UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000017377 |
|---|---|
| Receipt number | R000019994 |
| Scientific Title | A Phase I /II Study of Intra-putaminal Infusion of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase in Subjects with Parkinson's Disease |
| Date of disclosure of the study information | 2015/05/01 |
| Last modified on | 2018/05/03 10:52:35 |
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Basic information
Public title
A Phase I /II Study of Intra-putaminal Infusion of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase in Subjects with Parkinson's Disease
Acronym
AADC Gene Therapy for Parkinson's Disease
Scientific Title
A Phase I /II Study of Intra-putaminal Infusion of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase in Subjects with Parkinson's Disease
Scientific Title:Acronym
AADC Gene Therapy for Parkinson's Disease
Region
| Japan |
Condition
Condition
Advanced Parkinson's Disease
Classification by specialty
| Neurology |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
The purpose of this study is to evaluate the safety, efficacy of intra-putaminal infusion of AAV-hAADC-2 (adeno-associated virus encoding human aromatic L-amino acid decarboxylase) by stereotaxic surgery in patients with advanced Parkinson's disease.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Pragmatic
Developmental phase
Phase I,II
Assessment
Primary outcomes
The safety of intra-putaminal infusion of AAV-hAADC-2 in patients with advanced Parkinson's disease
Key secondary outcomes
1.The efficacy of intra-putaminal infusion of AAV-hAADC-2 in patient, which is judged based on the symptom diary, the clinical evaluation and the required dose of L-dopa
2.The expression level of the AAV-hAADC-2 injected intra-putaminaly, which is judged by FMT-PET
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
| Gene |
Interventions/Control_1
Cohort 1: The target putamen for AAV-hAADC-2 infusion is identified on MRI image that has been taken prior to the operation, and then subjects will be bilaterally infused with a total volume of 200 micro L at a total of 4 sites (2 sites in left putamen, 2 sites in right putamen; 50 maicro L per site) at a flow rate of 3 maicro L per minute on Day 0.
The number of vector genomes (vg) administered in this cohort is 3x10^11 vg/subject.
Cohort 2:The target putamen for AAV-hAADC-2 infusion is identified on MRI image that has been taken prior to the operation, and then subjects will be bilaterally infused with a total volume of 600 micro L at a total of 4 sites (2 sites in left putamen, 2 sites in right putamen; 150 micro L per site) at a flow rate of 3 micro L per minute on Day 0.
The number of vector genomes (vg) administered in this cohort is 9x10^11 vg/subject.
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
| 35 | years-old | <= |
Age-upper limit
| 75 | years-old | >= |
Gender
Male and Female
Key inclusion criteria
1.Patients with idiopathic Parkinson's disease meet diagnostic criteria for Specified Disease designated by the Ministry of Health, Labour and Welfare (1995): the Research Committee of CNS Degenerative Disease, L-Dopa is effective in the early disease stage and no findings suggestive of CNS Degenerative Disease are found.
2.Age<_75 years at the time of medical treatment.
3.Age at onset>_35 years.
4.Duration of L-dopa therapy>_5 years.
5.Hoehn and Yahr Stage IV in OFF state at the onset of medical treatment.
6.Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Scale Part III (MDS-UPDRS-III), minimum motor score of 30 to a maximum motor score of 100 in OFF state.
7.Positive response to dopaminergic therapy as evidenced by remarkable improvement in MDS-UPDRS-III motor score between the defined "OFF" and "ON" state: a minimum 16 points improvement in the MDS-UPDRS-III after dopaminergic therapy.
8.Patients who can undergo the stereotaxic surgery for Parkinson's disease due to the intolerable motor complication minimum score of 4 to a maximum score of 9 in the MDS-UPDRS-IV part B (diurnal fluctuation of symptom), not responsive to optimal medical therapy.
9.To be able to comply with the requirements, including the frequent clinical examination after medical treatment, in this study.
10.To keep the therapeutic medicine for Parkinson's disease for at least 2 months prior to participation in this study.
11.Written informed consent.
Key exclusion criteria
1.Patients who is suspected secondary/atypical parkinsonism .
2.Patients with history of 3 hours or more of intensive or violent dyskinesias in the past 6 months.
3.Patients with previous the stereotaxy for Parkinson's disease.
4.MMSE<_20 or patient with a diagnosis of dementia in the neuropsychological evaluation.
5.Patients with medical history of Hallucination, Delusion, schizophrenia or affective disorder within 6 months of informed consent.
6.Patients with history of significant cardiovascular disease including cerebrovascular accident.
7.Malignant neoplasm in the brain, clinically significant neurological disease.
8.History of other malignancy, with the exception of treated carcinoma cutaneum, within 5 years.
9.Uncontrolled hypertension.
10.Coagulopathy or need for anticoagulant therapy.
11.Clinically significant immune dysfunction.
12.GDS short scale>_10 points, or if on antidepressant, the score>_5 points.
13.On MAO-A inhibitors, or antipsychotic medications.
14.Unable to scan MRI.
15.Cases without abnormal finding in FMT-PET.
16.Premenopausal female or male who desire impregnating a female.
17.Past medical history of convulsive seizure within 3 years or receiving antiepileptic drug or patients with epileptic aberrance in the electroencephalography.
18.Past medical history of serious drug allergy.
19.Patients who have participated in other clinical trial within 6 months.
20.Patients who meet any of the following criteria:
a)Serious renal disorder
b)Serious hepatic disorder
c)Serious diabetes
21.Any other patients judged by investigators to be inappropriate for the subject of this study.
Target sample size
6
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Shinichi Muramatsu |
Organization
Jichi Medical University
Division name
Department of Medicine, Division of Neurology
Zip code
Address
311-1 Yakushiji, Shimono-shi, Tochigi, Japan
TEL
+81-285-58-7352
muramats@jichi.ac.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Shinichi Muramatsu |
Organization
Department of Medicine
Division name
Division of Neurology
Zip code
Address
3311-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan
TEL
+81-285-58-7352
Homepage URL
muramats@jichi.ac.jp
Sponsor or person
Institute
Jichi Medical University
Institute
Department
Personal name
Funding Source
Organization
Gene Therapy Research Institution Co., Ltd
Organization
Division
Category of Funding Organization
Profit organization
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
Takara Bio Inc.
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Address
Tel
Secondary IDs
Secondary IDs
YES
Study ID_1
NCT02418598
Org. issuing International ID_1
Clinical Trial gov.
Study ID_2
Org. issuing International ID_2
IND to MHLW
Institutions
Institutions
自治医科大学附属病院（栃木県）
Jichi Medical University Hospital(Tochigi)
Other administrative information
Date of disclosure of the study information
| 2015 | Year | 05 | Month | 01 | 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
Completed
Date of protocol fixation
| 2014 | Year | 10 | Month | 10 | Day |
Date of IRB
Anticipated trial start date
| 2015 | Year | 04 | Month | 20 | Day |
Last follow-up date
| 2017 | Year | 12 | Month | 31 | Day |
Date of closure to data entry
| 2017 | Year | 12 | Month | 31 | Day |
Date trial data considered complete
| 2017 | Year | 12 | Month | 31 | Day |
Date analysis concluded
| 2018 | Year | 03 | Month | 31 | Day |
Other
Other related information
Management information
Registered date
| 2015 | Year | 05 | Month | 01 | Day |
Last modified on
| 2018 | Year | 05 | Month | 03 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000019994
