Studio di sfida a dose singola, controllato con placebo, sul gaboxadolo in maschi adulti affetti da sindrome dell'X fragile (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.
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Tipo di studio
Iscrizione (Effettivo)
Fase
- Fase 2
Contatti e Sedi
Luoghi di studio
-
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Ohio
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Cincinnati, Ohio, Stati Uniti, 45229
- Cincinnati Children's Hospital Medical Center
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-
Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
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
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Scienza basilare
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione incrociata
- Mascheramento: Quadruplicare
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
|
Sperimentale: 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 |
|
Sperimentale: 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 |
|
Sperimentale: 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 |
|
Sperimentale: 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 |
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Target Engagement of Gaboxadol Treatment on EEG Power
Lasso di tempo: 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
Lasso di tempo: 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
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
To Investigate the Feasibility of Home Research Visits and Procedures in Adult Males With FXS
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
|
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To Determine Whether FMRP Levels Predict Treatment Response
Lasso di tempo: 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
|
Collaboratori e investigatori
Sponsor
Collaboratori
Investigatori
- Investigatore principale: Cragi A Erickson, MD, Children's Hospital Medical Center, Cincinnati
Pubblicazioni e link utili
Studiare le date dei record
Studia le date principali
Inizio studio (Effettivo)
Completamento primario (Effettivo)
Completamento dello studio (Effettivo)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
- Manifestazioni neurologiche
- Malattie del sistema nervoso
- Malattie genetiche, congenite
- Manifestazioni neurocomportamentali
- Anomalie congenite
- Malattie Eredodegenerative, Sistema Nervoso
- Disabilità intellettuale
- Malattie genetiche, legate all'X
- Disturbi del cromosoma sessuale
- Disturbi cromosomici
- Malattie e anomalie congenite, ereditarie e neonatali
- Disabilità intellettiva legata all'X
- Sindrome dell'X fragile
- gaboxadol
Altri numeri di identificazione dello studio
- 2023-0795
Informazioni su farmaci e dispositivi, documenti di studio
Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti
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