Trial of Sertraline to Treat Children With Fragile X Syndrome

NCT01474746 | Completed Phase 2 Results DMC

• 2 other identifiers: 271070R40MC22641
• Study Type: interventional
• Target: 57
• Locations: 1 country
• Sites: 1

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.

Conditions

Fragile X Syndrome

Keywords

Fragile X Syndrome; Pediatrics; sertraline; autism

Outcome Measures

Primary Outcomes (3)

• Change in Mullen Scales of Early Learning - Expressive Language Raw Score

  The Mullen Scales of Early Learning (MSEL) is a cognitive test to measure cognitive ability and language development. The test has five scales: gross motor, visual reception, fine motor, receptive language, and expressive language. Shown here are the baseline and 6-month follow-up raw scores from the expressive language scale. This scale's raw scores range from 0 to 50. The lower the score on this scale, the weaker the ability; the higher the score, the greater the ability. The MSEL was administered at the baseline visit and at the 6-month follow-up visit.

  From baseline visit to six-month visit.

• Clinical Global Impression - Improvement

  The Clinical Global Impression - Improvement (CGI-I) is used to measure the overall behavioral change of an individual and their therapeutic response. The CGI-I is a 3-item observer-rated scale administered by the physician to the caregiver, who assesses improvement using a 7-point scale: 1 = Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = No change; 5 = Minimally worse; 6 = Much worse; and 7 = Very much worse. Therefore, the lower the score, the greater the behavioral improvement as rated by the caregiver. Shown here are the CGI-I mean scores from the 6-month follow-up visit.

  6-month follow-up visit score

• Change in Mullen Scales of Early Learning - Expressive Language Standard T Score

  The Mullen Scales of Early Learning (MSEL) is a cognitive test to measure cognitive ability and language development. The test has five scales: gross motor, visual reception, fine motor, receptive language and expressive language. Based on the raw score obtained by the participant in each scale the scoring software computes T scores, percentile ranks, and age equivalents for each scale separately. Shown here are the baseline and 6-month follow-up T scores from the expressive language scale. T scores have a range of 20 to 80, a mean of 50, and a standard deviation of 10. Any child scoring at or below 1.5 standard deviations below the average is considered presenting significant delays. The lower the T score, the worse the outcome. The MSEL was administered at the baseline visit and at the 6-month follow-up visit.

  From baseline visit to six-month visit

Secondary Outcomes (26)

• Autism Diagnostic Observation Schedule

  At baseline visit

• Visual Analog Scale

  At baseline visit

• Eye Tracking

  At baseline visit

• Preschool Language Scale-fifth Edition (PLS-5): AC+EC Total Raw Score

  At baseline visit

• Sensory Processing Measure - Preschool (SPM-P) Social Participation: Raw Score

  At baseline visit

Study Arms (2)

Placebo

PLACEBO COMPARATOR

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

Active

EXPERIMENTAL

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

Drug: Sertraline

Interventions

Sertraline 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.

Also known as: Zoloft

Active

Placebo 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.

Placebo

Eligibility Criteria

• Age: 24 Months - 68 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Fragile X Syndrome

You may not qualify if:

• 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

Sponsors & Collaborators

• Randi J. Hagerman, MD: lead

Study Sites (1)

UC Davis M.I.N.D. Institute

Sacramento, California, 95817, United States

Location

MeSH Terms

Conditions

Fragile X Syndrome; Autistic Disorder

Interventions

Sertraline

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System; Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

1-Naphthylamine; Amines; Organic Chemicals; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Polycyclic Compounds

Limitations and Caveats

Several secondary analyses. Subject non-compliance with eye tracking led to unreliable/uninterpretable data. Subjects and caregivers were unblinded at the time they completed the study. Limited number of girls in the study (total 9 girls).

Results Point of Contact

• Title: Dr. Randi Hagerman
• Organization: University of California, Davis, MIND Institute

rjhagerman@ucdavis.edu

916-703-0247

Study Officials

• Randi J Hagerman, MD

  UC Davis MIND Institute

  PRINCIPAL INVESTIGATOR

• Kathleen Angkustsiri, MD

  UC Davis MIND Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principle Investigator

Study Record Dates

• First Submitted: November 10, 2011
• First Posted: November 18, 2011
• Study Start: January 1, 2012
• Primary Completion: August 1, 2015
• Study Completion: September 1, 2015
• Last Updated: September 25, 2017
• Results First Posted: September 25, 2017

Record last verified: 2017-08

Locations

US (1)