Placebokontrollerad endosutmaningsstudie av Gaboxadol hos vuxna män med fragilt X-syndrom (FXS)
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.
Studieöversikt
Status
Betingelser
Intervention / Behandling
Studietyp
Inskrivning (Faktisk)
Fas
- Fas 2
Kontakter och platser
Studieorter
-
-
Ohio
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Cincinnati, Ohio, Förenta staterna, 45229
- Cincinnati Children's Hospital Medical Center
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-
Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
Tar emot friska volontärer
Kön som är behöriga för studier
Beskrivning
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
- 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
- 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
- Has a clinically significant heart rate or blood pressure (BP) at Screening as judged by the Investigator
- Has received an investigational drug in any prior clinical study within 30 days or 5 half-lives (whichever is longer) prior to Screening
Studieplan
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Grundläggande vetenskap
- Tilldelning: Randomiserad
- Interventionsmodell: Crossover tilldelning
- Maskning: Fyrdubbla
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
|
Experimentell: Placebo in lab, Gaboxadol in home, Gaboxadol in lab, Placebo in home
Individuals in this condition received Placebo in lab, Gaboxadol in home, Gaboxadol in lab, then Placebo in home. |
Drug: Gaboxadol 10 mg as a single dose (two 5mg capsules) Two placebo capsules |
|
Experimentell: Gaboxadol in lab, Gaboxadol in home, Placebo in lab, Placebo in home
Individuals in this condition received Gaboxadol in lab, Gaboxadol in home, Placebo in lab, then Placebo in home. |
Drug: Gaboxadol 10 mg as a single dose (two 5mg capsules) Two placebo capsules |
|
Experimentell: Placebo in lab, Placebo in home, Gaboxadol in lab, Gaboxadol in home
Individuals in this condition received Placebo in lab, Placebo in home, Gaboxadol in lab, then Gaboxadol in home. |
Drug: Gaboxadol 10 mg as a single dose (two 5mg capsules) Two placebo capsules |
|
Experimentell: Gaboxadol in lab, Placebo in home, Placebo in lab, Gaboxadol in home
Individuals in this condition received Gaboxadol in lab, Placebo in home, Placebo in lab, then Gaboxadol in home. |
Drug: Gaboxadol 10 mg as a single dose (two 5mg capsules) Two placebo capsules |
Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Target Engagement of Gaboxadol Treatment on EEG Power
Tidsram: Pre-dose compared to 60 minutes post-dose on 4 study visits, up to day 42
|
Changes in EEG recordings with gaboxadol treatment (assessed at pre-dose, 60 minutes post-dose, average response across home and lab reported). compared with pre-dose on resting theta, alpha and gamma band absolute power. Absolute power measures reflect the median power averaged across 129 electrodes in μV²/Hz. Means are presented using the average of home and lab values. |
Pre-dose compared to 60 minutes post-dose on 4 study visits, up to day 42
|
|
Target Engagement of Gaboxadol Treatment on Auditory Chirp
Tidsram: Pre-dose compared to 60 minutes post-dose on 4 study visits, up to day 42
|
Changes in EEG recordings with gaboxadol treatment (60-90 minutes post-dose compared with pre-dose on brain response to auditory chirp stimuli), as measured by intertrial phase coherence (ITC). Means are presented using the average of home and lab values. |
Pre-dose compared to 60 minutes post-dose on 4 study visits, up to day 42
|
Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
To Investigate the Feasibility of Home Research Visits and Procedures in Adult Males With FXS
Tidsram: Pre-dose, 60 minutes post-dose
|
Quality and test-retest reliability of assessments performed at home compared with the hospital setting. This includes blood testing for Fragile X messenger ribonucleoprotein (FMRP: range of 0-40 picomolar) and eye-tracking of eye gaze to eye regions when viewing faces (range of value from 0 percent to 100 percent; higher values are associated with a neurotypical phenotype). |
Pre-dose, 60 minutes post-dose
|
|
To Investigate the Feasibility of High Density EEG Recording at Home in Adult Males With FXS
Tidsram: Pre-dose, across four sessions (2 in-lab, 2 in-home), up to day 42
|
This endpoint examined the reliability of pre-dose resting-state EEG absolute power estimates between in-lab and in-home assessments using intraclass correlation (ICC). ICCs were calculated separately for 129 electrodes using absolute power estimates (μV²/Hz). The reported ICC represents the mean ICC across electrodes, with the standard deviation reflecting variability in ICC values across electrodes. whole-head, pre-dose resting state EEG power estimates from data collected in-lab vs in-home using intraclass correlation (ICC). |
Pre-dose, across four sessions (2 in-lab, 2 in-home), up to day 42
|
|
To Potentially Explore the Pharmacokinetics of Gaboxadol Treatment in Single-dose Trial Design
Tidsram: Sixty minutes post-dose on 4 study visits, up to day 42
|
PK profile (the level of gaboxadol in human plasma) will be obtained at 60 +/- 10 minutes post-dose only). |
Sixty minutes post-dose on 4 study visits, up to day 42
|
|
To Investigate the Effect of Gaboxadol Treatment on Neuropsychological Assessments
Tidsram: Pre-dose compared to 60 minutes post-dose on 4 study visits, up to day 42
|
Changes in cognitive and neuropsychological assessment measures with gaboxadol treatment (RBANS List Learning). During the RBANS List Learning subtests, participants listen to a list of 10 words and repeat back as many as they can remember four times, for a maximum score of 40. Change scores were calculated by subtracting pre-dose words remembered from post-dose words remembered, with positive scores meaning an improvement in memory. |
Pre-dose compared to 60 minutes post-dose on 4 study visits, up to day 42
|
|
To Investigate the Effect of Gaboxadol Treatment on Eye Tracking Assessments
Tidsram: Baseline compared to 60 minutes post-dose on 4 study visits, up to day 42
|
Change in eye tracking outcomes (60-90 minutes post-dose compared to pre-dose on percent viewing of social versus geometric scene |
Baseline compared to 60 minutes post-dose on 4 study visits, up to day 42
|
|
To Investigate the Effect of Gaboxadol Treatment on Clinician-rated Measures
Tidsram: Post-dose on 4 study visits, up to day 42
|
Score on the Clinical Global Impressions - Improvement (CGI-I) scale, a clinician rating scale with "1" anchored to "very much improved" and "7" anchored to "very much worse." |
Post-dose on 4 study visits, up to day 42
|
|
To Determine Whether FMRP Levels Predict Treatment Response
Tidsram: Baseline compared to 60 minutes post-dose on 4 study visits, up to day 42
|
Relationship between FMRP (Fragile X messenger ribonucleoprotein) level and degree of change (baseline/screen to post-dose) across measures |
Baseline compared to 60 minutes post-dose on 4 study visits, up to day 42
|
Samarbetspartners och utredare
Sponsor
Samarbetspartners
Utredare
- Huvudutredare: Cragi A Erickson, MD, Children's Hospital Medical Center, Cincinnati
Publikationer och användbara länkar
Studieavstämningsdatum
Studera stora datum
Studiestart (Faktisk)
Primärt slutförande (Faktisk)
Avslutad studie (Faktisk)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Faktisk)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Nyckelord
Ytterligare relevanta MeSH-villkor
- Neurologiska manifestationer
- Sjukdomar i nervsystemet
- Genetiska sjukdomar, medfödda
- Neurobehavioral manifestationer
- Medfödda abnormiteter
- Heredodegenerativa störningar, nervsystemet
- Intellektuell funktionsnedsättning
- Genetiska sjukdomar, X-länkade
- Sexkromosomstörningar
- Kromosomstörningar
- Medfödda, ärftliga och neonatala sjukdomar och abnormiteter
- X-länkad intellektuell funktionsnedsättning
- Fragilt X-syndrom
- gaboxadol
Andra studie-ID-nummer
- 2023-0795
Läkemedels- och apparatinformation, studiedokument
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