Early Phase 1
N=29
Neurophysiological and Acute Pharmacological Studies in FXS Patients
Fragile X Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02998151 ↗Enrolled (actual)
29
Serious AEs
0.0%
Results posted
Nov 2021
Primary outcome: Primary: Change in EEG Relative Gamma Power — 0.0024; -0.0077; -0.0039; 0.0019 percent of power in gamma frequencies
Study Design & Population
- Study type
- Interventional
- Phase
- Early Phase 1
- Interventions
- Acamprosate (Drug); Lovastatin (Drug); Minocycline (Drug); Placebo (Drug); Baclofen (Drug)
- Age
- Pediatric, Adult · 15+ yrs
- Sex
- All
- Sponsor
- Children's Hospital Medical Center, Cincinnati
- Primary completion
- Nov 2020
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 | — |
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.
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.