Early Phase 1
Completed N=29
Neurophysiological and Acute Pharmacological Studies in FXS Patients
Source: ClinicalTrials.gov NCT02998151 ↗Enrolled (actual)
29
Serious AEs
0.0%
Results posted
Nov 2021
Primary outcomePrimary: Change in EEG Relative Gamma Power — 0.0024; -0.0077; -0.0039; 0.0019 percent of power in gamma frequencies
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in EEG Relative Gamma Power |
0.0024; -0.0077; -0.0039; 0.0019; -0.0160 | — |
| PRIMARY Clinical Global Impressions-Improvement |
3.70; 3.88; 3.97; 3.81; 3.94 | — |
| SECONDARY Woodcock Johnson Test of Cognitive Abilities - Auditory Attention Task |
32.84; 33.07; 32.93; 33.24; 33 | — |
| SECONDARY Change From Pre-dose in the Repeatable Battery for the Assessment of Neuropsychological Status at 4 Hours Post Dose |
-.20; -1.47; -1.25; -.69; -.88 | — |
| SECONDARY Test of Attentional Performance for Children (KiTAP) Test of Alertness |
13.76; -28.64; 18.59; 26.85; -31.44 | — |
Eligibility 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.
Data sourced from ClinicalTrials.gov (NCT02998151). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.