Psychological and Motor Effects of Testosterone Therapy in Adolescents With XXY/Klinefelter Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 48
- 试验地点
- 1
- 主要终点
- Change From Baseline on the Delis-Kaplan Executive Function System
研究概览
简要总结
The purpose of this study is to determine if testosterone replacement therapy leads to changes in psychological factors and/or motor skills in adolescent males with 47,XXY (also called Klinefelter syndrome). This study will also evaluate whether certain genetic factors of the X chromosome affect the psychological or motor features of XXY/Klinefelter syndrome.
详细描述
Klinefelter syndrome (47,XXY) is the most common chromosomal abnormality in humans and occurs in approximately 1 in 650 males. Testosterone deficiency develops during adolescence in the majority of individuals with XXY. However, there are no previous studies that evaluate the psychological and motor effects of testosterone replacement therapy in adolescents with XXY.
The study researchers are interested in learning if testosterone therapy initiated in early puberty in XXY (KS) will lead to improvements in psychological and/or motor skills. They are also interested in learning if genetic variations of the androgen receptor gene or the parent-of-origin of the extra X chromosome influence the response to testosterone therapy.
Specific psychological factors to be studied include verbal and nonverbal cognitive skills, attention, executive function (organization, problem-solving skills, inhibition), anxiety, language, self-esteem, and other behavioral factors. Motor skills to be studied include gross and fine motor skills, motor coordination, and motor planning.
Participants in the study will be randomized to one of two treatment groups, receiving either testosterone gel or placebo, for 12 months. 60% of the study group will receive testosterone, and 40% will receive placebo.
The research participants, parents/caregivers, and the members of the study team will be blinded to the treatment group, and will not know whether they are receiving testosterone gel or placebo treatments.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 8 Years 至 18 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Males with 47,XXY
- •Participants must be in early puberty (Tanner stage II-III)
- •Age 8 - 18 will be considered, but eligibility will be determined by review of laboratory results, bone age X-ray, and physical examination to determine stage of puberty
- •All racial and ethnic groups
排除标准
- •Other genetic variations of Klinefelter syndrome (48,XXXY, 48,XXYY, 49,XXXXY). The investigator has other studies for these groups and we encourage interested individuals with these disorders to contact us about other available studies.
- •47,XXY plus another genetic disorder
- •Non-English speaking individuals (because the psychological tests are administered in English)
- •Participants with a medical history of blood clotting problems, blindness, deafness, or cancer
研究组 & 干预措施
Testosterone gel 1%
Testosterone gel 1% applied to skin once per day for 1 year. Starting dose 1.25mL per day, titrated in 1.25mL increments for 1st 6 months of study after each study visit up to maximum of 5.0mL per day. Titration based on testosterone levels with target level in mid-range of normal for Tanner stage.
干预措施: Testosterone gel 1% (Drug)
Placebo gel
Placebo gel applied to skin once per day for 1 year. Starting dose 1.25mL per day. Dose randomly adjusted in 1.25mL increments for 1st 6 months of study after each study visit up to maximum of 5.0mL per day.
干预措施: Placebo gel (Drug)
结局指标
主要结局
Change From Baseline on the Delis-Kaplan Executive Function System
时间窗: Visit 1 (baseline) and Visit 5 (1 year)
A collection of subtests from the Delis-Kaplan Executive Function System will be used to evaluate different domains of executive functions including planning, problem solving, fluency, inhibition, and working memory. Possible scaled scores range from 0-20 (mean of 10), with higher scores indicating better performance. The value of the change in scaled score from baseline is reported.
Change From Baseline on the Bruininks-Oseretsky Test of Motor Development - 2
时间窗: Visit 1 (baseline) and Visit 5 (1 year)
This test evaluates many aspects of motor functioning including fine and gross motor skills, visual motor integration, motor planning, and motor coordination. Possible scores range from 20-80, with higher scores indicating a better outcome.
Change From Baseline on the Conners Parent Rating Scales: DSM Attention
时间窗: Visit 1 (baseline) and Visit 5 (1 year)
This questionnaire allows parents to rate many aspects of behavior including attention, hyperactivity, anxiety, social skills, and emotional lability. The range of possible scores is 0 to 100 (Mean T-score 50), with higher scores indicating a worse outcome.
Change From Baseline on the Conners Parent Rating Scales: DSM Hyperactivity
时间窗: Visit 1 (baseline) and Visit 5 (1 year)
This questionnaire allows parents to rate many aspects of behavior including attention, hyperactivity, anxiety, social skills, and emotional lability. The range of possible scores is 0 to 100 (Mean T-score 50), with higher scores indicating a worse outcome.
Change From Baseline on the Conners Parent Rating Scales: Anxiety
时间窗: Visit 1 (baseline) and Visit 5 (1 year)
This questionnaire allows parents to rate many aspects of behavior including attention, hyperactivity, anxiety, social skills, and emotional lability. The range of possible scores is 0 to 100 (Mean T-score 50), with higher scores indicating a worse outcome.
Change From Baseline on the Conners Parent Rating Scales: Social Skills
时间窗: Visit 1 (baseline) and Visit 5 (1 year)
This questionnaire allows parents to rate many aspects of behavior including attention, hyperactivity, anxiety, social skills, and emotional lability. The range of possible scores is 0 to 100, with higher scores indicating a worse outcome.
Change From Baseline on the Conners Parent Rating Scales: Emotional Lability
时间窗: Visit 1 (baseline) and Visit 5 (1 year)
This questionnaire allows parents to rate many aspects of behavior including attention, hyperactivity, anxiety, social skills, and emotional lability. The range of possible scores is 0 to 100, with higher scores indicating a worse outcome.
次要结局
- Change From Baseline on the Vineland Adaptive Behavior Scales - 2nd Edition(Visit 1 (baseline) and Visit 5 (1 year))
- Change From Baseline on the Wechsler Intelligence Scale for Children - Fourth Edition(Visit 1 (baseline) and Visit 5 (1 year))
- Change From Baseline on the Comprehensive Test of Phonological Processing(Visit 1 (baseline) and Visit 5 (1 year))