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안젤만 증후군의 자연사 연구 (GLOW-AS)

2026년 8월 19일 업데이트: Christopher Keary, Massachusetts General Hospital

엔젤만 증후군을 가진 성인과 어린이에 대한 종단적 자연 경과 연구

본 관찰 연구의 목적은 안젤만 증후군(AS)의 유전적 진단이 확인된 아동 및 성인에서 안젤만 증후군의 자연적 진행 과정에 대해 알아보는 것입니다. 이 연구가 답하고자 하는 주요 질문은 다음과 같습니다:

  • AS 환자에서 의사소통, 운동 능력, 적응 행동과 같은 발달 기술이 1년 동안 어떻게 변화하는가?
  • 시간이 지남에 따라 AS 증상 변화와 관련된 뇌 활동이나 수면의 특정 패턴이 있는가?

참가자는 다음과 같은 절차를 수행합니다:

  • 1년 동안 5회(약 3개월마다) 연구 현장을 방문하여 평가를 받습니다.
  • 보호자의 도움을 받아 발달, 행동, 수면에 관한 검사와 설문지를 작성합니다.
  • 뇌 활동을 측정하기 위해 뇌파검사(EEG)를 받고, 가정에서 수면 모니터링 장치를 착용하여(활동기록 데이터 수집) 활동기록 데이터를 수집합니다.

연구 개요

상태

모병

정황

상세 설명

  • 청소년 인구를 중심으로 한 40명의 AS 환자를 대상으로 한 종단적 자연사 연구입니다.
  • 향후 AS 임상 시험의 설계 및 해석을 위한 기준 데이터를 확립하고, AS에 대한 보다 정밀한 진단 도구 및 표적 치료 중재 개발을 지원하는 것을 목표로 합니다.
  • 표준화된 평가를 통해 적응 행동, 의사소통, 문제 행동, 일반 건강 상태, 전반적인 질병 중증도 및 일반적인 동반 질환(간질 및 수면 장애 포함)을 추적합니다.
  • EEG 및 활동 기록을 통해 수집된 바이오마커 데이터를 분석하여 임상 결과 및 신경생리학적 측정치의 연령 관련 변화를 평가합니다.
  • 참가자는 12개월 동안 최대 5회의 대면 연구 방문에 참석합니다: 0주차, 13주차, 26주차, 39주차, 52주차.

연구 유형

관찰

등록 (추정된)

40

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

연구 연락처 백업

연구 장소

  • 미국
    • Massachusetts
      • Lexington, Massachusetts, 미국, 02421
        • 모병
        • MGH Lurie Center for Autism
        • 수석 연구원:
          • Christopher J Keary, MD
        • 부수사관:
          • Ronald L Thibert, DO, MSPH
        • 연락하다:
        • 연락하다:

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

  • 어린이
  • 성인
  • 고령자

건강한 자원 봉사자를 받아들입니다

아니

샘플링 방법

비확률 샘플

연구 인구

유전적으로 확인된 AS 진단을 받은 최소 1세 이상의 개인.

설명

포함 기준:

  • 참가자는 인간 15q11.2-q13.3 유전자좌 내 UBE3A 유전자 기능에 영향을 미치는 문서화된 유전적 변이를 가진 앤젤만 증후군의 주요 임상 진단을 받았습니다. 동반 질환(예: 자폐 스펙트럼 장애, 뇌성마비, 지적 장애)은 허용되나, 앤젤만 증후군이 주요 임상 진단이어야 합니다.
  • 참가자는 남성 또는 여성(출생 시 지정 성별)이며 초기 연구 방문 시 만 1세 이상입니다.
  • 참가자는 아래 연구 파트너 기준을 충족하는 연구 파트너가 있습니다.
  • 참가자가 동의를 제공할 수 없는 경우, 현행 국제조화의회(ICH) 지침 및 적용 가능한 기관 규정에 따라 참가자를 대신하여 동의를 제공할 의사와 능력이 있는 만 18세 이상의 적절한 대리인이 있어야 합니다.

연구 파트너로 등록하려면 개인이 다음 기준을 충족해야 합니다:

  • 연구 파트너는 만 18세 이상의 부모 또는 주 보호자입니다.
  • 연구 파트너는 참가자와 지속적으로 접촉하며, 연구자의 의견으로 참가자의 현재 상태에 대해 충분히 알고 있어 정확하고 최신 정보를 제공할 수 있습니다.
  • 연구 파트너는 연구 파트너 평가를 완료할 수 있는 충분한 영어 능력을 보유합니다.
  • 연구 파트너는 ICH 지침 및 적용 가능한 기관 규정에 따라 자신을 대신하여 동의를 제공할 의사와 능력이 있습니다.
  • 연구 파트너는 연구자의 의견으로 신뢰할 수 있고 유능하며; 모든 연구 방문에 참가자를 동반하고 연구 절차를 준수할 의사와 능력이 있고; 필요 시 전화나 이메일로 연락 가능하며; 참가자의 현재 상태에 대해 충분히 알고 있어 참가자의 건강과 복지에 관한 정확하고 최신 정보를 제공할 수 있습니다.

제외 기준:

  • 참가자는 인간 15q11.2-q13.3 유전자좌 외부에 발달 장애를 유발할 가능성이 있거나 알려진 추가 유전적 이상을 적어도 하나 가지고 있습니다.
  • 참가자가 사용하는 적어도 하나의 표준 치료(약물 또는 보조 치료)가 첫 연구 방문 전 28일(4주) 동안 변경되었습니다. 치료에는 항전간제 용량, 행동 관리 약물, 수면제, 가바펜틴, 칸나비디올, 특별 식이, 보충제, 언어 치료, 작업 치료, 적용 행동 분석(ABA), 심리사회적 중재, 물리 치료 또는 영양 지원이 포함되나 이에 국한되지 않습니다.
  • 참가자는 초기 연구 방문 전 28일(4주) 이내에 발작 관련 문제로 응급실 방문이나 입원 경험이 있는 불안정한 간질을 가지고 있습니다.
  • 참가자가 임신 가능한 경우 임신 중이거나 수유 중이거나 적절한 피임법을 사용하지 않습니다; 금욕은 허용됩니다.
  • 참가자는 임상적으로 관련된 악성 종양 병력; 임상적으로 유의한 비정상 검사 결과; 임상적으로 유의한 심혈관, 혈액학적, 간, 근육, 신경학적 또는 신장 질환; 또는 연구자의 의견으로 참여에 부적합하게 만드는 다른 임상적 사건을 경험했습니다.
  • 참가자는 항의 올리고뉴클레오타이드 또는 유전자 편집 치료를 포함한 세포 또는 유전자 기반 치료를 평생 동안 받은 적이 있습니다.
  • 참가자는 초기 연구 방문 전 28일 또는 5 반감기(더 긴 쪽) 이내에 세포 또는 유전자 기반 치료 이외의 연구 치료를 받았습니다.
  • 참가자는 계획된 관찰 기간 동안 연구 약제나 장치를 포함하는 중재 연구에 현재 등록되어 있거나 등록할 계획입니다.
  • 참가자는 뇌파 검사, 활동 기록 검사 또는 평가 일정에 설명된 다른 연구 절차에 대한 알려진 금기 사항이 있습니다.
  • 참가자나 연구 파트너는 연구자의 의견으로 다른 어떤 방식으로든 참여에 부적합하며, 이는 연구 요구 사항을 충족할 수 없는 경우를 포함합니다.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

코호트 및 개입

그룹/코호트
만 1세부터 17세(포함)까지의 참가자
  • 코호트 1: 등록 시 연령이 최소 1세 이상 18세 미만이며 유전적으로 확인된 AS를 가진 참가자.
  • 이 코호트는 유년기와 청소년기에 걸쳐 AS의 자연 경과를 평가하기 위한 광범위한 그룹 역할을 합니다.
만 1세~12세(포함) 참가자
  • 코호트 2: 등록 시 1세 이상 13세 미만이며 유전적으로 확인된 AS를 가진 참가자.
  • 코호트 1의 하위 집단으로, 특히 사춘기 이전 아동에 초점을 맞춤.
  • 이 코호트는 성장의 중요한 시기 동안 발달적 이정표와 변화를 포착하여 잠재적 치료적 개입이 가장 큰 영향을 미칠 수 있는 시기를 목표로 함.
  • 1-12세 참가자는 먼저 코호트 2에 배정되며, 코호트 2의 정원이 차면 이후 1-12세 참가자는 코호트 1에만 등록됨.
18세 이상의 참가자
  • 코호트 3: 참가자 연령은 최소 18세 이상입니다.
  • 이 코호트는 성인의 AS 자연 경과를 탐구하기 위해 최대 5명의 참가자로 제한되지만, 본 연구의 주요 초점은 아닙니다.

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Cognitive Growth Score Equivalent at 12 Months
기간: Week 0, Week 26, Week 52
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.
Week 0, Week 26, Week 52
Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Receptive Communication Growth Score Equivalent at 12 Months
기간: Week 0, Week 26, Week 52
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.
Week 0, Week 26, Week 52
Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Expressive Communication Growth Score Equivalent at 12 Months
기간: Week 0, Week 26, Week 52
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.
Week 0, Week 26, Week 52
Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Fine Motor Growth Score Equivalent at 12 Months
기간: Week 0, Week 26, Week 52
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.
Week 0, Week 26, Week 52
Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Gross Motor Growth Score Equivalent at 12 Months
기간: Week 0, Week 26, Week 52
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.
Week 0, Week 26, Week 52

2차 결과 측정

결과 측정
측정값 설명
기간
Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Cognitive Age-Equivalent Score at 12 Months
기간: Week 0, Week 26, Week 52
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.
Week 0, Week 26, Week 52
Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Expressive Communication Age-Equivalent Score at 12 Months
기간: Week 0, Week 26, Week 52
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.
Week 0, Week 26, Week 52
Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Receptive Communication Age-Equivalent Score at 12 Months
기간: Week 0, Week 26, Week 52
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.
Week 0, Week 26, Week 52
Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Fine Motor Age-Equivalent Score at 12 Months
기간: Week 0, Week 26, Week 52
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.
Week 0, Week 26, Week 52
Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Gross Motor Age-Equivalent Score at 12 Months
기간: Week 0, Week 26, Week 52
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.
Week 0, Week 26, Week 52
Change from Baseline in Symptoms of Angelman Syndrome-Clinical Global Impressions Scale of Severity (SAS-CGI-S) Global Severity Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Aberrant Behavior Checklist-Community (ABC-C) Hyperactivity/Noncompliance Subscale Score (ABC-H) at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Aberrant Behavior Checklist-Community (ABC-C) Irritability Subscale Score (ABC-I) at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Caregiver-Reported Angelman Syndrome Scale for Impact (CASS-I) Overall AS Symptoms Rating at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) Total Sleep Disturbance Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) Sleep Duration Subscale Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
The CSHQ Sleep Duration subscale assesses the adequacy of sleep duration. Higher scores indicate shorter or insufficient sleep duration.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) Night Wakings Subscale Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
The CSHQ Night Wakings subscale assesses the frequency and severity of nighttime awakenings. Higher scores indicate more frequent or problematic night wakings.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Observer-Reported Communication Ability (ORCA) Overall Communication T-Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Observer-Reported Communication Ability (ORCA) Expressive Communication T-Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Observer-Reported Communication Ability (ORCA) Receptive Communication T-Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Observer-Reported Communication Ability (ORCA) Pragmatic Communication T-Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Receptive Communication Age-Equivalent Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Receptive Communication Growth Score Equivalent at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Expressive Communication Age-Equivalent Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Expressive Communication Growth Score Equivalent at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Personal Daily Living Skills Age-Equivalent Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Personal Daily Living Skills Growth Score Equivalent at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Domestic Daily Living Skills Age-Equivalent Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Domestic Daily Living Skills Growth Score Equivalent at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Community Daily Living Skills Age-Equivalent Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Community Daily Living Skills Growth Score Equivalent at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Interpersonal Relationships Age-Equivalent Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Interpersonal Relationships Growth Score Equivalent at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Play and Leisure Time Age-Equivalent Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Play and Leisure Time Growth Score Equivalent at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Coping Skills Age-Equivalent Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Coping Skills Growth Score Equivalent at 12 Months
기간: 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
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Gross Motor Skills Age-Equivalent Score at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Gross Motor Skills Growth Score Equivalent at 12 Months
기간: 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
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Fine Motor Skills Age-Equivalent Score at 12 Months
기간: 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
Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Fine Motor Skills Growth Score Equivalent at 12 Months
기간: 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
Change from Baseline in Frequency of Reported Seizure Events at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Average Total Sleep Time Measured by Actigraphy at 12 Months
기간: 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.
Week 0, Week 13, Week 26, Week 39, Week 52
Change from Baseline in Average Effective Sleep Measured by Actigraphy at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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
Change from Baseline in Average Sleep Efficiency Measured by Actigraphy at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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
Change from Baseline in Average Number of Nocturnal Awakenings Measured by Actigraphy at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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
Change from Baseline in Average Duration of Nocturnal Awakenings Measured by Actigraphy at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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
Change from Baseline in Average Sleep Onset Latency Measured by Actigraphy at 12 Months
기간: Week 0, Week 13, Week 26, Week 39, Week 52
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
Change from Baseline in Average Total Sleep Time Reported in Sleep Diaries at 12 Months
기간: 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
Change from Baseline in Average Duration of Nocturnal Awakenings Reported in Sleep Diaries at 12 Months
기간: 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

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Massachusetts General Hospital

협력자

Astellas Pharma Global Development, Inc.

수사관

  • 수석 연구원: Christopher J Keary, MD, Massachusetts General Hospital

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (추정된)

2026년 8월 1일

기본 완료 (추정된)

2028년 9월 1일

연구 완료 (추정된)

2029년 9월 1일

연구 등록 날짜

최초 제출

2026년 2월 11일

QC 기준을 충족하는 최초 제출

2026년 2월 11일

처음 게시됨 (실제)

2026년 2월 18일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 8월 21일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 8월 19일

마지막으로 확인됨

2026년 8월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • 2025P003327

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

아니요

IPD 계획 설명

참가자 개인정보 보호, 연구 대상 집단의 규모와 취약성으로 인한 재식별 위험, 그리고 현재 외부 데이터 공유를 허용하지 않는 동의서 자료와 연구 계획서의 제한 사항 등을 고려하여, 현재로서는 IPD(개별 참가자 데이터)를 외부와 공유할 계획이 없습니다. 기관 정책에 따르면 모든 데이터 공유 활동에는 특정 데이터 사용 계약(DUA)이 필요하며, 본 연구는 기밀 유지 증명서로 보호되어 식별 가능한 정보의 공개가 더욱 제한됩니다. 연구팀은 향후 연구 결과 발표와 함께 강력한 거버넌스 및 승인 메커니즘을 마련한 후, 비식별화된 데이터 공유를 고려할 수 있습니다.

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

아니

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

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