Neurophysiological and Acute Pharmacological Studies in FXS Patients

• Status: Completed
• Trial end date: 2020-11-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Early Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborator: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
• Treatments: Acamprosate; Baclofen; Dihydromevinolin; L 647318; Lovastatin; Minocycline

Criteria

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.