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

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 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


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

Value
https://center6.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