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自閉症スペクトラム障害児におけるhCT-MSC (IMPACT)

2026年5月28日 更新者：Joanne Kurtzberg, MD

自閉症スペクトラム障害児における臍帯由来間葉系間質細胞産物である hCT-MSC の第 II 相試験

このフェーズ II 研究の目的は、自閉症スペクトラム障害 (ASD) の子供の社会的コミュニケーション能力を改善するためのヒト臍帯組織由来間葉系間質細胞 (hCT-MSC) の有効性を判断することです。

調査の概要

状態

完了

条件

介入・治療

詳細な説明

この二重盲検第 II 相試験の目的は、自閉症スペクトラム障害 (ASD) の小児において、2 つの異なる投与戦略で投与されたヒト臍帯組織由来間葉系間質細胞 (hCT-MSC) の有効性を判断することです。

この研究では、4 ～ 11 歳の ASD の子供を登録します。 適格な被験者は、神経心理学的評価、EEGテスト、アイトラッキング、CVA評価、および研究製品の注入を受けます。 被験者は、2 つの研究群のうちの 1 つに無作為に割り付けられます。 1) ベースラインで 6.0x106 細胞/Kg の単回注入、その後 6 か月で盲検プラセボ注入、または 2) ベースラインでプラセボ注入、その後 6 か月で 6x106 細胞/Kg の静脈内投与。

この研究の主要評価項目は、ベースラインから 6 か月までの社会的コミュニケーション スキルの変化です。 MSC の注入に関連する潜在的なリスクには、製品への反応 (発疹、息切れ、喘鳴、呼吸困難、低血圧、口、喉または目の周りの腫れ、頻脈、発汗)、感染の伝播、および HLA 感作が含まれます。

研究の種類

介入

入学 (実際)

137

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

  • アメリカ
    • North Carolina
      • Durham、North Carolina、アメリカ、27705
        • Duke University Medical Center

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

4年～11年 (子)

健康ボランティアの受け入れ

いいえ

説明

包含基準

  1. -同意時の年齢が4歳以上12歳未満（11歳と364日）
  2. -自閉症の症状の簡単な観察（BOSA）および自閉症診断インタビュー改訂（ADI-R）によって通知されるDSM-5チェックリストを使用して、自閉症スペクトラム障害の臨床DSM-5診断を確認
  3. 壊れやすいXテストが実行され、陰性。 CMAおよび/または全エクソーム配列決定が実施され、結果は自閉症の診断にリンクされていません
  4. -研究製品の注入前の少なくとも2か月間、現在の精神医学的投薬計画（用量および投薬スケジュール）で安定している
  5. -正常な絶対リンパ球数（アフリカ系アメリカ人の参加者では≥1200 / uL、他のすべての参加者では≥1500 / uL）
  6. -研究担当者による認知テストによるGAI≧65
  7. 参加者と保護者は英語を話します
  8. デューク大学に 2 回 (ベースライン、6 か月) 渡航でき、保護者は中間調査とインタビューに参加できます
  9. 少なくとも 1 人の親/保護者による親/保護者の同意

除外基準

  1. 全般的：

    1. 医療記録および/またはスクリーニング評価のレビューは、ASD診断および/またはGAI > 65に自信がないことを示しています
    2. -次の共存する精神医学的状態のいずれかの既知の診断: うつ病, 双極性障害, 統合失調症, 双極性障害に関連する強迫性障害, トゥーレット症候群
    3. -スクリーニングデータは、参加者が研究チームによって評価された研究手順の要件を順守できないことを示唆しています
    4. 家族は、プロトコルのフォローアップを含む、すべての研究関連の評価への参加を望んでいない、または参加することができません
    5. 兄弟がこの（Duke IMPACT）研究に登録されている

  2. 遺伝:

    1. 記録によると、子供は、脆弱 X 症候群、神経線維腫症、レット症候群、結節性硬化症、PTEN 変異、嚢胞性線維症、筋ジストロフィー、または ASD に関連することが決定的に知られている遺伝的欠陥などの既知の遺伝的症候群を持っていることが示されています。
    2. -ASDに関連する既知の病原性変異またはコピー数変動（CNV）（例：16p11.2、 15q13.2、 2q13.3)

  3. 感染性:

    1. -既知の活動性CNS感染
    2. -記録または臨床評価に基づく制御されていない感染の証拠
    3. -既知のHIV陽性
    4. 過去 14 日間の COVID-19 への曝露、または過去 28 日間の COVID-19 検査陽性。 COVID-19 の過去の感染歴がある被験者は、最初の訪問の 14 日前から無症状でなければなりません。

  4. 医学：

    1. 既知の代謝障害
    2. 既知のミトコンドリア機能障害
    3. -不安定てんかんまたは制御不能な発作障害、乳児痙攣、レノックスガストー症候群、ドラベ症候群、または他の同様の慢性発作障害の病歴
    4. 進行中の悪性腫瘍または化学療法で治療された以前の悪性腫瘍
    5. -原発性免疫不全疾患の病歴
    6. -自己免疫性血球減少症の病歴（すなわち、ITP、AIHA）
    7. -子供を研究手順の合併症のリスクを高める併存する病状
    8. -将来の幹細胞移植を必要とする可能性のある同時遺伝性または後天性疾患または併存症（複数可）
    9. 重大な感覚障害（失明、難聴、未矯正の聴覚障害など）または運動障害（脳性麻痺など）
    10. -血清クレアチニン> 1.5mg / dLまたは総ビリルビン> 1.3mg / dLによって決定される腎機能または肝機能の障害、既知のギルバート病の患者を除く
    11. -次のように定義される重大な血液学的異常：ヘモグロビン<10.0 g / dL、血小板<150 x 10e9 / uL、WBC <3,000細胞/ mL、アフリカ系アメリカ人のALC <1200 / uLまたは他のすべての参加者のALC <1500 / uL。
    12. 神経発達状態に関連する異形の特徴を特定する訓練を受けた医学遺伝学者および精神科医を含む、PIまたは他の研究者によって評価された、遺伝的症候群を示す臨床的に関連する身体的異形の証拠。

  5. 現在/以前の治療:

    a.保管された認定自己臍帯血ユニットの入手可能性、または両親が認定自己臍帯血ユニットの使用を延期した ｂ． 以前の細胞療法の履歴 ｃ． ＮＳＡＩＤを除くＩＶＩＧまたは他の抗炎症薬の現在または以前の使用 ｄ． 現在または以前の免疫抑制療法 ｉ． -2週間を超えて持続した全身ステロイド療法はなく、登録前3か月以内に全身ステロイドはありません。 局所および吸入ステロイドは許可されています。

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：4倍

武器と介入

参加者グループ / アーム
介入・治療
実験的：MSC
6x10e6 細胞/kg の 1 回用量を静脈内投与。
生物学的：臍帯組織間葉系間質細胞
ヒト臍帯組織由来間葉系間質細胞 (hCT-MSC) は、同種の血縁関係のないドナーからの臍帯組織から分離および拡大されています。 6x10e6 細胞/kg の 1 回用量を静脈内投与。
プラセボコンパレーター：プラセボ注入
他の：プラセボ注入
プラセボ比較注入

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
Change on the Average Socialization and Communication Subscale Standard Scores on the Vineland Behavior Scales (VABS-3)
時間枠：Baseline, 6 months
The primary outcome measure is the mean of the Socialization and Communication Subscale Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from the Comprehensive Interview form. The primary endpoint is the change in this outcome measure from baseline to six months before the second infusion. A positive change in the scores indicates an improvement in socialization and communication. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months

二次結果の測定

結果測定
メジャーの説明
時間枠
Change in VABS-3 (Vineland Adaptive Behavior Scales) Socialization Standard Score
時間枠：Baseline, 6 months
The change in the Socialization Subscale Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from baseline to six months. Higher Socialization Standard scores indicate greater socialization. A positive change in the scores indicates an improvement in socialization. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months
Change in VABS-3 Communication Standard Score
時間枠：Baseline, 6 months
The change in the Communication Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from the Comprehensive Interview form from baseline to six months before the second infusion. Higher scores indicate greater communication. A positive change in the scores indicates an improvement in communication. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months
CGI-S (Clinical Global Impression - Severity of Illness) Overall Score
時間枠：6 months
The CGI-S Overall Score is a 7-point scale that requires the clinician to rate the severity of the participant's overall functioning and symptoms of autism at the time of assessment, relative to the clinician's experience with participants who have the same diagnosis. The clinician rates the severity of autism symptoms - 1, normal, no symptoms; 2, borderline level of symptoms; 3, mild symptoms; 4, moderate symptoms; 5, marked symptoms; 6, severe symptoms; or 7, extremely severe symptoms. The higher ratings indicate greater severity of overall functioning impairment.
6 months
CGI-I (Clinical Global Impression - Improvement) Overall Score
時間枠：6 months
The CGI-I Overall Score is a 7-point scale that requires the clinician to assess how much the participant's autism overall functioning and symptoms have improved or worsened relative to a baseline assessment. The symptoms are rated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. The lower scores indicate greater improvement.
6 months
Change in the Pediatric Quality of Life (PedsQL) Total Scale Score
時間枠：Baseline, 6 months
The PedsQL 4.0 Generic Core Scales is a 5-minute parent questionnaire that measures the child's functioning in the dimensions of physical, emotional, social, and school. The items use a Likert rating scale from 0 (Never) to 4 (Almost Always). This 0-4 scale is then transformed to 0=100, 1=75, 2=50, 3=25, and 4=0 for a reverse score. The Total Scale Score is then computed as the sum of all the items over the number of items answered on all the Scales for a total score range of 0 to 100. Higher scores indicate a better quality of life.
Baseline, 6 months

その他の成果指標

結果測定
メジャーの説明
時間枠
Number of Participants Experiencing an Infusion Reaction
時間枠：up to 12 months
To assess safety, participants were considered according to the treatment received at each time point, not what they were randomized. Patients received the infusion to which they were randomized at baseline and the alternate treatment at 6 months. Therefore, adverse events are reported by randomized treatment arm broken into periods: 0-6 months (post-baseline infusion, pre-six month infusion) and 6-12 months (post-six month infusion).
up to 12 months
Number of Participants Experiencing Product-related Infections
時間枠：up to 12 months
To assess safety, participants were considered according to the treatment received at each time point, not to what they were randomized. Patients received the infusion to which they are randomized at baseline, and the alternate treatment at 6 months. AEs are reported by randomized treatment arm broken into periods: 0-6 months (post-baseline infusion, pre-six month infusion) and 6-12 months (post-six month infusion). 6-12 month evaluation time points are further broken into whether or not the participant received a second infusion, since three MSC patients and one Placebo patient did not receive the 6-month incentive infusion but were still evaluated.
up to 12 months
Evidence of Formation of Anti-HLA Antibodies
時間枠：Baseline, 6 months, 12 months
Assess for anti-HLA antibodies
Baseline, 6 months, 12 months
Number of Participants Experiencing Graft Versus Host Disease (GVHD)
時間枠：up to 12 months
To assess safety, participants were considered according to the treatment received at each time point, not to what they were randomized. Patients received the infusion to which they are randomized at baseline, and the alternate treatment at 6 months. AEs are reported by randomized treatment arm broken into periods: 0-6 months (post-baseline infusion, pre-six month infusion) and 6-12 months (post-six month infusion). 6-12 month evaluation time points are further broken into whether or not the participant received a second infusion, since three MSC patients and one Placebo patient did not receive the 6-month incentive infusion but were still evaluated.
up to 12 months
Number of Participants Experiencing Unexpected Adverse Events Related to the Study Product
時間枠：up to 12 months
To assess safety, participants were considered according to the treatment received at each time point, not to what they were randomized. Patients received the infusion to which they are randomized at baseline, and the alternate treatment at 6 months. AEs are reported by randomized treatment arm broken into periods: 0-6 months (post-baseline infusion, pre-six month infusion) and 6-12 months (post-six month infusion). 6-12 month evaluation time points are further broken into whether or not the participant received a second infusion, since three MSC patients and one Placebo patient did not receive the 6-month incentive infusion but were still evaluated.
up to 12 months
Change on the Adaptive Behavior Composite Subscale Standard Score on the Vineland Behavior Scales (VABS-3)
時間枠：Baseline, 6 months
The change in the Vineland Adaptive Behavior Scales (VABS-3) Adaptive Behavior Composite Subscale from baseline to 6 months before the second infusion. Higher Adaptive Behavior Composite Standard scores indicate greater adaptive behavior. A positive change in the scores indicates an improvement in adaptive behavior. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months
Change in VABS-3 (Vineland Adaptive Behavior Scales) Daily Living Skills Standard Score
時間枠：Baseline, 6 months
The change in the Daily Living Skills Subscale Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from baseline to six months before the second infusion. Higher Daily Living Skills Standard scores indicate greater daily living skills. A positive change in the scores indicates an improvement in daily living skills. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months
Change in VABS-3 Motor Skills Standard Score
時間枠：Baseline, 6 months
The change in the Motor Skills Standard Scores on the Vineland Adaptive Behavior Scales (VABS-3) from the Comprehensive Interview form from baseline to six months before the second infusion. Higher scores indicate greater motor skills. A positive change in the scores indicates an improvement in motor skills. A standard score of 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Domain scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers.
Baseline, 6 months
Change in Aberrant Behavior Checklist-Community (ABC-C) Social Withdrawal
時間枠：Baseline, 6 months
The change in the Aberrant Behavior Checklist-Community (ABC-C) Social Withdrawal scale from baseline to six months before the second infusion. Higher scores indicate greater problems with social withdrawal. A positive change in the scores indicates a worsening in social withdrawal. This scale has 16 items that are scored on a 4-point Likert scale: 0 = not a problem, 1 = slight problem, 2 = moderately serious problem, 3 = severe problem. The individual items are added together to calculate the Social Withdrawal scale score.
Baseline, 6 months
Change in Pervasive Developmental Disorder Behavior Inventory (PDDBI) Total Score
時間枠：Baseline, 6 months
The change in the Pervasive Developmental Disorder Behavior Inventory (PDDBI) from baseline to 6 months before the second infusion. The PDDBI is a measure of problem behaviors and social, language, and learning or memory skills of children who have been diagnosed with autism spectrum disorder. Raw scores are converted to T-scores. The T-score has a mean of 50 with a standard deviation of 10 points with a range of 10-100. Higher scores indicate greater behavioral issues and a positive change in score indicates worsening.
Baseline, 6 months
Change in the Autism Impact Measure (AIM)
時間枠：Baseline, 6 months
The change in the Autism Impact Measure (AIM) from baseline to 6 months before second infusion. AIM is a measure of core Autism Spectrum Disorder Symptoms. It is a parent-report questionnaire that includes 41 core-symptom items rated on two corresponding 5-point scales: frequency (ranging from "never" to "always") and impact (ranging from "not at all" to "severely") over the previous two weeks. Frequency and impact ratings are combined, yielding a total score range of 82 to 410. Higher scores indicate greater symptom severity; a positive change in score indicates worsening of symptoms.
Baseline, 6 months
Change in the Behavior Rating Inventory of Executive Function (BRIEF) Emotional Control Subscale
時間枠：Baseline, 6 months
The change in the Behavior Rating Inventory of Executive Function (BRIEF) Emotional Control subscale from baseline to month 6 before the second infusion. BRIEF is a survey that assesses executive function and self-regulation with the following subscales: Inhibit, Shift, Emotional Control, Working Memory, and Planning and Organization. Higher scores indicate greater levels of dysfunction each respective domain. A positive change in score indicates worsening the respective domain. Raw scores for each domain are converted to t-scores (Mean = 50, SD = 10) and percentiles. T scores 60-64 are considered to be mildly elevated, 65-69 are considered to be potentially clinically elevated, and >= 70 are considered to be clinically elevated.
Baseline, 6 months
Change in the Behavior Rating Inventory of Executive Function (BRIEF) Working Memory Subscale
時間枠：Baseline, 6 months
The change in the Behavior Rating Inventory of Executive Function (BRIEF) Working Memory subscale from baseline to month 6 before the second infusion. The BREIF is a survey that assesses executive function and self-regulation with the following subscales: Inhibit, Shift, Emotional Control, Working Memory, and Planning and Organization. Higher scores indicate greater levels of dysfunction each respective domain. A positive change in score indicates worsening the respective domain. Raw scores for each domain are converted to t-scores (Mean = 50, SD = 10) and percentiles. T scores 60-64 are considered to be mildly elevated, 65-69 are considered to be potentially clinically elevated, and >= 70 are considered to be clinically elevated.
Baseline, 6 months
Change in the Behavior Rating Inventory of Executive Function (BRIEF) Inhibit Subscale
時間枠：Baseline, 6 months
The change in the Behavior Rating Inventory of Executive Function (BRIEF) Inhibit subscale from baseline to month 6 before the second infusion. The BRIEF is a survey that assesses executive function and self-regulation with the following subscales: Inhibit, Shift, Emotional Control, Working Memory, and Planning and Organization. Higher scores indicate greater levels of dysfunction each respective domain. A positive change in score indicates worsening the respective domain. Raw scores for each domain are converted to t-scores (Mean = 50, SD = 10) and percentiles. T scores 60-64 are considered to be mildly elevated, 65-69 are considered to be potentially clinically elevated, and >= 70 are considered to be clinically elevated.
Baseline, 6 months
Change in the Behavior Rating Inventory of Executive Function (BRIEF) Plan/Organization Subscale
時間枠：Baseline, 6 months
The change in the Behavior Rating Inventory of Executive Function (BRIEF) Plan/Organization subscale from baseline to month 6 before the second infusion. The BRIEF is a survey that assesses executive function and self-regulation with the following subscales: Inhibit, Shift, Emotional Control, Working Memory, and Planning and Organization. Higher scores indicate greater levels of dysfunction each respective domain. A positive change in score indicates worsening the respective domain. Raw scores for each domain are converted to t-scores (Mean = 50, SD = 10) and percentiles. T scores 60-64 are considered to be mildly elevated, 65-69 are considered to be potentially clinically elevated, and >= 70 are considered to be clinically elevated.
Baseline, 6 months
Change in the Behavior Rating Inventory of Executive Function (BRIEF) Shift Subscale
時間枠：Baseline, 6 months
The change in the Behavior Rating Inventory of Executive Function (BRIEF) Shift subscale from baseline to month 6 before the second infusion. The BRIEF is a survey that assesses executive function and self-regulation with the following subscales: Inhibit, Shift, Emotional Control, Working Memory, and Planning and Organization. Higher scores indicate greater levels of dysfunction each respective domain. A positive change in score indicates worsening the respective domain. Raw scores for each domain are converted to t-scores (Mean = 50, SD = 10) and percentiles. T scores 60-64 are considered to be mildly elevated, 65-69 are considered to be potentially clinically elevated, and >= 70 are considered to be clinically elevated.
Baseline, 6 months
Change in the Expressive Vocabulary Test (Third Edition, EVT-3)
時間枠：Baseline, 6 months
The change in the Expressive Vocabulary Test (Third Edition, EVT-3) from baseline to 6 months before the second infusion. The EVT-3 is a measure of a participant's ability to match a spoken word with an image of an object, action, or concept. The number of words that are retrieved is converted to a Standard Score, where 100 is the mean with a standard deviation of 15 points. A score of 100 should be understood as being similar to the typical population of the same age. Scores greater than or equal to 86 are considered adequate or above adequate. Scores less than or equal to 85 are considered moderately low to low and indicate the patient has a significant skill deficit when compared with similarly aged peers. A positive change in EVT-3 score indicates improvement.
Baseline, 6 months

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

Joanne Kurtzberg, MD

協力者

The Marcus Foundation
Cryo-Cell International

捜査官

  • 主任研究者：Lauren Franz, MBChB、Duke University
  • 主任研究者：Beth Shaz, MD、Duke University

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

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10月12日

一次修了 (実際)

2023年5月2日

研究の完了 (実際)

2024年2月1日

試験登録日

最初に提出

2019年9月12日

QC基準を満たした最初の提出物

2019年9月12日

最初の投稿 (実際)

2019年9月13日

学習記録の更新

投稿された最後の更新 (実際)

2026年6月24日

QC基準を満たした最後の更新が送信されました

2026年5月28日

最終確認日

2026年5月1日

詳しくは

本研究に関する用語

キーワード

追加の関連 MeSH 用語

その他の研究ID番号

  • Pro00113011
  • Pro00102894 (その他の識別子：Duke IRB)

個々の参加者データ (IPD) の計画

個々の参加者データ (IPD) を共有する予定はありますか?

いいえ

医薬品およびデバイス情報、研究文書

米国FDA規制医薬品の研究

はい

米国FDA規制機器製品の研究

いいえ

米国で製造され、米国から輸出された製品。

いいえ

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。

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