第 IIa 相無作為化プラセボ対照試験: iPD の疾患修飾療法としての間葉系幹細胞
2026年8月11日 更新者：Mya Schiess
特発性パーキンソン病の疾患修飾療法としての同種骨髄由来間葉系幹細胞のランダム化二重盲検プラセボ対照試験
この研究の目的は、パーキンソン病 (PD) の進行を遅らせるために、最も安全で効果的な同種骨髄由来間葉系幹細胞 (MSC) 注入の反復投与回数を選択することです。
調査の概要
詳細な説明
PDの疾患修飾療法としての二重盲検無作為対照試験における同種MSCの単一施設第IIa相試験。
このデザインには、45 人の患者を対象とする 3 つの治療群が含まれます。
研究の種類
介入
入学 (実際)
45
段階
- フェーズ2
連絡先と場所
このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。
研究場所
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Texas
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Houston、Texas、アメリカ、77030
- The University of Texas Health Science Center at Houston
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参加基準
研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。
適格基準
就学可能な年齢
50年～79年 (大人、高齢者)
健康ボランティアの受け入れ
いいえ
説明
包含基準:
- PDと運動緩慢の2つの主要徴候の存在を含む、英国の脳バンク基準によるパーキンソン病の診断。
- 軽度のミクロソミアから嗅覚障害。
- 3 以下の修正 Hoehn and Yahr ステージ。
- 3年から10年の間のPDの診断日
- ドーパミン作動性療法に対する強い反応。
除外基準:
- 非定型、血管性、または薬剤性パーキンソニズム。
- PD 症状の別の説明をサポートする非定型の DAT スキャンまたは MRI。
- -レボドパを含む薬を服用していない患者。
- 精神病または難治性幻覚の臨床的特徴。
- 25 未満のモントリオール認知評価 (MoCA) スコア。
- 制御されていない発作障害。
- 異常な腎臓および肝機能。
- -スクリーニングまたはベースライン訪問時の臨床的に難治性の起立性低血圧の存在。
- -ボディマス指数が35以上。
- 心臓病：うっ血性心不全の病歴、臨床的に重大な徐脈、2度または3度の房室ブロックの存在。
- 肺疾患：安静時または歩行時に酸素を必要とする COPD。または中程度から重度の喘息。
- -スクリーニング開始前の5年以内の活動性悪性腫瘍または悪性腫瘍の診断
- 現在の自殺念慮または行動。
- 自己免疫疾患または免疫不全状態の診断
- -中型または大型血管の脳血管障害の病歴。
- -意識喪失および残留神経症状を伴う外傷性脳損傷の病歴。
- -過去3か月以内に大手術を受けたか、その後6か月以内に計画されています。
- -スクリーニング訪問前の90日以内の治験薬の使用歴。
- PDの脳手術歴。
- 薬物乱用障害。
- -アクティブな抗凝固療法および/または異常なINR。
研究計画
このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：ランダム化
- 介入モデル：並列代入
- マスキング：トリプル
武器と介入
参加者グループ / アーム |
介入・治療 |
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実験的：間葉系幹細胞とプラセボ
10 X 10^6 MSC/kg の 2 回の注入と 1 回のプラセボ注入、すべての用量は 4 か月の間隔で投与されました。
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1回の投与量は10 X 10^6 MSC/kgです
他の名前：
プラセボは治験製品と同一ですが、間葉系幹細胞（MSC）は含まれません。
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実験的：間葉系幹細胞
10 X 10^6 MSC/kg を 3 回注入し、4 か月ごとに投与。
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1回の投与量は10 X 10^6 MSC/kgです
他の名前：
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プラセボコンパレーター：プラセボ
4か月ごとに3回のプラセボ投与。
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プラセボは治験製品と同一ですが、間葉系幹細胞（MSC）は含まれません。
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この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Bayesian Mean Estimate of the Proportion of Participants Achieving a ≥5-point Improvement on MDS-UPDRS Part III From Screening to Week 62, Compared Between Each Active MSC Dose Arm and Placebo
時間枠：From Baseline to Week 62
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The Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III assesses motor symptoms of Parkinson's disease. .
A responder was defined as a participant achieving a ≥5-point improvement (decrease of at least 5 points from screening) at Week 62. Reported are the Bayesian mean estimates of the proportion of participants achieving this response.
The 95% confidence interval represents the Bayesian 95% credible interval.
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From Baseline to Week 62
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Number of Participants With New-onset Organ Failure
時間枠：Baseline through week 88
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New-onset organ failure defined as a significant acute change in the kidney or liver function, leukocytosis, leukopenia or anemia sustained over 3 months; > 75 % reduction in GFR compared to baseline; altered liver function as defined by ALT >150 U/L and or total bilirubin >1.6 mg/dl; or leukopenia defined as < 4K WBC count or anemia as defined by Hgb < 12 for men and < 11 for women.
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Baseline through week 88
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Number of Participants Developing Donor-Specific Anti-HLA Antibodies
時間枠：Baseline through week 88
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Baseline through week 88
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Motor Function as Measured by the Timed-Up-and-Go (TUG) Scale
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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This test uses the time that a person takes to rise from a chair, walk 7 meters, turn around, walk back to the chair, and sit down.
Time is recorded in seconds, a longer duration of time indicates a worse outcome.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Number of Participants With Lifetime Suicidal Ideation (C-SSRS)
時間枠：Baseline
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The Columbia-Suicide Severity Rating Scale (C-SSRS) is an assessment tool that evaluates suicidal ideation and behavior.
Reported is the number of participants with a positive response to the lifetime suicidal ideation question, "Have you actually had any thoughts of killing yourself?"
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Baseline
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Parkinson's Disease Severity as Assessed by the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Total Score
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) assesses the severity of Parkinson's disease.
The total score ranges from 0 to 265, with higher scores indicating greater Parkinson's disease severity and disability.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Motor Symptoms of Parkinson's Disease as Assessed by the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III Motor Examination Score
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III Motor Examination assesses motor symptoms of Parkinson's disease.
The total score ranges from 0 to 132, with higher scores indicating greater motor impairment and more severe Parkinson's disease motor symptoms.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Global Measurement of Disability as Measured by the Change in the Screening "Off" Modified Hoehn and Yahr (H&Y)
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Modified Hoehn and Yahr (H&Y) Scale described the progress of Parkinson's disease.
Total score ranges from 0 to 5, with higher scores indicating a worse outcome.
The assessment was performed in the "off" medication state, and the score will be reported.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Quality of Life as Measured by the Modified Schwab and England Activities of Daily Living Scale (ADL)
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Schwab and England Activities of Daily Living (ADL) Scale assesses functional independence in individuals with Parkinson's disease.
Scores range from 0% to 100%, with higher percentages indicating greater independence and better functional ability in activities of daily living.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Quality of Life as Measured by the Parkinson's Disease Questionnaire-39 (PDQ-39)
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Parkinson's Disease Questionnaire-39 (PDQ-39) assesses health-related quality of life in individuals with Parkinson's disease.
Total scores range from 0 to 100, with higher scores indicating poorer quality of life.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Quality of Life as Measured by the EuroQol- 5 Dimension (EQ-5D) Index Score
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The EuroQol- 5 Dimension (EQ-5D) assesses quality of life.
The total score ranges between 0 to 1, a higher score indicating better quality of life.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Non- Motor Symptoms as Measured by the Non-Motor Symptoms Questionnaire (NMSQ)
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Non-Motor Symptoms Questionnaire (NMSQ) assesses the presence of non-motor symptoms associated with Parkinson's disease.
Total score ranges from 0 to 30, with a higher score indicating a greater number of non-motor symptoms.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Olfactory Function as Assessed by the University of Pennsylvania Smell Identification Test (UPSIT-40) Score
時間枠：Baseline, Week 49, Week 88
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The University of Pennsylvania Smell Identification Test (UPSIT-40) assesses olfactory function.
The total score ranges from 0 to 40 with a higher score indicating better olfactory function.
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Baseline, Week 49, Week 88
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Cognitive Function as Measured by the Montreal Cognitive Assessment (MoCA)
時間枠：Baseline, Week 49, Week 88
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The Montreal Cognitive Assessment (MoCA) assesses cognitive function.
The total score ranges from 0 to 30, a higher score indicates better cognitive function.
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Baseline, Week 49, Week 88
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Anxiety as Assessed by the Parkinson Anxiety Scale (PAS) Score
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Parkinson Anxiety Scale (PAS) assesses anxiety symptoms in individuals with Parkinson's disease.
The total score ranges from 0 to 48, with higher scores indicating greater anxiety severity.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Depressive Symptoms as Assessed by the Geriatric Depression Scale-Short Form (GDS-SF) Score
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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The Geriatric Depression Scale-Short Form (GDS-SF) assesses depressive symptoms.
The total score ranges from 0 to 15, with higher scores indicating greater depressive symptom severity.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Number of Participants Reporting Suicidal Ideation Since Last Visit
時間枠：Week 9, Week 27, Week 49, Week 62, Week 88
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The Columbia-Suicide Severity Rating Scale (C-SSRS) assesses suicidal ideation and behavior.
Reported is the number of participants with a positive response to the suicidal ideation question, "Have you actually had any thoughts of killing yourself?"
since the previous study visit.
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Week 9, Week 27, Week 49, Week 62, Week 88
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その他の成果指標
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Growth Factors
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Examples of these are Glial Derived Neurotrophic Factor, Brain Derived Neurotrophic Factor and Vascular Epidermal Growth Factor .
These will be measured by ELISA specific to blood and CSF.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Chemokines in Patient Blood Sample.
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Examples of these are CCL2 (MCP-1), CCL7 (MCP-3), CCL11 (Eotaxin) CCL2 (MDC), CXCL10 (IP-10), CX3CL1 (Fractalkine).
These will be measured by Magnetic Bead Panel - Multiplex Assay or ELISA, allowing for specificity in the blood.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Neurotransmitters
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Examples of these are homovanillic acid and 5-hydroxytryptamine.
These will be measured in CSF and blood by ELISA.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Alpha-synuclein Oligomers in the Blood (Serum or Plasma)
時間枠：Baseline, Week 40, Week 49
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Baseline, Week 40, Week 49
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Alpha-synuclein Oligomers in the Cerebral Spinal Fluid
時間枠：Baseline,week 49
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Baseline,week 49
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Measurement of Putative Paracrine Mechanism of MSCs as Measured by Concentration of Cytokines in Patient Blood Sample.
時間枠：Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Examples of these are IL-1beta, IL-6, TNF-alpha, COX-2 and PGE-2.
These are measured by Magnetic Bead Panel - Multiplex Assay or ELISA, allowing for specificity in the blood.
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Baseline, Week 9, Week 27, Week 40, Week 62, Week 88
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Measurement of Putative Paracrine Mechanism of MSCs Using Neuroimaging
時間枠：Baseline, week 40,week 88
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Baseline, week 40,week 88
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協力者と研究者
ここでは、この調査に関係する人々や組織を見つけることができます。
スポンサー
捜査官
- 主任研究者：Mya C Schiess, MD、The University of Texas Health Science Center, Houston
出版物と役立つリンク
研究に関する情報を入力する責任者は、自発的にこれらの出版物を提供します。これらは、研究に関連するあらゆるものに関するものである可能性があります。
一般刊行物
- Dorsey ER, Sherer T, Okun MS, Bloem BR. The Emerging Evidence of the Parkinson Pandemic. J Parkinsons Dis. 2018;8(s1):S3-S8. doi: 10.3233/JPD-181474.
- Braak H, Del Tredici K, Rub U, de Vos RA, Jansen Steur EN, Braak E. Staging of brain pathology related to sporadic Parkinson's disease. Neurobiol Aging. 2003 Mar-Apr;24(2):197-211. doi: 10.1016/s0197-4580(02)00065-9.
- Connolly BS, Lang AE. Pharmacological treatment of Parkinson disease: a review. JAMA. 2014 Apr 23-30;311(16):1670-83. doi: 10.1001/jama.2014.3654.
- Jellinger KA. Basic mechanisms of neurodegeneration: a critical update. J Cell Mol Med. 2010 Mar;14(3):457-87. doi: 10.1111/j.1582-4934.2010.01010.x. Epub 2010 Jan 11.
- Kortekaas R, Leenders KL, van Oostrom JC, Vaalburg W, Bart J, Willemsen AT, Hendrikse NH. Blood-brain barrier dysfunction in parkinsonian midbrain in vivo. Ann Neurol. 2005 Feb;57(2):176-9. doi: 10.1002/ana.20369.
- Gray MT, Woulfe JM. Striatal blood-brain barrier permeability in Parkinson's disease. J Cereb Blood Flow Metab. 2015 May;35(5):747-50. doi: 10.1038/jcbfm.2015.32. Epub 2015 Mar 11.
- Orr CF, Rowe DB, Halliday GM. An inflammatory review of Parkinson's disease. Prog Neurobiol. 2002 Dec;68(5):325-40. doi: 10.1016/s0301-0082(02)00127-2.
- Nagatsu T, Mogi M, Ichinose H, Togari A. Cytokines in Parkinson's disease. J Neural Transm Suppl. 2000;(58):143-51.
- Stypula G, Kunert-Radek J, Stepien H, Zylinska K, Pawlikowski M. Evaluation of interleukins, ACTH, cortisol and prolactin concentrations in the blood of patients with parkinson's disease. Neuroimmunomodulation. 1996 Mar-Jun;3(2-3):131-4. doi: 10.1159/000097237.
- Joyce N, Annett G, Wirthlin L, Olson S, Bauer G, Nolta JA. Mesenchymal stem cells for the treatment of neurodegenerative disease. Regen Med. 2010 Nov;5(6):933-46. doi: 10.2217/rme.10.72.
- Martinez-Lemus JD, Molony DA, Suescun J, Tharp E, Thomas TS, Green C, Onuigbo C, Ritter R 3rd, Schiess MC. Allogeneic bone marrow-derived mesenchymal stem cells in the aging kidney: secondary results of a Parkinson's disease clinical trial. Stem Cell Res Ther. 2025 Sep 24;16(1):493. doi: 10.1186/s13287-025-04577-y.
研究記録日
これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。
主要日程の研究
研究開始 (実際)
2020年11月9日
一次修了 (実際)
2023年7月30日
研究の完了 (実際)
2023年7月30日
試験登録日
最初に提出
2020年7月16日
QC基準を満たした最初の提出物
2020年8月6日
最初の投稿 (実際)
2020年8月10日
学習記録の更新
投稿された最後の更新 (実際)
2026年9月2日
QC基準を満たした最後の更新が送信されました
2026年8月11日
最終確認日
2026年8月1日
詳しくは
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。