Status:
ACTIVE_NOT_RECRUITING
Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Collaborating Sponsors:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Conditions:
Fragile X Syndrome
Eligibility:
All Genders
18-45 years
Phase:
PHASE2
Brief 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 us...
Eligibility Criteria
Inclusion
- Subjects ages 18-45, with FXS who completed the study entitled "Mechanisms and brain circuits underlying fragile X syndrome" (IRB # 2015-8425) or appropriate baseline measures through Biorepository (2013-7327).
- 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.
- Stanford Binet IQ \<85
- Stable dosing of psychotropic drugs for at least 4 weeks.
Exclusion
- Subjects with a history of intolerance to baclofen, roflumilast, or memantine 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 CNS neurological disease unrelated to FXS.
- Uncontrolled seizures or history of epilepsy with a seizure in the past 6 months
- Auditory or visual impairments that cannot be corrected based on visual and auditory screener benchmarks.
- Moderate to severe renal or hepatic impairment and determined by a study physician incorporating data from exam, medical history and laboratory value evaluation among other data points.
- Use of barbiturates, benzodiazepines, antiepileptics, or other GABAergic or glutamatergic modulators
- Current use of: Amifampridine, Butalbital, Codeine, Doxylamine, Ethanol, Hydrocodone, Isocarboxazid, Kava, Metoclopramide, Midazolam, Oxybate, Phenelzine, Promethazine, Thalidomide, Tranylcypromine, Trimethobenzamide, Erythromycin, Ketoconazole, Fluvoxamine, Enoxacin, and Cimetidine.
- Those taking other psychiatric medications must be on stable doses for 4 weeks before the baseline visit.
- Pregnancy or breast-feeding. For female subjects of child bearing potential, a urine pregnancy test will be performed.
- 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.
Key Trial Info
Start Date :
September 8 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 5 2026
Estimated Enrollment :
45 Patients enrolled
Trial Details
Trial ID
NCT05418049
Start Date
September 8 2022
End Date
March 5 2026
Last Update
December 23 2025
Active Locations (1)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229