Trial of Sertraline to Treat Children With Fragile X Syndrome

Randi J. Hagerman, MD

Status and phase: Completed; Phase 2

Conditions: Fragile X Syndrome

Treatments: Drug: Sertraline; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT01474746

271070

R40MC22641 (Other Grant/Funding Number)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 57 patients

Sex: All

Ages: 24 to 68 months old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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57 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo

Active

Experimental group

Description:

This arm will undergo identical treatment and assessments as the placebo group. This group will receive the active agent, sertraline.

Treatment:

Drug: Sertraline