加波沙朵对患有脆性 X 综合征 (FXS) 的成年男性的安慰剂对照、单剂量挑战研究
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.
研究概览
研究类型
注册 (实际的)
阶段
- 阶段2
联系人和位置
学习地点
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Ohio
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Cincinnati、Ohio、美国、45229
- Cincinnati Children's Hospital Medical Center
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
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
学习计划
研究是如何设计的？
设计细节
- 主要用途：基础科学
- 分配：随机化
- 介入模型：交叉作业
- 屏蔽：四人间
武器和干预
参与者组/臂 |
干预/治疗 |
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实验性的：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 |
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实验性的：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 |
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实验性的：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 |
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实验性的：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 |
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
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Target Engagement of Gaboxadol Treatment on EEG Power
大体时间：Pre-dose compared to 60 minutes post-dose on 4 study visits, up to day 42
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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
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Target Engagement of Gaboxadol Treatment on Auditory Chirp
大体时间：Pre-dose compared to 60 minutes post-dose on 4 study visits, up to day 42
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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
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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To Investigate the Feasibility of Home Research Visits and Procedures in Adult Males With FXS
大体时间：Pre-dose, 60 minutes post-dose
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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
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To Investigate the Feasibility of High Density EEG Recording at Home in Adult Males With FXS
大体时间：Pre-dose, across four sessions (2 in-lab, 2 in-home), up to day 42
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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
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To Potentially Explore the Pharmacokinetics of Gaboxadol Treatment in Single-dose Trial Design
大体时间：Sixty minutes post-dose on 4 study visits, up to day 42
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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
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To Investigate the Effect of Gaboxadol Treatment on Neuropsychological Assessments
大体时间：Pre-dose compared to 60 minutes post-dose on 4 study visits, up to day 42
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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
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To Investigate the Effect of Gaboxadol Treatment on Eye Tracking Assessments
大体时间：Baseline compared to 60 minutes post-dose on 4 study visits, up to day 42
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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
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To Investigate the Effect of Gaboxadol Treatment on Clinician-rated Measures
大体时间：Post-dose on 4 study visits, up to day 42
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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
大体时间：Baseline compared to 60 minutes post-dose on 4 study visits, up to day 42
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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
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合作者和调查者
合作者
调查人员
- 首席研究员：Cragi A Erickson, MD、Children's Hospital Medical Center, Cincinnati
出版物和有用的链接
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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