Trial of Sertraline to Treat Children With Fragile X Syndrome

NCT ID: NCT01474746

Last Updated: 2017-09-25

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 57 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-01-31
• Study Completion Date: 2015-09-30

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.

Conditions

Fragile X Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

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.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

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.

Active

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

Group Type: EXPERIMENTAL

Sertraline

Intervention Type: DRUG

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.

Interventions

Sertraline

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.

Intervention Type: DRUG

Placebo

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.

Intervention Type: DRUG

Other Intervention Names

Zoloft

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

• Minimum Eligible Age: 24 Months
• Maximum Eligible Age: 68 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Randi J. Hagerman, MD

OTHER

Sponsor Role: lead

Responsible Party

Randi J. Hagerman, MD

Principle Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Randi J Hagerman, MD

Role: PRINCIPAL_INVESTIGATOR

UC Davis MIND Institute

Kathleen Angkustsiri, MD

Role: PRINCIPAL_INVESTIGATOR

UC Davis MIND Institute

Locations

UC Davis M.I.N.D. Institute

Sacramento, California, United States

Countries

United States

Other Identifiers

R40MC22641

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

271070

Identifier Type: -

Identifier Source: org_study_id