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UMIN ID:

Recruitment status Open public recruiting
Unique ID issued by UMIN UMIN000023810
Receipt No. R000027343
Scientific Title Clinical research for auto-transplantation of lcat-gene transduced preadipocytes in patients with familial lecithin-cholesterol acyltransferase (LCAT) deficiency
Date of disclosure of the study information 2016/09/01
Last modified on 2019/04/15

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Basic information
Public title Clinical research for auto-transplantation of lcat-gene transduced preadipocytes in patients with familial lecithin-cholesterol acyltransferase (LCAT) deficiency
Acronym Clinical research for auto-transplantation of lcat-gene transduced preadipocytes in patients with familial lecithin-cholesterol acyltransferase (LCAT) deficiency
Scientific Title Clinical research for auto-transplantation of lcat-gene transduced preadipocytes in patients with familial lecithin-cholesterol acyltransferase (LCAT) deficiency
Scientific Title:Acronym Clinical research for auto-transplantation of lcat-gene transduced preadipocytes in patients with familial lecithin-cholesterol acyltransferase (LCAT) deficiency
Region
Japan

Condition
Condition Familial LCAT deficiency
Classification by specialty
Cardiology Endocrinology and Metabolism Nephrology
Ophthalmology
Classification by malignancy Others
Genomic information YES

Objectives
Narrative objectives1 Purpose of this study is to evaluate safety of LCAT replacement by auto-transplantation of lcat-gene transduced preadipocytes.
Basic objectives2 Safety
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes Purpose of this study is to evaluate adverse event(s) of LCAT replacement by auto-transplantation of lcat-gene transduced preadipocytes.
Key secondary outcomes Effects of LCAT replacement on main symptoms of the LCAT deficiency (Plasma (or serum) LCAT activity, HDL-cholesterol, cholesteryl ester/total cholesterol ratio, renal manifestation, visual impairment, corneal opacity, and anemia) are exploratively evaluated for 24weeks after transplantation.

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
Gene Other
Interventions/Control_1 Adipose tissue is extirpated from patient with familial LCAT deficiency syndrome, and subjected to primary culture by ceiling culture. Normal LCAT gene is transduced into the obtained preadipocytes by retroviral vector-mediated gene transduction. Propagated LCAT-secreting preadipocytes are then subcutaneously transplanted into the patient. The number of transplanted cells are 500,000,000 to 1,000,000,000.
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
16 years-old <=
Age-upper limit

Not applicable
Gender Male and Female
Key inclusion criteria Patients who are determined to be suffering from familial LCAT deficiency based on the following diagnosis criteria:
Patient with LCAT genetic abnormality, as determined by a genetic diagnosis (test) (Excluding, however, patients in which LCAT Full length proteins are not expressed due to occurrence of termination codon or frame shift mutation)
Patients present low HDL-cholesterol levels with one or some of following manifestation: Corneal opacity, Renal dysfunction (proteinuria), Hemolytic anemia
Plasma (or serum) LCAT activity (as determined by a Sekisui Medical Anasolv LCAT kit) is lower than the standard minimum limit (Standard value is 235-550 U.)
Patients whose poor quality of life and prognosis are predicted due to clinical symptoms (especially corneal opacity and renal dysfunction)
Patients older than 16.
Patient who provides written informed consent. If the patient is a minor, written consent must be obtained from the patient as well as a parent or guardian.
Key exclusion criteria 1) Patients that show no LCAT full length protein variation and/or patients in which LCAT proteins are not detected in the blood.
2) Patients with concomitant acute liver disease as a result of lipid metabolism (Acute hepatitis, cirrhosis of the liver) or kidney disease.
3) Mal- or undernourished, or suffering from a nutritional disorder such as Cachexia.
4) Will undergo a blood and/or plasma transfusion within one month prior receiving LCAT replacement therapy.
5) Patients testing positive for severe viral infection (Hepatitis B, Hepatitis C, HIV, Adult T-cell leukemia, Parvovirus B19)
6) Liposuction surgery deemed too challenging to undergo.
7) Women who are pregnant, nursing, or could become pregnant
8) Patients suffering from an illness that leads to low blood cholesterol other than LCAT deficiency (ApoA-1 hypercholesterolemia, Tangier disease)
9) Patients determined ineligible by the Physician in charge and/or the IRB for any reason.
Target sample size 3

Research contact person
Name of lead principal investigator
1st name Koutaro
Middle name
Last name Yokote
Organization Chiba university
Division name Chiba University hospital, Division of Diabetes, Metabolism and Endocrinology
Zip code 260-8670
Address 1-8-1 Inohana, Chuo-ku, Chiba
TEL 043-222-7171
Email kyokote@faculty.chiba-u.jp

Public contact
Name of contact person
1st name Daisuke
Middle name
Last name Kinoshita
Organization Chiba university
Division name Chiba University hospital, Division of Diabetes, Metabolism and Endocrinology
Zip code 260-8670
Address 1-8-1 Inohana, Chuo-ku, Chiba
TEL 043-222-7171
Homepage URL
Email d.kinoopyg.0722@gmail.com

Sponsor
Institute Chiba university
Chiba University hospital, Division of Diabetes, Metabolism and Endocrinology
Institute
Department

Funding Source
Organization Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization Government offices of other countries
Nationality of Funding Organization Japan

Other related organizations
Co-sponsor CellGenTech, Inc.
Name of secondary funder(s)

IRB Contact (For public release)
Organization The Second Certified Special Committee for Regenerative Medicine, Osaka University
Address 4F Center of Medical Innovation and Translational Research 2-2 Yamadaoka, Suita, Osaka
Tel 06-6210-8293
Email nintei@dmi.med.osaka-u.ac.jp

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 千葉大学医学部附属病院(千葉県)

Other administrative information
Date of disclosure of the study information
2016 Year 09 Month 01 Day

Related information
URL releasing protocol
Publication of results Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status Open public recruiting
Date of protocol fixation
2016 Year 08 Month 08 Day
Date of IRB
Anticipated trial start date
2016 Year 09 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2016 Year 08 Month 29 Day
Last modified on
2019 Year 04 Month 15 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000027343

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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