Clinical trial NCT06334419: Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)
Official title: Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Fragile X Syndrome
Keywords: Fragile X
Fragile X Syndrome
FXS
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
Collaborators: name: Healx Limited
First posted: 2024-03-28
Enrollment: 10
Sex: MALE
Minimum age (years): 18
Maximum age (years): 40
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This study was designed as a 2-intervention crossover, with all study participants receiving all possible interventions. These are placebo and Gaboxadol.
Primary purpose: BASIC_SCIENCE

Brief 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.


Eligibility criteria: Inclusion Criteria:

1. Subject consents to participate, or if they are not their own legal guardian, offers assent supported by legally authorized representative consent
2. 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
3. Subject and caregiver are both able to understand the spoken national language clearly and caregiver can read and write to complete study assessments
4. Males age 18 to 40 years (inclusive)
5. 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
6. Is in general good health as deemed by the Investigator, determined by physical examination, medical history, and laboratory tests
7. 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
8. Is not sexually active or can confirm at least one form of contraceptive

Exclusion Criteria:

1. 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
2. Diagnosed with diabetes (Type 1 or II) or receiving any anti-diabetic medication
3. 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
4. Changes in psychotropic or anti-convulsant (where taken for reasons other than seizure control) drug treatment within 30 days prior to Screening
5. Significant changes in any educational, behavioral, and/or dietary interventions the month prior to Screening
6. Planned initiation of new, or modification of ongoing, interventions during the study
7. Unable or unwilling to take oral medication (whole capsule, despite assistance with a spoonful of applesauce, yoghurt, or equivalent liquid food)
8. 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
9. Has abnormal baseline laboratory assessments including, but not limited to, alanine aminotransferase (ALT) or aspartate aminotransferase (AST) or total bilirubin \>1.5 × the upper limit of normal (ULN), serum creatinine \>1.5 x ULN or other clinically relevant laboratory abnormality
10. Has a clinically significant heart rate or blood pressure (BP) at Screening as judged by the Investigator
11. Has received an investigational drug in any prior clinical study within 30 days or 5 half-lives (whichever is longer) prior to Screening

Primary outcomes:
measure: To evaluate target engagement of gaboxadol treatment on high density EEG recordings
timeFrame: Pre-dose, 60 minutes post-dose
Secondary outcomes:
measure: To investigate the feasibility of home research visits and procedures in adult males with FXS
timeFrame: Pre-dose, 60 minutes post-dose
measure: To investigate the feasibility of high density EEG recording at home in adult males with FXS
timeFrame: Pre-dose, 60 minutes post-dose
measure: To potentially explore the pharmacokinetics of gaboxadol treatment in single-dose trial design
timeFrame: Pre-dose, 60 minutes post-dose
measure: To investigate the effect of gaboxadol treatment on neuropsychological assessments
timeFrame: Pre-dose, 60 minutes post-dose
measure: To investigate the effect of gaboxadol treatment on eye tracking assessments
timeFrame: Pre-dose, 60 minutes post-dose
measure: To investigate the effect of gaboxadol treatment on clinician-rated measures
timeFrame: Pre-dose and post-dose
measure: To determine whether FMRP levels predict treatment response
timeFrame: Pre-dose and post-dose
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Cragi A Erickson, MD
role: PRINCIPAL_INVESTIGATOR
References: