Parkinson's disease
Conditions
Interventions
AAV-AADC/GCH/TH is designed to be injected into the striatum (putamen) of the patients with Parkinson's disease, according to the stereotactic neurosurgical method.
Sponsors
Nakajima Takeshi
Japan Agency for Medical Research and Develop ment, ONODERA GT Pharma.Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Patients diagnosed with Parkinson's disease by MDS Clinical Diagnostic Criteria for Parkinson's Disease. -Patients aged 40 to 80 years at the time of obtaining informed consent. -Patients onset of an illness> 35 years of age. -Patients who have received L-dopa for at least 5 years. -Patients whose severity of Hoehn & Yahr in the OFF state at the time of informed consent was > IV. -Patients who do not achieve satisfactory therapeutic outcomes despite appropriate pharmacological treatment. -Patients whose severity of Hoehn & Yahr in the OFF state at the time of informed consent was> IV. -Patients with a baseline total MDS-UPDRS-III score (OFF) of 30 to 100. -Patients who show a response to L-dopa and have improved MDS-UPDRS-III by at least 16 points at baseline ON and OFF. -Untolerable motor complications in patients with a total score of at least 4 on MDS-UPDRS Part IV, Item B (motor fluctuations) at baseline. -Patients who can undergo stereotactic brain surgery. -Patients who are able to comply with the necessary conditions for this clinical trial, including frequent visits for assessments after administration of the investigational product. -Patients who do not need dosage changes for the Parkinson's Disease treatment for at least 8 weeks, prior to the start of the study substance administration. -Patients who are able to provide informed consent personally.
Exclusion criteria
Exclusion criteria: -Patients with a history of cerebrovascular disease, exposure to antipsychotics or toxins, encephalitis, symptoms such as progressive supranuclear palsy or cerebellar symptoms, pyramidal signs, autonomic signs, dementia, hallucinations or delusions, or MRI findings such as lachner infarction or atrophy of the midbrain tegment, pons and cerebellum suggesting secondary or atypical parkinsonism. -Patients who have already undergone stereotactic neurosurgery (pallidal coagulation, thalamic coagulation, deep brain stimulation) and MR-guided focused ultrasound therapy for Parkinson's disease. -Patients with a Mini-Mental State Examination (MMSE) score of 20 or less or with a diagnosis of dementia. -Patients with a history of schizophrenia or affective disorders. -Patients with apparent vascular diseases, including cerebrovascular disorders. -Patients with neoplastic disease of the central nervous system, clinically evident neurological disease (e.g., age-inappropriate, obvious brain atrophy). -Patients with a concomitant malignancy or a history of other malignancies other than previously treated skin cancers within 5 years before obtaining informed consent. -Patients with uncontrolled hypertension. -Patients with coagulopathy or who require antithrombotic therapy. -Patients with clinically prominent evidence of immunodeficiency who require immunosuppressant treatments excluding steroid therapy. -Patients with a score of 10 or more on the Geriatric Depression Scale-short form (GDS-S) or 5 or more on antidepressants. -Patients who cannot undergo MRI. -Individuals who are pregnant, may be pregnant, or wish to have children, except those who cryopreserve sperm or eggs prior to the administration of the investigational product and use them to conceive children. -Patients with a history of convulsive seizures within 3 years, those taking antiepileptic drugs, or those with abnormal electroencephalograms showing epileptic abnormalities. -Individuals with a history of or current hypersensitivity to the components used in the manufacturing process of the investigational product (Sf-RVN and Benzonase) or to the ingredients of the investigational product itself. -Patients with a history of severe drug allergies. -Patients who have participated in another clinical trial or intervention-based clinical study within 6 months prior to enrollment -Patients with the following complications that are hard to manage. 1.Patients with severe renal disorders of the magnitude of both the serum creatinine levels of > 2.0 mg/dl and BUN>25 mg/dl. 2.Patients with severe hepatic disorders of the magnitude of either AST/GOT or ALT/GPT is above 2.5 folds of the upper limit of the normal levels. 3.Patients with diabetes of the magnitude of both >200 mg/dl casual or postprandial blood glucose levels and >9% HbA1c. -Any patients whom the investigator or sub-investigator deems unsuitable for inclusion in this clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To examine the safety and efficacy in patients with Parkinson's disease, after intracerebral administrations of AAV-AADC/GCH/TH into the putamen. | — |
Secondary
| Measure | Time frame |
|---|---|
| To examine the efficacy in patients with Parkinson's disease, after intracerebral administrations of AAV-AADC/GCH/TH into the putamen. | — |
Contacts
Public ContactTakeshi Nakajima
Jichi Medical University Hospital
Outcome results
None listed