Trial of Sertraline to Treat Children With Fragile X Syndrome
Condition: Fragile X Syndrome
MeSH terms: Fragile X Syndrome; Syndrome
Study type: Interventional
Phase: Phase 2

Summary: 
      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.
    

Detailed description: 
      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.
    

Intervention name(s): Sertraline; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): Liquid sertraline (20 mg/mL) will be dosed in an age-dependent manner. Participants aged 2-3 years of age will be given 2.5 mg (0.125 mL) of liquid sertraline once per day for a period of six months. Participants aged 4 years to 5 years 8 months will be given 5 mg (0.25 mL) of liquid sertraline once per day for a period of six months.; The placebo will be dosed in an age-dependent manner. Participants aged 2-3 years of age will be given 2.5 mg (0.125 mL) of liquid placebo once per day for a period of six months. Participants aged 4 years to 5 years 8 months will be given 5 mg (0.25 mL) of liquid placebo once per day for a period of six months.
Other intervention name(s): Zoloft
Intervention MeSH terms: Sertraline
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment
Keywords: Fragile X Syndrome; Pediatrics; sertraline; autism