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Recruitment status Terminated
Unique ID issued by UMIN UMIN000020591
Receipt No. R000023759
Scientific Title A phase I clinical trial evaluating the safety of allogeneic Adipose Tissue-derived multilineage progenitor cells-transplantation therapy in severe familial hypercholesterolemia patients
Date of disclosure of the study information 2016/01/16
Last modified on 2019/01/24

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Basic information
Public title A phase I clinical trial evaluating the safety of allogeneic Adipose Tissue-derived multilineage progenitor cells-transplantation therapy in severe familial hypercholesterolemia patients
Acronym A phase I clinical trial evaluating the safety of allogeneic Adipose Tissue-derived multilineage progenitor cells-transplantation therapy in severe familial hypercholesterolemia patients
Scientific Title A phase I clinical trial evaluating the safety of allogeneic Adipose Tissue-derived multilineage progenitor cells-transplantation therapy in severe familial hypercholesterolemia patients
Scientific Title:Acronym A phase I clinical trial evaluating the safety of allogeneic Adipose Tissue-derived multilineage progenitor cells-transplantation therapy in severe familial hypercholesterolemia patients
Region
Japan

Condition
Condition Severe familial hypercholesterolemia (mainly homozygotes) undergoing LDL-apheresis
Classification by specialty
Cardiology Endocrinology and Metabolism
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 The aim of this study is to evaluate the safety of allogeneic Adipose tissue-derived multilineage progenitor cells-transplantation therapy in severe familial hypercholesterolemia patients undergoing LDL-apheresis.
Basic objectives2 Safety
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase Phase I

Assessment
Primary outcomes Safety evaluation
1) Systemic adverse events
(1) Allergic reaction, rejection reaction and thrombosis by cell-transplantation

Fever, eosinophilia, and abnormality of PT-INR, D-Dimer, AST, ALT
(2) Complications in the blood vessel by cell-transplantation
Bleeding, presence and severity of embolism

2) Adverse events of the puncture site
Hematoma, presence and disease severity of the infectious disease

3) Other adverse events
Presence, name, type, disease severity, frequency and period of adverse events are evaluated.

4) Other safety evaluations
Vital signs (blood pressure, heart rate, body temperature), electrocardiograms, blood test and urinalysis are evaluated.
Key secondary outcomes Secondary end-point about the safety
1) Safety evaluation about the immunosuppressive drug use
Correlation of dose immunosuppressive drug and renal function, blood sugar level, and affection of the infectious disease are evaluated
2) Evaluation of transplanted ADMPC in the systemic circulation
The STR-PCR is examined in the systemic circulation before and after transplantation.

Secondary end-point about the efficacy
1) Decrease in LDL-C, changes in serum lipid (T-cho, HDL-C, TG, Lp(a))

(1) The rate of change in LDL-C (actual value), LDL-C(Friedewald formula) among 8-11weeks, 13-16 weeks and 17-20 weeks after transplantation

(2) The change over time in LDL-C (actual value), LDL-C(Friedewald formula), T-cho, HDL-C, TG and Lp(a)

(3) The association with reascension of LDL-C (actual value) and LDL-C (Friedewald formula) after LDL-apheresis and the transplanted cell counts is examined.

(4) The association with the change of LDL-C (actual value) and LDL-C (Friedewald formula) in 8-11 weeks, 13-16 weeks and 17-20 weeks and the transplanted cell counts is examined.

2) Secession possibility of LDL-apheresis
3) The association of the background of donor (age, sex and weight) and the quantity of collected adipose tissue is evaluated.

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
Maneuver
Interventions/Control_1 Allogeneic adipose tissue-derived multi-lineage progenitor cells are isolated from donors and cultured. Total 1.6x10^5/kg-1x10^7/kg cells in the heparin-added lactated Ringer aree transplanted into liver via portal vein.
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
70 years-old >
Gender Male and Female
Key inclusion criteria The patient who meets the following criteria is enrolled.

1) The patient diagnosed with familial hypercholesterolemia (based on the diagnostic criteria of the Japanese arteriosclerosis society)
2) The patient who undergoes LDL-apheresis
3) The patient who is older than or equal to 20 years old and younger than 80 years when obtained informed consents are obtained
4) The patient who can understand the contents of the study, have the ability for agreement and write a written agreement

The patient whose relative (blood relationships in the sixth degree), spouse or relative (by marriage in the third degree) can meet the eligibility criteria of the cell donor and agree to become a cell donor with a written agreement.
Key exclusion criteria The patients corresponding to any of the following criteria are excluded from the study.
1) The patients who have or are suspected to have a malignant tumor or a precancerous lesion; or have the past history of those within five years
2) The patients who have a coronary disease or cerebrovascular disorder, which require any treatment.
3) The patients who have renal dysfunction, liver dysfunction and blood disorders.
4) The patient who have diabetes mellitus or have more than 6.5% HbA1c.
5) The patient who have an active infectious disease.
6) The patient who have any of infectious disease such as HBV (carrier and the existing infected patient), HCV, HTLV-I (ATLA), HIV, parvoviral B19, Treponema pallidum, gonococcus, tuberculosis, cytomegalovirus, EB virus, one of waist Nile viruses.
7) The patient who requires to have any treatment (surgical care) affecting the liver function test during the study
8) The patient Person with the communicable spongy encephalopathic and doubt and dementia
9) The pregnant patient, the possible pregnant patient or the patient who wishes to have pregnancy during a study
10) The patient who gives breast-feeding
11) The patient who has the past of an alcoholism or the drug dependency within six months before registration
12) The patient who are diagnosed by the doctor operating this study not to be able to recognize the significance of the clinical study from a mental disease.
13) Others, the patient who are diagnosed not to be eligible to be enrolled into this study by the doctor.
Target sample size 4

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Shizuya Yamashita
Organization Osaka University Graduate School of Medicine
Division name Community Medicine
Zip code
Address 2-2 Yamadaoka, Suita, Osaka, Japan
TEL 06-6879-3633
Email shizu@cardiology.med.osaka-u.ac.jp

Public contact
Name of contact person
1st name
Middle name
Last name Masahiro Koseki
Organization Osaka University Graduate School of Medicine
Division name Cardiology
Zip code
Address 2-2 Yamadaoka, Suita, Osaka, Japan
TEL 06-6879-3633
Homepage URL
Email koseki@cardiology.med.osaka-u.ac.jp

Sponsor
Institute Osaka University Graduate School of Medicine
Institute
Department

Funding Source
Organization Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor Osaka University Hospital
Medical Center for Translational Research
Name of secondary funder(s)

IRB Contact (For public release)
Organization
Address
Tel
Email

Secondary IDs
Secondary IDs YES
Study ID_1 PA8150003
Org. issuing International ID_1 Ministry of Health, Labour, Sports and Welfare
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions 大阪大学医学部附属病院(大阪府)

Other administrative information
Date of disclosure of the study information
2016 Year 01 Month 16 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 Terminated
Date of protocol fixation
2015 Year 12 Month 25 Day
Date of IRB
Anticipated trial start date
2016 Year 01 Month 15 Day
Last follow-up date
2018 Year 07 Month 26 Day
Date of closure to data entry
2019 Year 06 Month 30 Day
Date trial data considered complete
Date analysis concluded
2019 Year 06 Month 30 Day

Other
Other related information

Management information
Registered date
2016 Year 01 Month 16 Day
Last modified on
2019 Year 01 Month 24 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000023759

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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