protocolSection identificationModule nctId: NCT06334419, orgStudyIdInfo id: 2023-0795, briefTitle: Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS), statusModule overallStatus: RECRUITING, startDateStruct date: 2024-01-29, primaryCompletionDateStruct date: 2024-09-30, completionDateStruct date: 2024-09-30, studyFirstPostDateStruct date: 2024-03-28, lastUpdatePostDateStruct date: 2024-03-28, sponsorCollaboratorsModule leadSponsor name: Craig Erickson, class: OTHER, collaborators name: Healx Limited, descriptionModule briefSummary: This is a single dose, placebo-controlled study. Male subjects aged 18 to 40 years (inclusive) with a diagnosis of FXS. Eligible subjects may enroll in this study comprised of two in home and two in clinic visits each 14 days apart, for a total of four visits. Subjects will be given single dose gaboxadol (10 mg) or matched placebo at each of these visits to take orally. Thus, all enrolled subjects will receive placebo at home and in clinic and receive gaboxadol at home and in clinic in a blinded fashion., conditionsModule conditions: Fragile X Syndrome, designModule studyType: INTERVENTIONAL, phases: PHASE2, designInfo allocation: NA, interventionModel: SINGLE_GROUP, interventionModelDescription: This study was designed as a 2-intervention crossover, with all study participants receiving all possible interventions. These are placebo and Gaboxadol., primaryPurpose: BASIC_SCIENCE, maskingInfo masking: NONE, maskingDescription: Quadruple masking (participant, care provider, investigator and outcomes assessor), enrollmentInfo count: 10, type: ESTIMATED, armsInterventionsModule interventions name: Gaboxadol, interventions name: Placebo, outcomesModule primaryOutcomes measure: To evaluate target engagement of gaboxadol treatment on high density EEG recordings, secondaryOutcomes measure: To investigate the feasibility of home research visits and procedures in adult males with FXS, secondaryOutcomes measure: To investigate the feasibility of high density EEG recording at home in adult males with FXS, secondaryOutcomes measure: To potentially explore the pharmacokinetics of gaboxadol treatment in single-dose trial design, secondaryOutcomes measure: To investigate the effect of gaboxadol treatment on neuropsychological assessments, secondaryOutcomes measure: To investigate the effect of gaboxadol treatment on eye tracking assessments, secondaryOutcomes measure: To investigate the effect of gaboxadol treatment on clinician-rated measures, secondaryOutcomes measure: To determine whether FMRP levels predict treatment response, eligibilityModule sex: MALE, minimumAge: 18 Years, maximumAge: 40 Years, stdAges: ADULT, contactsLocationsModule locations facility: Cincinnati Children's Hospital Medical Center, status: RECRUITING, city: Cincinnati, state: Ohio, zip: 45229, country: United States, contacts name: Ashley Dapore, role: CONTACT, phone: 513-517-1580, email: ashley.dapore@cchmc.org, contacts name: Hannah J Sachs, MPA, role: CONTACT, phone: 513-636-2592, email: hannah.sachs@cchmc.org, contacts name: Craig A Erickson, MD, role: PRINCIPAL_INVESTIGATOR, contacts name: Kelli Dominick, MD, PhD, role: SUB_INVESTIGATOR, contacts name: Meredith Nelson, PhD, role: SUB_INVESTIGATOR, contacts name: Ernest Pedapati, MD, role: SUB_INVESTIGATOR, contacts name: Debra Reisinger, PhD, role: SUB_INVESTIGATOR, contacts name: Lauren Schmitt, PhD, role: SUB_INVESTIGATOR, geoPoint lat: 39.12713, lon: -84.51435, hasResults: False