Androgen Effect on Motor/Cognitive Outcome in Klinefelter Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 93
- 试验地点
- 1
- 主要终点
- Motor Function/Strength
研究概览
简要总结
The purpose of this study is to evaluate the effects of low-dose androgen on the motor and cognitive development of boys with Klinefelter syndrome.
详细描述
Klinefelter syndrome (KS), a genetic disorder that affects males only, is characterized by having an extra X chromosome. The phenotype - or physical and learning features - includes testicular failure, tall stature, and specific cognitive and behavioral attributes such as diminished motor function, language-based learning difficulties, poor self-image, and shyness. The KS phenotype may be the result of androgen deficiency in utero, infancy, and childhood. For individuals with KS, androgen replacement is standard treatment in adolescence and adulthood but has not been used earlier in childhood or included in the standard medical care of KS children ages 4 to 12.
The purpose of this study is to examine the effects of androgen on learning and development in boys with KS. Researchers also want to determine if low-dose androgen replacement at an early age will improve some of the learning difficulties associated with the disorder. The overall goal of this study is to address questions regarding the relationship of early androgen deficiency to learning and motor function.
Participants in the study will be randomized to one of two treatment groups, receiving either oxandrolone (low-dose androgen) or placebo, for two years. All participants will be evaluated for safety at the beginning of the study and at 3, 6, 12, 18, and 24 months. Also at the beginning of the study and every 3 to 6 months thereafter (for a total of 6 visits), the researchers will perform a careful history and physical examination and a bone age X-ray, and obtain a blood sample.
Participation in the trial will last two years and includes 6 clinic visits.
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
- 4 Years 至 12 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Karyotype diagnosis of Klinefelter syndrome
- •Chronological age of 4-12 years
- •No treatment with androgen in the past year
排除标准
- •Major liver, kidney or other systemic disease
- •Variant karyotypes including 47,XYY males
- •Evidence of spontaneous onset of puberty, defined as testicular size > 4ml
研究组 & 干预措施
Oxandrolone
Androgen oxandrolone: Oxandrolone, 0.6 > mg/kg/day, orally, for 2 years.
干预措施: androgen oxandrolone (Drug)
Placebo
An inactive substance.
干预措施: placebo (Other)
结局指标
主要结局
Motor Function/Strength
时间窗: 2 years per subject
Outcome measures were tested using the following assessments: Bruininks-Osertesky Test of Motor Proficiency (BOT) subscales of (1) Visual Motor Control, (2) Upper limb Speed, and (3) Strength, Physical and Neurological Evaluation for Soft Signs (PANESS), and Hand Strength Dynamometer. BOT assess the child's motor development and includes standard scores (mean=100, SD=15) and subtest scores and is normed for sex and age (4-14.5 years). PANESS assesses the time required to press thumb to 4 fingers 20 times for the dominant and nondominant hands and includes standard scores (mean=100, SD=15) with age-specific norms (4-18 years). Hand strength dynamometer assess hand strength in the dominant and nondominant hands and includes standard scores (mean=100, SD=15). Data is expressed as standard scores with mean of 100 and SD of 15. The minimum standard score is 50; the maximum standard score is 145. Higher scorers imply better function.
次要结局
- Cognitive Function and Language(2 years per subject)
- Working Memory/Attention(2 years per participant)
- Psychosocial and Behavior Domain(2 years per subject)