UMIN-CTR Clinical Trial

BACK TOP
UMIN-CTR English Home Glossary (Simple) FAQ Search clinical trials

Name:
UMIN ID:

Recruitment status Suspended
Unique ID issued by UMIN UMIN000033565
Receipt No. R000038279
Scientific Title Kyoto Trial to Evaluate the Safety and Efficacy of Tacrolimus in the iPSC-based Therapy for Parkinson's Disease
Date of disclosure of the study information 2018/07/31
Last modified on 2019/09/13

* This page includes information on clinical trials registered in UMIN clinical trial registed system.
* We don't aim to advertise certain products or treatments


Basic information
Public title Kyoto Trial to Evaluate the Safety and Efficacy of Tacrolimus in the iPSC-based Therapy for Parkinson's Disease
Acronym Kyoto Trial to Evaluate the Safety and Efficacy of Tacrolimus in the iPSC-based Therapy for Parkinson's Disease
Scientific Title Kyoto Trial to Evaluate the Safety and Efficacy of Tacrolimus in the iPSC-based Therapy for Parkinson's Disease
Scientific Title:Acronym Kyoto Trial to Evaluate the Safety and Efficacy of Tacrolimus in the iPSC-based Therapy for Parkinson's Disease
Region
Japan

Condition
Condition Parkinson's disease
Classification by specialty
Neurology
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 To evaluate the safety and efficacy of tacrolimus in patients with Parkinson's disease after transplantation of human induced pluripotent stem cell-derived dopaminergic progenitors into the corpus striatum
Basic objectives2 Safety,Efficacy
Basic objectives -Others
Trial characteristics_1 Confirmatory
Trial characteristics_2
Developmental phase Phase III

Assessment
Primary outcomes 1)Cumulative rejection-free rate (non-rejection rate) at 12 weeks after transplantation
2)Cumulative rejection-free rate (non-rejection rate) at 12 months after transplantation
Key secondary outcomes 1)Cumulative engraftment rate of cells at 12 months after transplantation
2)Cumulative survival rate of patients at 12 months after transplantation
3)Cumulative rejection-free rate (non-rejection rate) at 24 months after transplantation
4)Cumulative engraftment rate of cells at 24 months after transplantation
5)Cumulative survival rate of patients at 24 months after transplantation

[Safety evaluation]
Incidence and severity of adverse events, and laboratory values
[Evaluation of blood drug concentration]
Time course of trough blood concentration of tacrolimus

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
Other
Interventions/Control_1 Approximately 2.4 X 10^6 human iPSC-derived dopaminergic progenitors per side will be transplanted into the bilateral putamen of each subject under general anesthesia using a stereotactic brain surgery system. Immunosuppressant treatment will be started in the morning on the day of transplantation, continued until 52 weeks after transplantation, and then tapered off to zero over 12 weeks. In the early phase, the immunosuppressant is orally administered 0.03 to 0.15 mg/kg, twice a day, and the targeted blood concentration is within a range of 5-10 ng/mL as a trough value.
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
50 years-old <=
Age-upper limit
70 years-old >
Gender Male and Female
Key inclusion criteria 1)The patient has a diagnosis of PD (clinically established or clinically probable) in accordance with the MDS Clinical Diagnostic Criteria for Parkinson's Disease (2015).
2)The patient plans to undergo transplantation of human iPSC-derived dopaminergic progenitors.
3)The patient has a poor response to existing drug treatments.
4)The patient is >= 50 years and < 70 years of age at the time of informed consent.
5)The patient has had PD for at least 5 years.
6)The patient has both ON and OFF (as demonstrated by the MDS-UPDRS Part III and a symptom diary).
7)The patient is in stage 3 or higher on the Hoehn and Yahr scale at OFF time.
8)The patient is in stage 3 or lower on the Hoehn and Yahr scale at ON time.
9)The patient has an L-dopa response of 30% or more without influence of antiparkinsonian drugs.
10)The patient has a decrease pattern characteristic to PD in the basal ganglia region on DAT scan.
11)The patient has the following organ functions as determined by laboratory tests within 7 days before registration:
i)Neutrophil count >= 2,000/microL
ii)Platelet count >= 5.0 X 10^4/microL
iii)AST, ALT =< 3.0 X upper limit of normal at the study site
iv)Total bilirubin =< 1.5 X upper limit of normal at the study site
v)eGFR >= 60 mL/min/1.73 m2
vi)eGFR (mL/min/1.73 m2) = 194 X Cr^-1.094 X age^-0.287 (X 0.739 for females)
12)The patient provides written informed consent to participate in the study.
A patient who cannot write due to the disease may be enrolled if he or she provides verbal consent and a witness signs the informed consent form.
Key exclusion criteria 1)The patient has a symptomatic organic lesion as detected by head MRI.
2)The patient has abnormal immune function.
3)The patient is demented or deemed at high risk of dementia.
4)The patient has bleeding tendency or abnormal coagulation function.
5)The patient is HBs antigen-positive, or HBs antibody- or HBc antibody-positive with evidence of HBV-DNA.
6)The patient is anti-HIV antibody-positive.
7)The patient is anti-HTLV-1 antibody-positive.
8)The patient has active infection such as hepatitis C or syphilis (STS/TPHA).
9)The patient has contraindication to the study drug (tacrolimus), concomitant drugs (e.g., levodopa, carbidopa, MRI contrast), and/or their components.
10)The patient has hypersensitivity to the study drug (tacrolimus), concomitant drugs (e.g., levodopa, carbidopa, MRI contrast), and/or their components.
11)The patient has severe allergic to gentamicin, a bovine-derived ingredient l or a pig-derived ingredient.
12)The patient has undergone transplantation of human iPSC-derived dopaminergic progenitors.
13)The patient has any of the following diseases concurrently:
. Malignant neoplasm
. Epilepsy
. Mental disease (e.g., depression, bipolar disorder, schizophrenia)
. Other serious concurrent diseases (e.g., cerebrovascular disorder, heart disease, chronic respiratory disease, inadequately controlled hypertension, diabetes mellitus)
14) The patient has a history of any of the following:
. Malignant neoplasm
. Epilepsy
. Cerebral hemorrhage
. Mental disease (e.g., depression, bipolar disorder, schizophrenia)
. Pallidotomy, thalamotomy, or deep brain stimulation
15)The patient is pregnant or lactating, or does not agree to avoid pregnancy throughout the study.
16)The patient, in the opinion of the investigator or subinvestigator, is not appropriate to conduct the study safely.
Target sample size 7

Research contact person
Name of lead principal investigator
1st name Ryosuke
Middle name
Last name Takahashi
Organization Kyoto University Hospital
Division name Department of Neurology
Zip code 606-8507
Address 54 Kawahara-cho, Shogoin, Sakyo-ku, Kyoto 606-8507
TEL 075-751-3771
Email neuroofc@kuhp.kyoto-u.ac.jp

Public contact
Name of contact person
1st name Nobukatsu
Middle name
Last name Sawamoto
Organization Kyoto University Hospital
Division name Department of Neurology
Zip code 606-8507
Address 54 Kawahara-cho, Shogoin, Sakyo-ku, Kyoto 606-8507
TEL 075-751-3771
Homepage URL
Email neuroofc@kuhp.kyoto-u.ac.jp

Sponsor
Institute Kyoto University Hospital
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
Name of secondary funder(s) Sumitomo Dainippon Pharma Co., Ltd.

IRB Contact (For public release)
Organization Kyoto University Hospital Institutional Review Board
Address 54 Kawahara-cho, Shogoin, Sakyo-ku, Kyoto 606-8507
Tel 075-751-4389
Email tiken@kuhp.kyoto-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 初回届出年月日:2018年6月4日  届出回数:第1回

Institutions
Institutions 京都大学医学部附属病院(京都府)

Other administrative information
Date of disclosure of the study information
2018 Year 07 Month 31 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 Suspended
Date of protocol fixation
2018 Year 04 Month 06 Day
Date of IRB
2018 Year 05 Month 08 Day
Anticipated trial start date
2018 Year 08 Month 01 Day
Last follow-up date
2023 Year 03 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2018 Year 07 Month 30 Day
Last modified on
2019 Year 09 Month 13 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000038279

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


Contact us.