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Completed

NCT Number: NCT01053156

Trial of Minocycline to Treat Children With Fragile X Syndrome

This is a single center study at the UC Davis MIND Institute in patients age 3.5-16 years of age with fragile X syndrome (FXS), funded by a National Fragile X Foundation Grant. It is a controlled trial of minocycline, an antibiotic commonly used in children for infection or for treatment of neurodegenerative disorders. We are investigating its use in FXS because it lowers matrix metalloproteinase 9 (MMP9) levels, which are high in FXS, and it also strengthens brain connections in the animal models of FXS. We hypothesize that minocycline will likely be helpful for language, behavior and/or cognition in fragile X patients.

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Key information

Conditions

Fragile X Syndrome Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Sex Chromosome Disorders X-Linked Intellectual Disability cyclopia sequence

Age range

42 month–16 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

M.I.N.D. Institute at University of California at Davis Medical Center

Sacramento, California, 95817, United States

About this study

This is a single center study at the UC Davis MIND Institute in patients age 3.5-16 years of age with fragile X syndrome (FXS), funded by a National Fragile X Foundation Grant. It is a controlled trial of minocycline, an antibiotic commonly used in children for infection or for treatment of neurodegenerative disorders. We are investigating its use in FXS because it lowers matrix metalloproteinase 9 (MMP9) levels, which are high in FXS, and it also strengthens brain connections in the animal models of FXS. We hypothesize that minocycline will likely be helpful for language, behavior and/or cognition in fragile X patients.

The aim of this study is to carry out a double-blind placebo controlled trial of minocycline treatment in children with FXS who are 3.5 to 16 years of age. At baseline, we will assess behavior and perceptual and cognitive development. After the children have been treated for 3 months with either minocycline or placebo, they undergo the same baseline testing. They will then cross over and be treated for a second 3 months. We will carry out testing again at the end of the second 3 month period. We will also assess the side effects of minocycline treatment throughout the study.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Must have fragile X syndrome with molecular documentation
  • Current pharmacological treatment regimen has been stable for at least 4 weeks

Exclusion criteria

  • Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study
  • subjects who are unable to take oral medication
  • subjects who have been on minocycline previously
  • subjects who are allergic to minocycline or tetracyclines
  • subjects who are pregnant
  • subjects with history of lupus or hepatic dysfunction

Treatment and study plan

Minocycline Hydrochloride

Drug

Minocycline hydrochloride dosed orally once a day for 3 months.

Placebo

Drug

Placebo will be given daily for 3 months.

Primary outcomes

  1. Clinical Global Impression Scale (CGI)

    Time frame: 3 months (post first treatment) and 6 months (post second treatment)

    The CGI-I utilizes history from primary caregivers and incorporates it into a seven step clinical rating for follow up throughout treatment, from 1 "very much improved" to 7 "very much worse". Lower scores indicate more improvement. Scores were obtained post treatments. Scores from when the patients were on minocycline either first or second were combined and averaged to determine a least squares mean and placebo scores were obtained in the same manner.

  2. Visual Analogue Scale- Behavior 1

    Time frame: Baseline, 3 months, 6 months

    A VAS is used to represent a caregiver's assessment of given behaviors, which were chosen by the parents. Caregivers marked a 10 cm horizontal line representing a visual continuum of each behavior from "worst behavior" to "behavior not a problem." Greater values indicate greater improvement. This measure represents the first behavior that the caregivers noted, out of three.

Secondary outcomes

  1. Visual Analogue Scale- Behaviors 2

    Time frame: Baseline, 3 months, 6 months

    A VAS is used to represent a caregiver's assessment of given behaviors, which were chosen by the parents. Caregivers marked a 10 cm horizontal line representing a visual continuum of each behavior from "worst behavior" to "behavior not a problem." Greater values indicate greater improvement. This measure represents the second behavior that the caregivers noted, out of three.

  2. Expressive Vocabulary Test-2

    Time frame: Baseline, 3 months and 6 months

    The EVT-2 standard score assesses language development through a participant's one word synonym response to visual stimuli. Standard scores range from 20-160. A standard score of 100 is average, with a 15 point standard deviation. Higher values represent a better outcome.

  3. Vineland Adaptive Behavior Scale-II (VABS-II)Adaptive Behavior Composite Score

    Time frame: Baseline, 3 months, and 6 months

    The VABS-II Adaptive Behavior Composite Score was used to assess adaptive skills. An Adaptive Behavior Composite Score may range from 20-160 with an average of 100 with a standard deviation of 15. Higher scores show improvement.

  4. Aberrant Behavior Checklist-Community Edition (ABC-C)Composite Score

    Time frame: Baseline, 3 months, and 6 months

    The ABC-C composite scores were used to quantify the severity of a patient's behaviors. A composite score consists of subscale scores including Irritability and Agitation, Lethargy and Social Withdrawal, Stereotypic Behavior, Hyperactivity and Noncompliance, and Inappropriate Speech. The composite score may range from 0-174. Lower scores indicate improvement.

  5. Visual Analogue Scale Behavior 3- VAS3

    Time frame: Baseline, 3 months, 6 months

    A VAS is used to represent a caregiver's assessment of given behaviors, which were chosen by the parents. Caregivers marked a 10 cm horizontal line representing a visual continuum of each behavior from "worst behavior" to "behavior not a problem." Greater values indicate greater improvement. This measure represents the third behavior that caregivers noted, out of three.

  6. VAS Categorized by Behavior: Aggression/ ADHD

    Time frame: Baseline, 3 months, 6 months

    A VAS is used to represent a caregiver's assessment of given behaviors, which were chosen by the parents. Caregivers marked a 10 cm horizontal line representing a visual continuum of each behavior from "worst behavior" to "behavior not a problem." Greater values indicate greater improvement. This measure represents the least squares mean of all behaviors having to do with aggression or ADHD behaviors.

  7. VAS Categorized by Behavior:Anxiety/ Mood

    Time frame: Baseline, 3 months, 6 months

    A VAS is used to represent a caregiver's assessment of given behaviors, which were chosen by the parents. Caregivers marked a 10 cm horizontal line representing a visual continuum of each behavior from "worst behavior" to "behavior not a problem." Greater values indicate greater improvement. This measure represents the least squares mean of all behaviors having to do with anxiety or mood related behaviors.

  8. VAS Categorized by Behavior:Language/ Cognition

    Time frame: Baseline, 3 months, 6 months

    A VAS is used to represent a caregiver's assessment of given behaviors, which were chosen by the parents. Caregivers marked a 10 cm horizontal line representing a visual continuum of each behavior from "worst behavior" to "behavior not a problem." Greater values indicate greater improvement. This measure represents the least squares mean of all behaviors having to do with language or cognitive symptoms.

  9. VAS Categorized by Behavior: Other

    Time frame: Baseline, 3 months, 6 months

    A VAS is used to represent a caregiver's assessment of given behaviors, which were chosen by the parents. Caregivers marked a 10 cm horizontal line representing a visual continuum of each behavior from "worst behavior" to "behavior not a problem." Greater values indicate greater improvement. This measure represents the least squares mean of all behaviors having to do with other behaviors that were not able to be categorized.

Sponsors and collaborators

Lead sponsor

University of California, Davis

Other

Collaborators

  • The National Fragile X Foundation

Registry information

Official study title

Randomized Double-Blind Controlled Cross Over Trial of Minocycline in Children With Fragile X Syndrome

Important dates

Study start
2010
Primary completion
2011
Study completion
2011
First posted
Jan 21, 2010
Registry last updated
May 30, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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