NCT05509153招募中2 期
A Randomised Controlled Trial, of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 160
- 试验地点
- 5
- 主要终点
- Caudate Atrophy Rate on volumetric MRI
研究概览
简要总结
NAC-preHD is a phase II randomized placebo controlled study of oral NAC among premanifest HD gene expansion carriers, with clinical and radiological outcome at three years.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Able to provide informed consent
- •Huntingtin gene expansion carrier with >= 39 CAG repeats
- •Absence of unequivocal motor signs of HD - that is, UHDRS
- •Diagnostic Confidence Level needs to be <4 upon enrolment
- •Expected to develop clinical HD within 10 years of trial enrolment using the Langbehn formula
- •Availability of an informant for corroborative history
- •Negative serum pregnancy test for women of childbearing potential
- •If of childbearing potential, is able and agrees to remain abstinent or use adequate contraceptive methods
- •Ability to tolerate MRI scans
- •Ability to tolerate blood draws
- •Able to comply with all study protocol requirements, according to the investigators judgement
- •In the opinion of the investigator, medically, psychiatrically and neurologically stable at the time of enrolment
排除标准
- •Diagnosis of clinical HD
- •Known hypersensitivity to NAC
- •Pregnancy, breastfeeding or intention to do so prior to the end of the study
- •Exposure to any investigational drugs within 30 days of Baseline Visit
- •Use of supplemental NAC
- •Abnormalities in laboratory measurements, ECG or vital signs at screening, which precludes safe participation in the study
- •Current or history of substance abuse within one year of Baseline visit
- •Unstable psychiatric or acute medical illness including cancer, as determined by investigator
- •Current use of antipsychotic medications or Tetrabenazine
- •History of gene therapy, cell transplantation, or any experimental brain surgery
- •History of attempted suicide or suicidal ideation within 12 months prior to screening
- •Pre-existing structural brain lesion as assessed by a centrally read MRI scan during the screening period
研究组 & 干预措施
NAC
Experimental
1g N-Acetylcysteine capsules, taken orally twice a day.
干预措施: NAC (Drug)
Placebo
Placebo Comparator
Coated Placebo capsules, taken orally twice a day
干预措施: Placebo (Drug)
结局指标
主要结局
Caudate Atrophy Rate on volumetric MRI
时间窗: Baseline through end of study (up to 3 years)
Blinded assessment
Rate of motor phenoconversion
时间窗: Baseline through end of study (up to 3 years)
Defined by conversion to Diagnostic Confidence Level 4, upon blinded assessment using the UHDRS motor subscale
次要结局
- Stroop Word(Baseline through end of study (up to 3 years))
- UHDRS motor subscale (total score)(Baseline through end of study (up to 3 years))
- Trail Making Test(Baseline through end study (up to 3 years))
- Montreal Cognitive Assessment(Baseline through end of study (up to 3 years))
- Symbol Digit Modality Test(Baseline through end of study (up to 3 years))
- Changes in Daily Function(Baseline through end of study (up to 3 years))
- Change to Quality of Life(Baseline through end of study (up to 3 years))
- Incidence of adverse and/or serious adverse events (Safety and Tolerability)(Baseline through end of study (up to 3 years))
- Changes in Mood and Behavioural symptoms(Baseline through end of study (up to 3 years))
- Incidence of abnormal laboratory values and/or 12-lead ECG changes (Safety and Tolerability)(Baseline through end of study (up to 3 years))
- Study completion (Safety and Tolerability)(Baseline through end of study (up to 3 years))
研究者
Clement Loy
Director and Neurologist, Huntington Disease Service
Western Sydney Local Health District
研究点 (5)
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