Skip to main content
OpenTrials
Completed

NCT Number: NCT02998151

Neurophysiological and Acute Pharmacological Studies in FXS Patients

The aim of this study is to utilize neurophysiologic assessments, behavioral measures and clinical measures to assess how much deficits associated with Fragile X Syndrome from pre-dose to post-dose using pharmacology.

Completed

Looking for future studies?

Notify Me

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

Age range

15 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Subjects ages 15-55, with fragile X syndrome (FXS) who completed the study entitled "Mechanisms and brain circuits underlying fragile X syndrome (IRB # 2015-8425). FXS is defined as full FMR1 mutations (>200 CGG repeats) confirmed by genetic testing.
  • General good health as determined by physical exam, medical history and laboratory work up.

Exclusion criteria

  • Subjects with a history of intolerance to acamprosate, lovastatin, or minocycline will be excluded.
  • Subjects will also be excluded if they have taken any investigational drug within 3 months, have a history of substance abuse or dependence within 6 months, or significant psychiatric or central nervous system neurological disease unrelated to FXS.
  • Uncontrolled seizures impact EEG data as do anticonvulsants, barbiturates, lithium and benzodiazepines and are exclusions (within 5 half-lives). Those taking other psychiatric medications must be on stable doses for 4 weeks before any testing.
  • For female subjects of child bearing potential, a positive urine pregnancy test.
  • Potential subjects with a creatinine clearance < 50 mL/min will be excluded.
  • Identified medical issues, inability to tolerate study procedures or study drug per the discretion of the Principal Investigator.

Treatment and study plan

acamprosate

Drug

two 666mg pills

lovastatin

Drug

two 20mg pills

Minocycline

Drug

two 135mg pills

Placebo

Drug

placebo pill

Baclofen

Drug

one 30mg pill

Primary outcomes

  1. Change in EEG Relative Gamma Power

    Time frame: Pre-dose, 4-hour post-dose

    EEG relative gamma power at rest was calculated as the percent of power in the gamma frequencies relative to the sum of power in all frequency bands, averaged across electrodes, and calculated separately at pre-dose and post-dose timepoints. To assess the impact of drug, the pre-dose relative gamma power was subtracted from post-dose relative gamma power. Higher numbers indicate more relative gamma power post-dose; lower numbers indicate more relative gamma power pre-dose.

  2. Clinical Global Impressions-Improvement

    Time frame: 4-hour post-dose

    The Clinical Global Impressions - Improvement (CGI-I) requires the clinician to assess how much the patient's illness has changed relative to pre-dose, from 1 (very much improved) to 7 (very much worse).

Secondary outcomes

  1. Woodcock Johnson Test of Cognitive Abilities - Auditory Attention Task

    Time frame: 4-hour post-dose

    Woodcock Johnson Test of Cognitive Abilities III Auditory Attention subscale. Participants must identify orally presented words amid increasingly intense background noise. The scores for this subtask range from 0-50, with higher scores indicating a better outcome. Raw scores for this subscale are reported (rather than standard scores, or age- or grade-equivalents).

  2. Change From Pre-dose in the Repeatable Battery for the Assessment of Neuropsychological Status at 4 Hours Post Dose

    Time frame: Pre-dose, 4-hour post dose

    Four 10-item lists of unrelated words were presented orally to the examinee who was then required to immediately recall words presented, at both pre-dose and post-dose timepoints. The impact of drug was assessed by subtracting the number of words remembered post-dose from the number of words remembered pre-dose. Lower numbers indicate more words remembered post-dose; higher numbers indicate more words remembered pre-dose.

  3. Test of Attentional Performance for Children (KiTAP) Test of Alertness

    Time frame: Predose, 4-hour post-dose

    Computerized task where an examinee is required to push a key when a target stimulus is presented on the screen. Scores are presented as change in median reaction time (RT), in milliseconds.

Sponsors and collaborators

Lead sponsor

Children's Hospital Medical Center, Cincinnati

Other

Collaborators

  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Registry information

Important dates

Study start
2016
Primary completion
2020
Study completion
2020
First posted
Dec 20, 2016
Registry last updated
Nov 26, 2021

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.

Related clinical trials

Published trials that share one or more normalized conditions with this study.