Overview

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.