Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndro… (NCT06334419) | Clinical Trial Compass
CompletedPhase 2
Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)
United States10 participantsStarted 2024-01-29
Plain-language summary
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.
Who can participate
Age range
18 Years – 40 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Subject consents to participate, or if they are not their own legal guardian, offers assent supported by legally authorized representative consent
. Subject is willing and able to comply with the study procedures as specified in the protocol and to comply with the study drug administration. Caregiver also commits to the study requirements prior to any study-related procedures
. Subject and caregiver are both able to understand the spoken national language clearly and caregiver can read and write to complete study assessments
. Males age 18 to 40 years (inclusive)
. Has FXS with molecular genetic confirmation of the full FMR1 mutation (\>200 cysteine-guanine-guanine \[CGG\] repeats). May have been confirmed historically or at Screening
. Is in general good health as deemed by the Investigator, determined by physical examination, medical history, and laboratory tests
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To evaluate target engagement of gaboxadol treatment on high density EEG recordings
. If receiving serotonin-selective reuptake inhibitor (SSRI), serotonin-norepinephrine reuptake inhibitor (SNRI), or serotonin antagonist and reuptake inhibitor (SARI), is on a stable, well-tolerated dose for the previous 3 months with no further changes anticipated
. Is not sexually active or can confirm at least one form of contraceptive
Exclusion criteria
. Any chronic major medical comorbid condition deemed by the Investigator as presenting added risk to the subject, including but not limited to, refractory hypertension, kidney disease, or liver disease
. Diagnosed with diabetes (Type 1 or II) or receiving any anti-diabetic medication
. Unstable seizure disorder, defined by any seizure within 6 months prior to baseline visit and/or a change in any anti-convulsant drug dosing in the 60 days prior to study consent
. Changes in psychotropic or anti-convulsant (where taken for reasons other than seizure control) drug treatment within 30 days prior to Screening
. Significant changes in any educational, behavioral, and/or dietary interventions the month prior to Screening
. Planned initiation of new, or modification of ongoing, interventions during the study
. Unable or unwilling to take oral medication (whole capsule, despite assistance with a spoonful of applesauce, yoghurt, or equivalent liquid food)
. Consumption of liver enzyme inducers or inhibitors including and not limited to foods, medicines, herbal remedies and supplements three days prior to any Visit. Foods or beverages containing CYP3A4/5 inhibitors (e.g., grapefruit, pomegranate, pomelo, and star fruit) should be avoided before taking study medication and for up to 1 hour post dose throughout the study