ac.jp

Public contact
Name of contact person	Masa-aki Kawashiri
Organization	Kanazawa University Hospital
Division name	Department of Cardiology
Address	13-1 Takara-machi, Kanazawa
TEL	076-265-2251
Homepage URL
Email	mk@med.kanazawa-u.ac.jp

Sponsor
Institute	Kanazawa University Hospital
Institute
Department

Funding Source
Organization	Kanazawa University Hospital
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2016 Year 06 Month 04 Day

Progress
Recruitment status	Terminated
Date of protocol fixation	2016 Year 05 Month 25 Day
Anticipated trial start date	2016 Year 06 Month 06 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2016 Year 06 Month 04 Day
Last modified on	2017 Year 06 Month 08 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000026056