Trial of Sertraline to Treat Children With Fragile X Syndrome

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Randi J. Hagerman, MD, University of California, Davis
ClinicalTrials.gov Identifier:
NCT01474746
First received: November 10, 2011
Last updated: August 12, 2015
Last verified: August 2015
  Purpose

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.


Condition Intervention Phase
Fragile X Syndrome
Drug: Sertraline
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: A Controlled Trial of Sertraline in Young Children With Fragile X Syndrome

Resource links provided by NLM:


Further study details as provided by University of California, Davis:

Primary Outcome Measures:
  • Mullen Scales of Early Learning [ Time Frame: Baseline and six months. ] [ Designated as safety issue: No ]
    The Mullen Scales of Early Learning is a cognitive test to measure cognitive ability and language development.

  • Clinical Global Impression [ Time Frame: Baseline and six months. ] [ Designated as safety issue: No ]
    The Clinical Global Impression will be used to measure the overall behavioral change of an individual.


Secondary Outcome Measures:
  • Autism Diagnostic Observation Schedule [ Time Frame: Baseline and six months. ] [ Designated as safety issue: No ]
    The Autism Diagnostic Observation Schedule assess and diagnoses autism and pervasive developmental disorder across ages, developmental levels, and language skills.

  • Visual Analog Scale [ Time Frame: Baseline and six months. ] [ Designated as safety issue: No ]
    The Visual Analog Scale will be used to measure the severity of specific behavioral symptoms chosen by the caregiver(s).

  • Eye tracking [ Time Frame: Baseline and six months. ] [ Designated as safety issue: No ]
    There are several eye tracking measures, each intended to measure different outcomes including social gaze, social reciprocity, and attention.

  • Preschool Language Scale [ Time Frame: Baseline and six months. ] [ Designated as safety issue: No ]
    The Preschool Language Scale is designed to measure language comprehension and communication in young children.

  • Sensory Processing Measure [ Time Frame: Baseline and six months ] [ Designated as safety issue: No ]
    The Sensory Processing Measure is a questionnaire that will be used to measure specific problems, including under- and over-responsiveness, sensory-seeking behavior, and perceptual problems

  • Sensory Profile [ Time Frame: Baseline and six months ] [ Designated as safety issue: No ]
    The Sensory Profile is designed to measure sensory-related difficulties.

  • Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) [ Time Frame: Baseline and six months ] [ Designated as safety issue: No ]
    The Vineland-II measures the personal and social skills of individuals from birth through adulthood.


Enrollment: 57
Study Start Date: January 2012
Estimated Study Completion Date: September 2015
Primary Completion Date: August 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: 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.
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.
Experimental: Active
This arm will undergo identical treatment and assessments as the placebo group. This group will receive the active agent, sertraline.
Drug: 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.
Other Name: Zoloft

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.

  Eligibility

Ages Eligible for Study:   24 Months to 68 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01474746

Locations
United States, California
UC Davis M.I.N.D. Institute
Sacramento, California, United States, 95817
Sponsors and Collaborators
University of California, Davis
Investigators
Principal Investigator: Randi J Hagerman, MD UC Davis MIND Institute
Principal Investigator: Kathleen Angkustsiri, MD UC Davis MIND Institute
  More Information

No publications provided

Responsible Party: Randi J. Hagerman, MD, Principle Investigator, University of California, Davis
ClinicalTrials.gov Identifier: NCT01474746     History of Changes
Other Study ID Numbers: 271070, R40MC22641
Study First Received: November 10, 2011
Last Updated: August 12, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by University of California, Davis:
Fragile X Syndrome
Pediatrics
sertraline
autism

Additional relevant MeSH terms:
Fragile X Syndrome
Syndrome
Chromosome Disorders
Congenital Abnormalities
Disease
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Intellectual Disability
Mental Retardation, X-Linked
Nervous System Diseases
Neurobehavioral Manifestations
Neurologic Manifestations
Pathologic Processes
Sex Chromosome Disorders
Sertraline
Antidepressive Agents
Central Nervous System Agents
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Neurotransmitter Uptake Inhibitors
Pharmacologic Actions
Physiological Effects of Drugs
Psychotropic Drugs
Serotonin Agents
Serotonin Uptake Inhibitors
Therapeutic Uses

ClinicalTrials.gov processed this record on September 01, 2015