NCT04823052撤回2 期
A Phase 2, Placebo-controlled, Adaptive Design Study to Explore the Safety and Efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) and Possible Other Treatments in Adolescent and Adult Males With Fragile X Syndrome (FXS)
Healx Limited10 个研究点 分布在 2 个国家开始时间: 2022年5月25日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 试验地点
- 10
- 主要终点
- FXS Domain Specific Concerns
研究概览
简要总结
This study is to investigate the safety, tolerability and efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in males with Fragile X Syndrome (FXS) with confirmed full FMR1 mutation treated over a 10 week period in an outpatient setting.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 13 Years 至 40 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subject consents to participate, or if the subject are not the subjects own legal guardian, offers assent supported by legally authorized representative consent. Caregiver also commits to the study requirements prior to any study-related procedures
- •Willing and able to comply with the study procedures as specified in the protocol and to comply with the study drug administration
- •Subject and caregiver are both able to understand the spoken national language clearly and caregiver can read and write to complete study assessments
- •Males aged 13 to 40 years (inclusive)
- •Has FXS with molecular genetic confirmation of the full FMR1 mutation (>200 CGG repeats). May have been confirmed historically or at Screening
- •Weight ≥45 kg
- •CGI-S score ≥4
- •Is in general good health as deemed by the Investigator, determined by physical examination, medical history and laboratory tests
- •If receiving sertraline, is on a stable, well-tolerated dose for the previous 3 months with no further changes anticipated
- •Agrees not to discuss treatment outcomes on social media until subject has completed their End of Therapy visit
排除标准
- •Active or history of peptic or gastric ulcer or hemorrhage
- •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 entry
- •Patients with cardiovascular disease risk factors: Uncontrolled high blood pressure (systolic blood pressure >150 mmHg), Unstable angina, History of documented myocardial infarction or cerebrovascular accident, NYHA Class III and IV heart failure, Known uncontrolled hyperlipidemia as LDL-C ≥190 mg/dL or triglycerides ≥ 500 mg/dL
- •Chronic use of NSAIDs or other anti-inflammatory agents
- •Currently taking or have taken any cannabidiol (CBD) preparation within 30 days prior to screening
- •Currently taking or have taken sulindac or gaboxadol within 30 days prior to screening
- •Currently taking GABAergic agents (i.e., acamprosate, baclofen, vigabatrin, tiagabine, riluzole, benzodiazepines, and gabapentin)
- •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
- •History of adverse effects of sulindac or other NSAIDs that would prevent safe study completion
- •Unable or unwilling to take oral medication (whole capsule) or history of dysphagia or malabsorption
- •Has abnormal baseline laboratory assessments including, but not limited to, ALT or AST or total bilirubin >1.5 × ULN, or other clinically relevant laboratory abnormality
- •Has a clinically significant abnormal ECG, heart rate or BP at Screening as judged by the Investigator
- •Has received an investigational drug (either approved or not approved) in any prior clinical study within 30 days or 5 half-lives (whichever is longer) prior to Screening
研究组 & 干预措施
Placebo
Placebo Comparator
One capsule, twice a day
干预措施: Placebo (Drug)
Sulindac (HLX-0201), dose strength 1
Active Comparator
One capsule, twice a day
干预措施: Sulindac (HLX-0201), dose strength 1 (Drug)
Sulindac (HLX-0201), dose strength 2
Active Comparator
One capsule, twice a day
干预措施: Sulindac (HLX-0201), dose strength 2 (Drug)
Gaboxadol (HLX-0206)
Active Comparator
One capsule, twice a day
干预措施: Gaboxadol (HLX-0206) (Drug)
结局指标
主要结局
FXS Domain Specific Concerns
时间窗: Day 70
The Clinician/Caregiver FXS Domain Specific Concerns allows for the subject specific symptoms of concern to be assessed on an ongoing basis throughout the study. The specific concerns that correlate to the 6 domains (Clinician) or 3 Domains (Caregiver) will be assessed using a 7-point Likert scale.
NIH Cognitive Toolbox
时间窗: Day 70
Aberrant Behavior Checklist
时间窗: Day 70
Anxiety, Depression, and Mood Scale
时间窗: Day 70
Clinical Global Impression - I
时间窗: Day 70
次要结局
- Kiddie Test of Attentional Performance (KiTAP)(Day 70)
- To assess the safety and tolerability of each dose(Day 70)
- Emotional Faces Tobii Eye Tracking(Day 70)
- EEG(Day 70)
- CGI-S(Day 70)
研究者
研究点 (10)
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