A Controlled Trial of Sertraline in Young Children With Fragile X Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 57
- 试验地点
- 1
- 主要终点
- Change in Mullen Scales of Early Learning - Expressive Language Raw Score
研究概览
简要总结
This study is a control trial of sertraline (Zoloft) in fragile X syndrome children aged 2 years to 5 years 8 months old. The trial is six months long, and each participant will receive a series of tests at both the beginning and end of the study. The researchers hope to show improvements in language and a decrease in autistic symptoms.
详细描述
This is a single center study and the UC Davis MIND Institute for fragile X syndrome (FXS) patients aged between 2 years and 5 years, 8 months old funded by the Health Resources and Services Administration (HRSA). It is a double-blind control trial of sertraline (Zoloft), an anti-depressant typically used in the treatment of depression, obsessive-compulsive disorder, panic disorder, and other conditions. The researchers are investigating the use of this selective serotonin reuptake inhibitor (SSRI) in FXS because a retrospective study has shown significant improvements in language and decreases in autistic behavior. There is also emerging evidence regarding the stimulation of brain-derived neurotrophic factor (BDNF) and the stimulation of neurogenesis when an SSRI is given early on in the development of animal models of Down syndrome. The researchers hope to see improvements in language stimulation, social gaze and social reciprocity, spatial attention, and a decrease in autistic behaviors.
The aim of this study is to carry out a double-blind placebo controlled trial of sertraline in children with FXS who are between the ages of 2 years and 5 years, 8 months old. At baseline, the researchers will assess behavioral and cognitive development. These children will be treated for six months with either sertraline or placebo. At the end of the six months, the researchers assess the same behavioral and cognitive measures as at the beginning of the study. The researchers will also assess the side effects of the sertraline treatment throughout the study.
On March 14, 2017 two secondary outcomes for each of the primary outcomes were added for clarification of measurements at baseline visit and at six-month visit. In April 2017 additional updates were made to report primary and secondary outcome results. By June 2, 2017 all outstanding secondary outcome results had been added.
In July 2017, additional updates to the outcome measures and limitations sections were made to address PRS review comments. In the process of responding to these comments, a duplicate reported outcome measure was identified and deleted.
In August 2017, additional updates to the outcome measures were made to address PRS review comments issued in response to the July 2017 updates.
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
- 24 Months 至 68 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Fragile X Syndrome
排除标准
- •Current or past SSRI treatment
- •Current or past MAOI (monoamine oxidase inhibitor ) treatment
- •Serious co-morbid medical disorder affecting brain function and behavior (not including fragile X syndrome).
- •Uncontrolled seizure disorder or epilepsy
- •Bipolar disorder
- •Latex allergies
研究组 & 干预措施
Placebo
This arm will undergo identical treatment and assessments as the experimental group, with the exception of the active agent, sertraline. This group will be placed on a placebo.
干预措施: Placebo (Drug)
Active
This arm will undergo identical treatment and assessments as the placebo group. This group will receive the active agent, sertraline.
干预措施: Sertraline (Drug)
结局指标
主要结局
Change in Mullen Scales of Early Learning - Expressive Language Raw Score
时间窗: From baseline visit to six-month visit.
The Mullen Scales of Early Learning (MSEL) is a cognitive test to measure cognitive ability and language development. The test has five scales: gross motor, visual reception, fine motor, receptive language, and expressive language. Shown here are the baseline and 6-month follow-up raw scores from the expressive language scale. This scale's raw scores range from 0 to 50. The lower the score on this scale, the weaker the ability; the higher the score, the greater the ability. The MSEL was administered at the baseline visit and at the 6-month follow-up visit.
Clinical Global Impression - Improvement
时间窗: 6-month follow-up visit score
The Clinical Global Impression - Improvement (CGI-I) is used to measure the overall behavioral change of an individual and their therapeutic response. The CGI-I is a 3-item observer-rated scale administered by the physician to the caregiver, who assesses improvement using a 7-point scale: 1 = Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = No change; 5 = Minimally worse; 6 = Much worse; and 7 = Very much worse. Therefore, the lower the score, the greater the behavioral improvement as rated by the caregiver. Shown here are the CGI-I mean scores from the 6-month follow-up visit.
Change in Mullen Scales of Early Learning - Expressive Language Standard T Score
时间窗: From baseline visit to six-month visit
The Mullen Scales of Early Learning (MSEL) is a cognitive test to measure cognitive ability and language development. The test has five scales: gross motor, visual reception, fine motor, receptive language and expressive language. Based on the raw score obtained by the participant in each scale the scoring software computes T scores, percentile ranks, and age equivalents for each scale separately. Shown here are the baseline and 6-month follow-up T scores from the expressive language scale. T scores have a range of 20 to 80, a mean of 50, and a standard deviation of 10. Any child scoring at or below 1.5 standard deviations below the average is considered presenting significant delays. The lower the T score, the worse the outcome. The MSEL was administered at the baseline visit and at the 6-month follow-up visit.
次要结局
- Autism Diagnostic Observation Schedule(At baseline visit)
- Visual Analog Scale(At baseline visit)
- Eye Tracking(At six-month visit)
- Preschool Language Scale-fifth Edition (PLS-5): AC+EC Total Raw Score(At six-month visit)
- Sensory Processing Measure - Preschool (SPM-P) Social Participation: Raw Score(At baseline visit)
- Sensory Profile - Sensation Seeking Subscale Raw Score(At six-month visit)
- Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) - Adaptive Behavior Composite Standard Score(At baseline visit)
- Mullen Scales of Early Learning - Visual Reception Raw Score(At six-month visit)
- Mullen Scales of Early Learning - Fine Motor Raw Score(At six-month visit)
- The Autism Diagnostic Observation Schedule (ADOS-2)(At six month visit)
- The Visual Analog Scale(At six-month visit)
- Sensory Processing Measure-Preschool (SPM-P) Social Participation: Raw Score(At six-month visit)
- Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Adaptive Behavior Composite Standard Score(At six-month visit)
- Mullen Scales of Early Learning - Visual Reception Age-equivalent Score(At six-month visit)
- Mullen Scales of Early Learning - Fine Motor Age-equivalent Score(At six-month visit)
- Mullen Scales of Early Learning - Cognitive T Score Sum(At six-month visit)
- Mullen Scales of Early Learning - Summary Age-equivalent Score(At six-month visit)
研究者
Randi J. Hagerman, MD
Principle Investigator
University of California, Davis