UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000033565 |
|---|---|
| Receipt number | 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 | 2024/07/16 20:06:33 |
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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
Under general anesthesia using a stereotactic brain surgery system, human iPSC-derived dopaminergic progenitors will be transplanted into the bilateral putamen. For the first 3 patients, approximately 2.4X10^6 cells will be delivered at each side. For the 4th and later patients, 4.2-5.4X10^6 cells will be transplanted at each side.
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.
Human artificial pluripotent cell processed product, Tacrolimus hydrate, Device for iPS Cell Injection(Tentative Name), Fluorodopa(18F), alovudine, GEH120714(18F), carbidopa
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
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
neuroofc@kuhp.kyoto-u.ac.jp
Sponsor or person
Institute
Kyoto University Hospital
Institute
Department
Personal name
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 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
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
7
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
Completed
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 | 09 | Month | 25 | Day |
Last follow-up date
| 2023 | Year | 12 | Month | 08 | Day |
Date of closure to data entry
| 2024 | Year | 01 | Month | 18 | Day |
Date trial data considered complete
| 2024 | Year | 01 | Month | 29 | Day |
Date analysis concluded
| 2024 | Year | 07 | Month | 02 | Day |
Other
Other related information
Management information
Registered date
| 2018 | Year | 07 | Month | 30 | Day |
Last modified on
| 2024 | Year | 07 | Month | 16 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000038279
