UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000020591 |
|---|---|
| Receipt number | 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 20:12:50 |
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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
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
koseki@cardiology.med.osaka-u.ac.jp
Sponsor or person
Institute
Osaka University Graduate School of Medicine
Institute
Department
Personal name
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
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000023759
