アンジェルマン症候群の自然史研究 (GLOW-AS)
2026年8月19日 更新者：Christopher Keary、Massachusetts General Hospital
アンジェルマン症候群の成人および小児を対象とした縦断的自然経過観察研究
この観察研究の目的は、遺伝子診断で確認されたアンジェルマン症候群（AS）を持つ小児および成人におけるASの自然経過について理解することです。 本研究が答えようとする主な質問は以下の通りです：
- ASを持つ人々において、コミュニケーション、運動能力、適応行動などの発達スキルは、1年間の期間でどのように変化するか？
- 脳活動や睡眠に特定のパターンがあり、それらが時間の経過に伴うAS症状の変化と関連しているか？
参加者は以下のことを行います：
- 1年間に5回（約3か月ごと）に研究施設を訪問して評価を受けます。
- 介護者の助けを借りて、発達、行動、睡眠に関する検査や質問票を完了します。
- 脳活動を測定するために脳波検査（EEG）を受け、自宅で睡眠モニタリングデバイス（アクティグラフィデータを収集するため）を装着します。
調査の概要
状態
募集
条件
詳細な説明
- 40名のAS患者を対象とした縦断的自然歴研究で、思春期集団を重点的に取り上げています。
- ASの臨床試験の将来の設計と解釈を支援する規範データの確立を目指し、ASのより正確な診断ツールと標的治療介入の開発をサポートします。
- 標準化された評価を通じて、適応行動、コミュニケーション、問題行動、一般的な健康状態、全体的な疾患重症度、および一般的な併存疾患（てんかんや睡眠障害を含む）を追跡します。
- EEGと活動量計記録を通じて収集されたバイオマーカーデータを分析し、臨床転帰および神経生理学的測定値の加齢に伴う変化を評価します。
- 参加者は12ヶ月間に最大5回の対面研究訪問に参加します：第0週、第13週、第26週、第39週、第52週。
研究の種類
観察的
入学 (推定)
40
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。
研究連絡先
- 名前：Leena Phan
- 電話番号：781-860-1711
- メール：MGHGLOW-AS@mgb.org
研究連絡先のバックアップ
- 名前：Riley Shubb
- 電話番号：781-860-1711
- メール：MGHGLOW-AS@mgb.org
研究場所
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Massachusetts
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Lexington、Massachusetts、アメリカ、02421
- 募集
- MGH Lurie Center for Autism
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主任研究者：
- Christopher J Keary, MD
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副調査官：
- Ronald L Thibert, DO, MSPH
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コンタクト：
- Leena Phan
- 電話番号：781-860-1711
- メール：MGHGLOW-AS@mgb.org
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コンタクト：
- Riley Shubb
- 電話番号：781-860-1711
- メール：MGHGLOW-AS@mgb.org
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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
- 子
- 大人
- 高齢者
健康ボランティアの受け入れ
いいえ
サンプリング方法
非確率サンプル
調査対象母集団
遺伝子検査で確認されたASの診断を受けている、少なくとも1歳以上の方。
説明
適格基準:
- 参加者は、ヒト15q11.2-q13.3遺伝子座内のUBE3A遺伝子機能に影響を与える文書化された遺伝的変異を伴う、アンジェルマン症候群の主要な臨床診断を有している。併存疾患（例：自閉症スペクトラム障害、脳性麻痺、知的障害）は許可されるが、アンジェルマン症候群が主要な臨床診断である必要がある。
- 参加者は男性または女性（出生時割り当て性別）であり、初回研究訪問時に年齢が1歳以上である。
- 参加者は、以下に示す研究パートナー基準を満たす研究パートナーを有している。
- インフォームドコンセントを提供できない参加者は、現在の国際調和会議（ICH）ガイドラインおよび適用される施設規制に従って参加者に代わってインフォームドコンセントを提供する意思と能力を有する、少なくとも18歳以上の適切な代理人を有している。
個人は、研究パートナーとして登録されるために以下の基準を満たさなければならない:
- 研究パートナーは、少なくとも18歳以上の親または主介護者である。
- 研究パートナーは参加者と定期的に接触しており、研究者の意見では、参加者の継続的な状態について正確かつ最新の情報を提供するのに十分な知識を有している。
- 研究パートナーは、研究パートナー評価を完了するのに十分な英語能力を有している。
- 研究パートナーは、ICHガイドラインおよび適用される施設規制に従って、自己のためにインフォームドコンセントを提供する意思と能力を有している。
- 研究パートナーは、研究者の意見では、信頼性と能力を有し；全ての研究訪問に参加者に同伴し、研究手順に従う意思と能力を有し；必要に応じて電話または電子メールで連絡可能であり；参加者の健康と幸福に関する正確かつ最新の情報を提供するのに十分な、参加者の継続的な状態についての知識を有している。
除外基準:
- 参加者は、ヒト15q11.2-q13.3遺伝子座外に、発達障害の可能性または既知の原因となる少なくとも1つの追加の既知の遺伝的異常を有している。
- 参加者が使用している少なくとも1つの標準的治療（薬物または補助療法）が、初回研究訪問前28日間（4週間）に変更された。治療には、抗てんかん薬の用量、行動管理薬、睡眠薬、ガバペンチン、カンナビジオール、特別食、サプリメント、言語療法、作業療法、応用行動分析（ABA）、心理社会的介入、理学療法、または栄養サポートが含まれるがこれらに限定されない。
- 参加者は、初回研究訪問前28日間（4週間）以内に、発作関連の懸念で救急部門を受診または入院したと定義される不安定なてんかんを有している。
- 参加者は妊娠可能であり、妊娠中、授乳中、または適切な避妊法を使用していない；禁欲は許容される。
- 参加者は、臨床的に関連する悪性腫瘍の既往歴；臨床的に有意な異常検査結果；臨床的に有意な心血管、血液、肝臓、筋肉、神経、または腎臓疾患；または研究者の意見では、参加を不適切にする他の臨床的イベントを経験している。
- 参加者は、アンチセンスオリゴヌクレオチドまたは遺伝子編集療法を含む、細胞または遺伝子ベースの治療の生涯治療歴を有している。
- 参加者は、初回研究訪問前28日または5半減期（いずれか長い方）以内に、細胞または遺伝子ベースの治療以外の研究治療を何らか受けている。
- 参加者は、計画された観察期間中に、研究薬剤または機器を含む介入研究に現在登録されている、または登録を計画している。
- 参加者は、脳波検査、活動量計、または評価スケジュールに記載された他の研究手順に対する既知の禁忌を有している。
- 参加者または研究パートナーは、研究者の意見では、研究要件を満たせないことを含め、他のいかなる方法でも参加に不適切である。
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
コホートと介入
グループ/コホート |
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参加者年齢1歳から17歳まで（含む）
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1歳から12歳までの参加者（両端を含む）
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18歳以上の参加者
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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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Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Cognitive Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 26, Week 52
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The Bayley-4 Cognitive subscale assesses cognitive development in children.
Growth score equivalents are derived from developmental growth scale values (GSVs) that provide an equal-interval scale for measuring developmental change over time.
Higher scores indicate greater cognitive development.
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Week 0, Week 26, Week 52
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Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Receptive Communication Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 26, Week 52
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The Bayley-4 Receptive Communication subscale assesses receptive language skills.
Growth score equivalents are derived from developmental GSVs that provide an equal-interval scale for measuring developmental change over time.
Higher scores indicate more advanced receptive communication abilities.
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Week 0, Week 26, Week 52
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Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Expressive Communication Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 26, Week 52
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The Bayley-4 Expressive Communication subscale assesses expressive language skills.
Growth score equivalents are derived from developmental GSVs that provide an equal-interval scale for measuring developmental change over time.
Higher scores indicate more developed expressive communication abilities.
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Week 0, Week 26, Week 52
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Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Fine Motor Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 26, Week 52
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The Bayley-4 Fine Motor subscale assesses fine motor skill development.
Growth score equivalents are derived from developmental GSVs that provide an equal-interval scale for measuring developmental change over time.
Higher scores indicate more developed fine motor skills.
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Week 0, Week 26, Week 52
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Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Gross Motor Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 26, Week 52
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The Bayley-4 Gross Motor subscale assesses gross motor skill development.
Growth score equivalents are derived from developmental GSVs that provide an equal-interval scale for measuring developmental change over time.
Higher scores indicate more developed gross motor skills.
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Week 0, Week 26, Week 52
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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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Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Cognitive Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 26, Week 52
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The Bayley-4 Cognitive subscale age-equivalent score represents the age in months at which the participant's raw score is the median score for typically developing children.
Higher age-equivalent scores indicate more advanced cognitive development.
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Week 0, Week 26, Week 52
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Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Expressive Communication Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 26, Week 52
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The Bayley-4 Expressive Communication subscale age-equivalent score represents the age in months at which the participant's raw score is the median score for typically developing children.
Higher age-equivalent scores indicate more advanced expressive communication abilities.
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Week 0, Week 26, Week 52
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Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Receptive Communication Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 26, Week 52
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The Bayley-4 Receptive Communication subscale age-equivalent score represents the age in months at which the participant's raw score is the median score for typically developing children.
Higher age-equivalent scores indicate more advanced receptive communication abilities.
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Week 0, Week 26, Week 52
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Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Fine Motor Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 26, Week 52
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The Bayley-4 Fine Motor subscale age-equivalent score represents the age in months at which the participant's raw score is the median score for typically developing children.
Higher age-equivalent scores indicate more advanced fine motor development.
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Week 0, Week 26, Week 52
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Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Gross Motor Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 26, Week 52
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The Bayley-4 Gross Motor subscale age-equivalent score represents the age in months at which the participant's raw score is the median score for typically developing children.
Higher age-equivalent scores indicate more advanced gross motor development.
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Week 0, Week 26, Week 52
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Change from Baseline in Symptoms of Angelman Syndrome-Clinical Global Impressions Scale of Severity (SAS-CGI-S) Global Severity Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The SAS-CGI-S is a clinician-rated assessment of global symptom severity in Angelman syndrome.
Scores range from 1 (normal, not at all ill) to 7 (among the most extremely ill patients), with higher scores indicating greater global symptom severity.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Aberrant Behavior Checklist-Community (ABC-C) Hyperactivity/Noncompliance Subscale Score (ABC-H) at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The ABC-C Hyperactivity subscale assesses hyperactive and non-compliant behaviors.
Scores range from 0 to 48, with higher scores indicating more severe hyperactivity symptoms.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Aberrant Behavior Checklist-Community (ABC-C) Irritability Subscale Score (ABC-I) at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The ABC-C Irritability subscale assesses irritability, aggression, self-injury, and negative emotional states.
Scores range from 0 to 45, with higher scores indicating more severe irritability symptoms.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Caregiver-Reported Angelman Syndrome Scale for Impact (CASS-I) Overall AS Symptoms Rating at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The CASS-I is a caregiver-reported assessment of overall symptom severity and impact in Angelman syndrome.
The scale provides a global rating of AS symptoms from the caregiver's perspective, with higher ratings indicating greater symptom severity and impact.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) Total Sleep Disturbance Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The CSHQ is a parent-report questionnaire assessing sleep problems in children.
The total score ranges from 33 to 99, with higher scores indicating greater sleep disturbance.
A total score of 41 or higher indicates clinically significant sleep problems.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) Sleep Duration Subscale Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The CSHQ Sleep Duration subscale assesses the adequacy of sleep duration.
Higher scores indicate shorter or insufficient sleep duration.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) Night Wakings Subscale Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The CSHQ Night Wakings subscale assesses the frequency and severity of nighttime awakenings.
Higher scores indicate more frequent or problematic night wakings.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Observer-Reported Communication Ability (ORCA) Overall Communication T-Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The ORCA is an observer-reported measure of communication abilities in individuals with Angelman syndrome.
T-scores are standardized scores with a mean of 50 and standard deviation of 10.
Higher t-scores indicate greater proficiency in overall communication abilities.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Observer-Reported Communication Ability (ORCA) Expressive Communication T-Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The ORCA Expressive Communication subscale assesses expressive language abilities.
T-scores are standardized scores with a mean of 50 and standard deviation of 10.
Higher t-scores indicate greater proficiency in expressive communication.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Observer-Reported Communication Ability (ORCA) Receptive Communication T-Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The ORCA Receptive Communication subscale assesses receptive language abilities.
T-scores are standardized scores with a mean of 50 and standard deviation of 10.
Higher t-scores indicate greater proficiency in receptive communication.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Observer-Reported Communication Ability (ORCA) Pragmatic Communication T-Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The ORCA Pragmatic Communication subscale assesses social and contextual use of communication.
T-scores are standardized scores with a mean of 50 and standard deviation of 10.
Higher t-scores indicate greater proficiency in pragmatic communication.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Receptive Communication Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Receptive Communication subdomain age-equivalent score represents the age in years and months at which the participant's score is typical for an average individual.
Higher age-equivalent scores indicate more advanced receptive communication skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Receptive Communication Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Receptive Communication GSV provides an equal-interval scale for measuring developmental change over time.
Higher growth score equivalents indicate greater developmental progress in receptive communication.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Expressive Communication Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Expressive Communication subdomain age-equivalent score represents the age in years and months at which the participant's score is typical for an average individual.
Higher age-equivalent scores indicate more advanced expressive communication skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Expressive Communication Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Expressive Communication subdomain GSV provides an equal-interval scale for measuring developmental change over time.
Higher growth score equivalents indicate greater developmental progress in expressive communication.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Personal Daily Living Skills Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Personal Daily Living Skills subdomain age-equivalent score represents the age in years and months at which the participant's score is typical for an average individual.
Higher age-equivalent scores indicate more advanced personal daily living skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Personal Daily Living Skills Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Personal Daily Living Skills subdomain GSV provides an equal-interval scale for measuring developmental change over time.
Higher growth score equivalents indicate greater developmental progress in personal daily living skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Domestic Daily Living Skills Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Domestic Daily Living Skills subdomain age-equivalent score represents the age in years and months at which the participant's score is typical for an average individual.
Higher age-equivalent scores indicate more advanced domestic daily living skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Domestic Daily Living Skills Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Domestic Daily Living Skills subdomain GSV provides an equal-interval scale for measuring developmental change over time.
Higher growth score equivalents indicate greater developmental progress in domestic daily living skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Community Daily Living Skills Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Community Daily Living Skills subdomain age-equivalent score represents the age in years and months at which the participant's score is typical for an average individual.
Higher age-equivalent scores indicate more advanced community daily living skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Community Daily Living Skills Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Community Daily Living Skills subdomain GSV provides an equal-interval scale for measuring developmental change over time.
Higher growth score equivalents indicate greater developmental progress in community daily living skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Interpersonal Relationships Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Interpersonal Relationships subdomain age-equivalent score represents the age in years and months at which the participant's score is typical for an average individual.
Higher age-equivalent scores indicate more advanced interpersonal relationship skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Interpersonal Relationships Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Interpersonal Relationships subdomain GSV provides an equal-interval scale for measuring developmental change over time.
Higher growth score equivalents indicate greater developmental progress in interpersonal relationships.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Play and Leisure Time Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Play and Leisure Time subdomain age-equivalent score represents the age in years and months at which the participant's score is typical for an average individual.
Higher age-equivalent scores indicate more advanced play and leisure skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Play and Leisure Time Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Play and Leisure Time subdomain GSV provides an equal-interval scale for measuring developmental change over time.
Higher growth score equivalents indicate greater developmental progress in play and leisure skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Coping Skills Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Coping Skills subdomain age-equivalent score represents the age in years and months at which the participant's score is typical for an average individual.
Higher age-equivalent scores indicate more advanced coping skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Coping Skills Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
|
The Vineland-3 Coping Skills subdomain GSV provides an equal-interval scale for measuring developmental change over time.
Higher growth score equivalents indicate greater developmental progress in coping skills.
|
Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Gross Motor Skills Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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The Vineland-3 Gross Motor Skills subdomain age-equivalent score represents the age in years and months at which the participant's score is typical for an average individual.
Higher age-equivalent scores indicate more advanced gross motor skills.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Gross Motor Skills Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
|
The Vineland-3 Gross Motor Skills subdomain GSV provides an equal-interval scale for measuring developmental change over time.
Higher growth score equivalents indicate greater developmental progress of gross motor skills.
|
Week 0, Week 13, Week 26, Week 39, Week 52
|
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Fine Motor Skills Age-Equivalent Score at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
|
The Vineland-3 Fine Motor Skills subdomain age-equivalent score represents the age in years and months at which the participant's score is typical for an average individual.
Higher age-equivalent scores indicate more advanced fine motor skills.
|
Week 0, Week 13, Week 26, Week 39, Week 52
|
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Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Fine Motor Skills Growth Score Equivalent at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
|
The Vineland-3 Fine Motor Skills subdomain GSV provides an equal-interval scale for measuring developmental change over time.
Higher growth score equivalents indicate greater developmental progress in fine motor skills.
|
Week 0, Week 13, Week 26, Week 39, Week 52
|
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Change from Baseline in Frequency of Reported Seizure Events at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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Seizure frequency is assessed through caregiver-reported seizure diaries documenting the number and type of seizure events over each measurement period.
Seizure counts are analyzed as rates to account for variability in the duration of time covered by seizure diaries.
Lower seizure rates indicate decreased seizure frequency.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Average Total Sleep Time Measured by Actigraphy at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
|
Total sleep time is the total amount of sleep obtained during the sleep period as measured by actigraphy, expressed in minutes.
Higher values indicate longer sleep duration.
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Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Average Effective Sleep Measured by Actigraphy at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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Effective sleep is the percentage of time asleep during the sleep period as measured by actigraphy.
Values range from 0% to 100%, with higher percentages indicating more consolidated sleep.
|
Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Average Sleep Efficiency Measured by Actigraphy at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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Sleep efficiency is the ratio of total sleep time to time in bed, expressed as a percentage.
Values range from 0% to 100%, with higher percentages indicating greater sleep efficiency.
|
Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Average Number of Nocturnal Awakenings Measured by Actigraphy at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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Nocturnal awakenings are the number of times a participant awakens during the sleep period as measured by actigraphy.
Lower values indicate fewer sleep disruptions.
|
Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Average Duration of Nocturnal Awakenings Measured by Actigraphy at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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Duration of nocturnal awakenings is the average length of time spent awake during the sleep period as measured by actigraphy, expressed in minutes.
Lower values indicate shorter awakening durations.
|
Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Average Sleep Onset Latency Measured by Actigraphy at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
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Sleep onset latency is the time from attempting to sleep to the onset of sleep as measured by actigraphy, expressed in minutes.
Lower values indicate faster sleep onset.
|
Week 0, Week 13, Week 26, Week 39, Week 52
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Change from Baseline in Average Total Sleep Time Reported in Sleep Diaries at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
|
Total sleep time as reported in caregiver-completed sleep diaries, representing the total amount of sleep the participant obtained during the sleep period, expressed in minutes.
Higher values indicate longer sleep duration.
|
Week 0, Week 13, Week 26, Week 39, Week 52
|
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Change from Baseline in Average Duration of Nocturnal Awakenings Reported in Sleep Diaries at 12 Months
時間枠：Week 0, Week 13, Week 26, Week 39, Week 52
|
Duration of nocturnal awakenings as reported in caregiver-completed sleep diaries, representing the average length of time the participant spent awake during the sleep period, expressed in minutes.
Lower values indicate shorter awakening durations.
|
Week 0, Week 13, Week 26, Week 39, Week 52
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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。
捜査官
- 主任研究者：Christopher J Keary, MD、Massachusetts General Hospital
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 (推定)
2026年8月1日
一次修了 (推定)
2028年9月1日
研究の完了 (推定)
2029年9月1日
試験登録日
最初に提出
2026年2月11日
QC基準を満たした最初の提出物
2026年2月11日
最初の投稿 (実際)
2026年2月18日
学習記録の更新
投稿された最後の更新 (実際)
2026年8月21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19日
最終確認日
2026年8月1日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- 2025P003327
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
IPD プランの説明
現時点では、参加者のプライバシーに関する考慮事項、研究対象集団の規模と脆弱性を考慮した再識別のリスク、および現在外部データ共有を許可していない同意書とプロトコルの制限により、外部にIPDを共有する計画はありません。
機関の方針では、あらゆるデータ共有活動に対して特定のデータ使用契約（DUA）が必要であり、この研究は機密保持証明書によって保護されており、識別可能な情報の開示がさらに制限されています。
研究チームは、結果の公表と堅牢なガバナンスおよび承認メカニズムの実施を条件として、将来、非識別化データの共有を検討する可能性があります。
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
いいえ
米国FDA規制機器製品の研究
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。