跳至主要内容
MedPath Trial logoMedPath TrialMedPath Trial Home
临床试验/NCT07417137
NCT07417137招募中不适用

A Longitudinal Natural History Study of Adults and Children With Angelman Syndrome

Massachusetts General Hospital1 个研究点 分布在 1 个国家目标入组 40 人开始时间: 2026年8月1日最近更新:
Share to TwitterShare to FacebookShare to LinkedInShare to Reddit
适应症

试验速览

阶段
不适用
状态
招募中
入组人数
40
试验地点
1
主要终点
Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Cognitive Growth Score Equivalent at 12 Months
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

The goal of this observational study is to learn about the natural progression of Angelman syndrome (AS) in children and adults with a confirmed genetic diagnosis of AS. The main questions it aims to answer are:

  • How do developmental skills, such as communication, motor abilities, and adaptive behaviors, change over a 1-year period in people with AS?
  • Are there specific patterns in brain activity or sleep that are associated with changes in AS symptoms over time?

Participants will:

  • Attend 3 in-person and 2 virtual study visits over 1 year for assessments.
  • Complete tests and questionnaires about development, behaviors, and sleep with the help of their caregivers.
  • Undergo electroencephalograms (EEGs) to measure brain activity and wear a sleep-monitoring device at home (to collect actigraphy data).

详细描述

  • Longitudinal, natural history study of 40 individuals with AS, enriched for a child and adolescent population.
  • Seeks to establish normative data to inform future design and interpretation of clinical trials for AS and to support the development of more precise diagnostic tools and targeted therapeutic interventions for AS.
  • Track adaptive behavior, communication, challenging behaviors, general health, overall disease severity, and common co-occurring conditions (including epilepsy and sleep disturbances) through standardized assessments.
  • Biomarker data collected through EEGs and actigraphy recordings will be analyzed to assess age-related changes in clinical outcomes and neurophysiological measures.
  • Participants will attend up to 5 study visits over the course of 12 months: Week 0, Week 13, Week 26, Week 39, Week 52. Weeks 0, 26, 52 will be in-person. Weeks 13 and 39 will be remote.

研究设计

研究类型
Observational
观察模型
Cohort
时间视角
Prospective

入排标准

年龄范围
1 Year 至 —（Child, Adult, Older Adult）
性别
All
接受健康志愿者
否

入选标准

  • •The participant has a primary clinical diagnosis of Angelman syndrome with documented genetic variation(s) affecting the function of the UBE3A gene within the human 15q11.2-q13.3 locus. Co-occurring conditions (e.g., autism spectrum disorder, cerebral palsy, intellectual disability) are permitted; however, Angelman syndrome must be the primary clinical diagnosis.
  • •The participant is male or female (assigned sex at birth) and aged ≥1 year at the initial study visit.
  • •The participant has a study partner who meets the study partner criteria below.
  • •The participant, if unable to provide informed consent, has an appropriate surrogate who is at least 18 years of age and willing and able to provide informed consent on behalf of the participant in accordance with current International Council for Harmonisation (ICH) guidelines and applicable institutional regulations.
  • •Individuals must satisfy the following criteria to be enrolled as study partners:
  • •The study partner is a parent or primary caregiver who is at least 18 years of age.
  • •The study partner has consistent contact with the participant and, in the opinion of the investigator, is sufficiently knowledgeable about the participant's ongoing condition to provide accurate and current information.
  • •The study partner has sufficient English-language proficiency to complete study partner assessments.
  • •The study partner is willing and able to provide informed consent on their own behalf in accordance with ICH guidelines and applicable institutional regulations.
  • •The study partner is, in the opinion of the investigator, reliable and competent; willing and able to accompany the participant to all study visits and comply with study procedures; reachable by telephone or email as needed; and sufficiently knowledgeable about the participant's ongoing condition(s) to provide accurate and current information regarding the participant's health and well-being.

排除标准

  • •The participant has at least one additional known genetic abnormality outside the human 15q11.2-q13.3 locus causing a probable or known developmental disability.
  • •At least one standard-of-care treatment (medication or adjunctive therapy) used by the participant was changed during the 28 days (4 weeks) prior to the first study visit. Treatments include, but are not limited to, doses of anti-epileptic medications, behavioral management medications, sleep medications, gabapentin, cannabidiol, special diets, supplements, speech therapy, occupational therapy, applied behavioral analysis (ABA), psychosocial interventions, physical therapy, or nutritional support.
  • •The participant has unstable epilepsy, defined as having an emergency department visit or hospitalization for seizure-related concerns within the 28 days (4 weeks) preceding the initial study visit.
  • •The participant is of childbearing potential and is either pregnant, breastfeeding, or not using an adequate method of contraception; abstinence is acceptable.
  • •The participant has a clinically relevant history of malignancy; clinically significant abnormal test results; clinically significant cardiovascular, hematologic, hepatic, muscular, neurologic, or renal disease; or has experienced other clinical events which, in the opinion of the investigator, render participation unsuitable.
  • •The participant has a lifetime history of treatment with any cell- or gene-based therapy, including antisense oligonucleotides or gene-editing therapies.
  • •The participant has received any investigational therapy other than a cell- or gene-based therapy within 28 days or 5 half-lives (whichever is longer) preceding the initial study visit.
  • •The participant is currently enrolled or plans to enroll in an interventional study involving an investigational agent or device during the planned observation period.
  • •The participant has a known contraindication to electroencephalography, actigraphy, or any other study procedure described in the schedule of assessments.
  • •The participant or study partner is, in the opinion of the investigator, unsuitable for participation in any other way, including an inability to fulfill study requirements.

研究组 & 干预措施

Participants aged 1-17 years (inclusive)

  • Cohort 1: Participants aged at least 1 year old and less than 18 years old at the time of enrollment with genetically confirmed AS.
  • This cohort serves as the broad group for assessing the natural history of AS across childhood and adolescence.

Participants aged 18 years and older

  • Cohort 3: Participants aged at least 18 years old.
  • This cohort is limited to up to 5 participants to explore the natural history of AS in adults, though it is not the primary focus of this study.

Participants aged 1-12 years (inclusive)

  • Cohort 2: Participants aged at least 1 year old and younger than 13 years old at the time of enrollment with genetically confirmed AS.
  • A subset of Cohort 1, specifically focused on preadolescents.
  • This cohort aims to capture developmental milestones and changes during a critical period of growth where potential therapeutic interventions may have the most significant impact.
  • Participants aged 1-12 years will first be assigned to Cohort 2 until its capacity is reached. Once Cohort 2 is full, participants aged 1-12 years will be enrolled in Cohort 1 only.

结局指标

主要结局

Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Cognitive Growth Score Equivalent at 12 Months

时间窗: 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.

Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Receptive Communication Growth Score Equivalent at 12 Months

时间窗: 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.

Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Expressive Communication Growth Score Equivalent at 12 Months

时间窗: 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.

Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Fine Motor Growth Score Equivalent at 12 Months

时间窗: 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.

Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Gross Motor Growth Score Equivalent at 12 Months

时间窗: 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.

Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Cognitive Growth Score Equivalent at 12 Months

时间窗: Baseline, 3 months, 6 months, 9 months, and 12 months

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.

Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Receptive Communication Growth Score Equivalent at 12 Months

时间窗: Baseline, 3 months, 6 months, 9 months, and 12 months

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.

Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Expressive Communication Growth Score Equivalent at 12 Months

时间窗: Baseline, 3 months, 6 months, 9 months, and 12 months

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.

Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Fine Motor Growth Score Equivalent at 12 Months

时间窗: Baseline, 3 months, 6 months, 9 months, and 12 months

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.

Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Gross Motor Growth Score Equivalent at 12 Months

时间窗: Baseline, 3 months, 6 months, 9 months, and 12 months

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.

次要结局

  • Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Cognitive Age-Equivalent Score at 12 Months(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(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(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(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(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(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(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(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(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(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(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(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(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(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(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(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(Week 0, Week 13, Week 26, Week 39, Week 52)
  • Change from Baseline in Average Effective Sleep Measured by Actigraphy at 12 Months(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(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(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(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(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(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(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(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(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(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(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(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(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(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(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(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(Week 0, Week 13, Week 26, Week 39, Week 52)
  • Change from Baseline in Average Sleep Efficiency Measured by Actigraphy at 12 Months(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(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(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(Week 0, Week 13, Week 26, Week 39, Week 52)
  • Change from Baseline in Frequency of Reported Seizure Events at 12 Months(Week 0, Week 13, Week 26, Week 39, Week 52)
  • Change from Baseline in Average Total Sleep Time Measured by Actigraphy at 12 Months(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(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(Week 0, Week 13, Week 26, Week 39, Week 52)
  • Change from Baseline in Average Sleep Onset Latency Measured by Actigraphy at 12 Months(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(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(Week 0, Week 13, Week 26, Week 39, Week 52)
  • Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Cognitive Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Expressive Communication Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Receptive Communication Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Fine Motor Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Bayley Scale of Infant Development, Fourth Edition (Bayley-4) Gross Motor Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Symptoms of Angelman Syndrome-Clinical Global Impressions Scale of Severity (SAS-CGI-S) Global Severity Score at 12 Months(Baseline, 3 months, 6 months, 9 months, 12 months)
  • Change from Baseline in Aberrant Behavior Checklist-Community (ABC-C) Hyperactivity/Noncompliance Subscale Score (ABC-H) at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Aberrant Behavior Checklist-Community (ABC-C) Irritability Subscale Score (ABC-I) at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Caregiver-Reported Angelman Syndrome Scale for Impact (CASS-I) Overall AS Symptoms Rating at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) Total Sleep Disturbance Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) Sleep Duration Subscale Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ) Night Wakings Subscale Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Observer-Reported Communication Ability (ORCA) Overall Communication T-Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Observer-Reported Communication Ability (ORCA) Expressive Communication T-Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Observer-Reported Communication Ability (ORCA) Receptive Communication T-Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Observer-Reported Communication Ability (ORCA) Pragmatic Communication T-Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Receptive Communication Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Receptive Communication Growth Score Equivalent at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Expressive Communication Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Expressive Communication Growth Score Equivalent at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Personal Daily Living Skills Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Personal Daily Living Skills Growth Score Equivalent at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Domestic Daily Living Skills Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Domestic Daily Living Skills Growth Score Equivalent at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Community Daily Living Skills Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Community Daily Living Skills Growth Score Equivalent at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Interpersonal Relationships Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Interpersonal Relationships Growth Score Equivalent at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Play and Leisure Time Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Play and Leisure Time Growth Score Equivalent at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Coping Skills Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Coping Skills Growth Score Equivalent at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Gross Motor Skills Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Gross Motor Skills Growth Score Equivalent at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Fine Motor Skills Age-Equivalent Score at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Fine Motor Skills Growth Score Equivalent at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Frequency of Reported Seizure Events at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Average Total Sleep Time Measured by Actigraphy at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Average Effective Sleep Measured by Actigraphy at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Average Sleep Efficiency Measured by Actigraphy at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Average Number of Nocturnal Awakenings Measured by Actigraphy at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Average Duration of Nocturnal Awakenings Measured by Actigraphy at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Average Sleep Onset Latency Measured by Actigraphy at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Average Total Sleep Time Reported in Sleep Diaries at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)
  • Change from Baseline in Average Duration of Nocturnal Awakenings Reported in Sleep Diaries at 12 Months(Baseline, 3 months, 6 months, 9 months, and 12 months)

研究者

申办方类型
Other
责任方
Principal Investigator
主要研究者

Christopher Keary

Behavioral Director (Angelman Syndrome Clinic) & Staff Psychiatrist (Lurie Center for Autism)

Massachusetts General Hospital

研究点 (1)

Loading locations...

相似试验

招募中
不适用
A Study in Children With AchondroplasiaAchondroplasia
NCT07301463Abbisko Therapeutics Co, Ltd260
招募中
不适用
Evolution of Lesions in Repeated Biparametric Prostate Magnetic Resonance Imaging
NCT07369570University of Eastern Finland100
尚未招募
不适用
Lung Cancer Screening Implementation Project in the Brazilian Public Health Care System (LUSIP)Lung Cancer
NCT07279948AstraZeneca477
招募中
不适用
geniculaR Artery eMBolization for Knee Osteoarthritis (RAMBO) Registry
NCT07380906Joint & Vascular Institute1,200
招募中
不适用
Real-world Outcomes of Peripheral T-cell Lymphoma: A Multicenter Retrospective and Prospective Cohort Study
NCT07270861Fudan University3,000