Clinical trial NCT01474746: Trial of Sertraline to Treat Children With Fragile X Syndrome
Official title: A Controlled Trial of Sertraline in Young Children With Fragile X Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fragile X Syndrome
Keywords: Fragile X Syndrome
Pediatrics
sertraline
autism
Lead sponsor: University of California, Davis
Sponsor class: OTHER
First posted: 2011-11-18
Enrollment: 57
Sex: ALL
Minimum age (years): 24
Maximum age (years): 68
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

Eligibility criteria: Inclusion Criteria:

* Fragile X Syndrome

Exclusion Criteria:

* 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

Primary outcomes:
measure: Change in Mullen Scales of Early Learning - Expressive Language Raw Score
timeFrame: From baseline visit to six-month visit.
measure: Clinical Global Impression - Improvement
timeFrame: 6-month follow-up visit score
measure: Change in Mullen Scales of Early Learning - Expressive Language Standard T Score
timeFrame: From baseline visit to six-month visit
Secondary outcomes:
measure: Autism Diagnostic Observation Schedule
timeFrame: At baseline visit
measure: Visual Analog Scale
timeFrame: At baseline visit
measure: Eye Tracking
timeFrame: At baseline visit
measure: Preschool Language Scale-fifth Edition (PLS-5): AC+EC Total Raw Score
timeFrame: At baseline visit
measure: Sensory Processing Measure - Preschool (SPM-P) Social Participation: Raw Score
timeFrame: At baseline visit
measure: Sensory Profile - Sensation Seeking Subscale Raw Score
timeFrame: At baseline visit
measure: Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) - Adaptive Behavior Composite Standard Score
timeFrame: At baseline visit
measure: Mullen Scales of Early Learning - Visual Reception Raw Score
timeFrame: At baseline visit
measure: Mullen Scales of Early Learning - Fine Motor Raw Score
timeFrame: At baseline visit
measure: The Autism Diagnostic Observation Schedule (ADOS-2)
timeFrame: At six month visit
measure: The Visual Analog Scale
timeFrame: At six-month visit
measure: Eye Tracking
timeFrame: At six-month visit
measure: Preschool Language Scale-fifth Edition (PLS-5): AC+EC Total Raw Score
timeFrame: At six-month visit
measure: Sensory Processing Measure-Preschool (SPM-P) Social Participation: Raw Score
timeFrame: At six-month visit
measure: Sensory Profile - Sensation Seeking Subscale Raw Score
timeFrame: At six-month visit
measure: Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) Adaptive Behavior Composite Standard Score
timeFrame: At six-month visit
measure: Mullen Scales of Early Learning - Visual Reception Raw Score
timeFrame: At six-month visit
measure: Mullen Scales of Early Learning - Visual Reception Age-equivalent Score
timeFrame: At baseline visit
measure: Mullen Scales of Early Learning - Visual Reception Age-equivalent Score
timeFrame: At six-month visit
measure: Mullen Scales of Early Learning - Fine Motor Raw Score
timeFrame: At six-month visit
measure: Mullen Scales of Early Learning - Fine Motor Age-equivalent Score
timeFrame: At baseline visit
measure: Mullen Scales of Early Learning - Fine Motor Age-equivalent Score
timeFrame: At six-month visit
measure: Mullen Scales of Early Learning - Cognitive T Score Sum
timeFrame: At baseline visit
measure: Mullen Scales of Early Learning - Cognitive T Score Sum
timeFrame: At six-month visit
measure: Mullen Scales of Early Learning - Summary Age-equivalent Score
timeFrame: At baseline visit
measure: Mullen Scales of Early Learning - Summary Age-equivalent Score
timeFrame: At six-month visit
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: UC Davis M.I.N.D. Institute
state: California
Study officials:
affiliation: UC Davis MIND Institute
name: Randi J Hagerman, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: UC Davis MIND Institute
name: Kathleen Angkustsiri, MD
role: PRINCIPAL_INVESTIGATOR
References: