Neurophysiological and Acute Pharmacological Studies in FXS Patients

Fragile X Syndrome Clinical Trial

Official title:

Neurophysiological and Acute Pharmacological Studies in FXS Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02998151
• Other study ID #: CIN001 - {LAM}
• Secondary ID: U54HD082008
• Status: Completed
• Phase: Early Phase 1
• Start date: January 2016
• Est. completion date: November 2020

Study information

• Verified date: November 2021
• Source: Children's Hospital Medical Center, Cincinnati
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 29
• Est. completion date: November 2020
• Est. primary completion date: November 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 15 Years to 55 Years

Eligibility

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.

Related Conditions & MeSH terms

• Fragile X Syndrome

Intervention

Drug:
• Acamprosate
two 666mg pills
• Lovastatin
two 20mg pills
• Minocycline
two 135mg pills
• Placebo
placebo pill
• Baclofen
one 30mg pill

Locations

Country	Name	City	State
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
Children's Hospital Medical Center, Cincinnati	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in EEG Relative Gamma Power	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.	Pre-dose, 4-hour post-dose
Primary	Clinical Global Impressions-Improvement	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).	4-hour post-dose
Secondary	Woodcock Johnson Test of Cognitive Abilities - Auditory Attention Task	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).	4-hour post-dose
Secondary	Change From Pre-dose in the Repeatable Battery for the Assessment of Neuropsychological Status at 4 Hours 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.	Pre-dose, 4-hour post dose
Secondary	Test of Attentional Performance for Children (KiTAP) Test of Alertness	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.	Predose, 4-hour post-dose