試験ID jRCT2033250070
最終情報更新日：2026年10月23日
AADC/GCH/TH発現AAVベクター線条体内投与による進行期パーキンソン病遺伝子治療の臨床試験
基本情報
| 試験ID | jRCT2033250070 | |
|---|---|---|
| 研究名称 / Scientific Title（Acronym） | AADC/GCH/TH発現AAVベクター線条体内投与による進行期パーキンソン病遺伝子治療の臨床試験 | Phase I/II Study of Gene Therapy for Parkinson's disease |
| 平易な研究名称 / Public Title（Acronym） | Phase I/II Study of Gene Therapy for Parkinson's disease | |
| 試験進捗状況/Recruitment status | Suspended | |
| 登録日時 | 2025年4月28日 | |
| 最終情報更新日 | 2026年10月23日 | |
| 試験開始日（予定日） | ||
| 試験終了日（予定日） | ||
| 組入れ開始日 / Date of first enrollment | ||
試験概要
| 試験実施地域 / Region | 日本 | |
|---|---|---|
| 実施都道府県 | 栃木県 | |
| 目標症例数/Target sample size | 12 | |
| 対象疾患 / Health condition(s) or Problem(s) studied | パーキンソン病 | Parkinson's disease |
| 試験のタイプ / Study type | 介入 | Interventional |
| 試験デザイン / Study design | ||
| ランダム化 / Randomization | ||
| 介入1 | パーキンソン病患者の線条体（被殻）に、定位脳手術の手法によってAADC/GCH/TH発現AAVベクターを注入する。 | |
| 介入2 | パーキンソン病患者の線条体（被殻）に、定位脳手術の手法によってAADC/GCH/TH発現AAVベクターを注入する。 | |
| 介入3 | パーキンソン病患者の線条体（被殻）に、定位脳手術の手法によってAADC/GCH/TH発現AAVベクターを注入する。 | |
| 介入4 | パーキンソン病患者の線条体（被殻）に、定位脳手術の手法によってAADC/GCH/TH発現AAVベクターを注入する。 | |
| 介入5 | パーキンソン病患者の線条体（被殻）に、定位脳手術の手法によってAADC/GCH/TH発現AAVベクターを注入する。 | |
| 介入6 | パーキンソン病患者の線条体（被殻）に、定位脳手術の手法によってAADC/GCH/TH発現AAVベクターを注入する。 | |
| 主要評価項目 / Primary outcomes | パーキンソン病に対し、AADC/GCH/TH発現AAVベクター被殻内投与をした際の安全性及び有効性を確認する。 | To examine the safety and efficacy in patients with Parkinson's disease, after intracerebral administrations of AAV-AADC/GCH/TH into the putamen. |
| 副次評価項目 / Secondary outcomes | パーキンソン病に対し、AADC/GCH/TH発現AAVベクター被殻内投与をした際の有効性を確認する。 | To examine the efficacy in patients with Parkinson's disease, after intracerebral administrations of AAV-AADC/GCH/TH into the putamen. |
適格性
| 年齢（下限）/ Age minimum | 40歳以上 | >= 40age old |
|---|---|---|
| 年齢（上限）/ Age maximum | 80歳未満 | <= 80age old |
| 性別 / Gender | 男女両方 | Both |
| 選択基準 / Include 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. | |
| 除外基準 / Exclude 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. |
責任研究者
| 責任研究者 / Name of lead principal investigator | Takeshi Nakajima | |
|---|---|---|
| 組織名 / Organization | ||
| 部署名 / Division | Jichi Medical University Hospital | |
| 住所 / Address | 3311-1,Yakushiji, Shimotsuke-shi, Tochigi Tochigi Japan 329-0498 | |
| 電話 / Telephone | +81-285-44-2111 | |
| 実施責任組織 / Affiliation | 中嶋 剛 | Jichi Medical University Hospital |
| 研究費提供組織 / Funding Source | ||
| 共同実施組織 / Funding Source | ||
| 受付ID |
試験問い合わせ窓口
| 住所 / Address | 3311-1,Yakushiji, Shimotsuke-shi, Tochigi Tochigi Japan 329-0498 | |
|---|---|---|
| 電話 / Telephone | +81-285-35-3373 | |
| ホームページURL | ||
| chiken@jichi.ac.jp | ||
| 担当者 / Name of contact person | Takeshi Nakajima |
倫理審査委員会
| 認定臨床研究審査委員会又は倫理審査委員会の名称 | ||
|---|---|---|
| 上記委員会の認定番号 | 住所 / Address | |
| 電話番号 | ||
| 審査受付番号 | ||
| 当該臨床研究に対する審査結果 | ||
| 認定臨床研究審査委員会の承認日 | ||
変更・中止の場合
| 中止届出日 | ||
|---|---|---|
| 中止年月日 | ||
| 中止の理由 | ||
終了の場合
| 終了届出日 | ||
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| 観察期間終了日 | ||
| 実施症例数 | ||
| 参加者の流れ（Participant flow） | ||
| 研究対象者の背景情報 | ||
| 疾病等の発生状況のまとめ | ||
| 主要評価項目及び副次評価項目のデータ解析及び結果 | ||
| 公開予定日 | ||
| 要約 | ||
| 研究実施計画書のURL | ||
| 結果に関する最初の出版物での発表日 | ||
| 結果と出版物に関するURL | ||
IPD data sharing
| 個々の研究対象者単位のデータ（IPD）を共有する計画 | ||
|---|---|---|
| 計画の説明 | ||