NCT05122650已完成2 期
A Phase 2b, 12-week, Double-blind, Placebo-controlled, Randomized, Parallel Group, Multicenter Study of the Safety and Efficacy of JZP385 in the Treatment of Adults With Moderate to Severe Essential Tremor
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 420
- 试验地点
- 73
- 主要终点
- Change From Baseline to Week 12 on the TETRAS Composite Outcome Score as Summarized by Each Dose of JZP385 and Placebo
研究概览
简要总结
This is a 12-week, double-blind, placebo-controlled, randomized, parallel-group, multicenter study of the safety and efficacy of JZP385 in the treatment of adult participants with moderate to severe ET.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be 18 to 80 years of age inclusive, at the time of signing the informed consent.
- •Participants who are diagnosed with ET (including ET plus) according to the MDS Consensus Statement on the Classification of Tremors from the Task Force on Tremor of the International Parkinson's and Movement Disorder Society.
- •Participants have moderate to severe disability associated with tremor as determined by a score of ≥ 22 on the TETRAS-ADL subscale and a CGI-S rating of at least moderate for participants' ability to function.
- •Sex and Contraceptive/Barrier Requirements
- •During the study intervention and for at least 30 days after the last dose of study intervention male participants must refrain from donating sperm. Non-abstinent males must agree to use a male condom in combination with female partner use of a highly effective contraceptive method with a failure rate of < 1% per year. All male participants must agree to use a male condom when engaging in any activity that allows for passage of ejaculate to another person.
- •Female participants must not be pregnant or breastfeeding, are either women of non-childbearing potential (WONCBP), or are women of childbearing potential (WOCBP) using a highly effective contraceptive method with a failure rate of < 1% during the study intervention period and for at least 30 days after the last dose of study intervention. Male partners of WOCBP are required to use barrier protection, eg, condoms, from the first dose of study intervention until 30 days after the last dose of study intervention.
- •A WOCBP must have a negative highly sensitive serum pregnancy test at Screening Visit 1 and negative urine pregnancy tests (unless serum is required by local regulations) at the Screening Visit 2 (if applicable) and at the Baseline Visit
- •If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.
- •Prior/Concomitant Antitremor Medications
- •If currently treated with antitremor medications, potential participants must be on a stable dosage for at least 6 weeks prior to Screening and must not anticipate making any changes to their antitremor medication for the duration of the study. Note: Treatment with some antitremor medications (eg, primidone) is not allowed in accordance with other exclusion criteria.
排除标准
- •Known history or current evidence of other medical or neurological conditions that may cause or explain the participant's tremor.
- •Has evidence at Screening of severe cognitive impairment as defined by a Montreal Cognitive Assessment (MoCA; score < 20) or has cognitive impairment that in the opinion of the investigator would prevent completion of study procedures (including the ability to accurately self-report on study questionnaires) or the ability to provide informed consent.
- •Current suicidal risk as determined from history, by presence of active suicidal ideation as indicated by positive response to item 4 or 5 on the C-SSRS (within the past 24 months), or any history of suicide attempt; current or past (within 1 year) major depressive episode according to DSM-5 criteria.
- •History (within past 2 years at screening) or presence of a diagnosed substance use disorder (including alcohol, tobacco, and cannabis) according to DSM-5 criteria, known drug dependence, or seeking treatment for alcohol or substance abuse related disorder.
- •Prior magnetic resonance (MR)-guided focused ultrasound thalamotomy, surgical intervention (eg, deep brain stimulation, ablative thalamotomy, gamma knife thalamotomy), or inability to refrain from using a device for treatment of tremor for the duration of the treatment period.
- •Botulinum toxin injection for the treatment of upper limb tremor in the 6 months before screening or planned use at any time during the study.
- •Treatment with any medication that could produce tremor taken within 2 weeks or 5 half-lives (whichever is longer) before screening or anticipated use at any time during participation in the study.
- •Use of prescription of nonprescription drugs or other products known to be inducers of CYP3A4 that are known to decrease AUC by > 30% (eg, primidone) and which cannot be discontinued at least 4 weeks before Baseline or planned use at any time during the study.
- •Use of prescription or nonprescription drugs, or other products (eg, grapefruit, grapefruit juice, or Seville oranges) known to be strong or moderate inhibitors of CYP3A4, that cannot be discontinued 2 weeks or 5 half-lives, whichever is longer, before Baseline or planned use at any time during the study.
- •Use of proton pump inhibitors that cannot be discontinued at least 2 weeks before Baseline, or planned use at any time during the study. Occasional use of antacids or histamine-2 receptor antagonists will be permitted, but antacids should be taken at least 4 hours before or after study intervention; and histamine-2 receptor antagonists should be taken at least 4 hours after and at least 12 hours before study intervention.
- •Inability to refrain from use of medication/substance(s) that might produce tremor or interfere with the evaluation of tremor on study visit days prior to discharge such as, but not limited to, stimulant decongestants, beta-agonist bronchodilators, and alcohol.
- •Regular use of more than 3 units of alcohol per day.
- •Regular consumption of caffeine > 400 mg/day or > 4 cups of coffee per day
研究组 & 干预措施
10 milligram (mg) JZP385
Experimental
Participants will initially receive 5 mg/day from Day 1 through Day 7, and 10 mg/day starting on Day 8.
干预措施: JZP385 (Drug)
20 mg JZP385
Experimental
Participants will initially receive 5 mg/day from Day 1 through Day 7, 10 mg/day from Day 8 through Day 14, and 20 mg/day starting on Day 15.
干预措施: JZP385 (Drug)
30 mg JZP385
Experimental
Participants will initially receive 5 mg/day from Day 1 through Day 7, 10 mg/day from Day 8 through Day 14, 20 mg/day from Day 15 through Day 21, and 30 mg/day starting on Day 22.
干预措施: JZP385 (Drug)
Placebo
Placebo Comparator
Participants will receive placebo from Day 1.
干预措施: Placebo (Other)
结局指标
主要结局
Change From Baseline to Week 12 on the TETRAS Composite Outcome Score as Summarized by Each Dose of JZP385 and Placebo
时间窗: Change from baseline to week 12
The TETRAS composite outcome score is the sum of modified items 1 - 11 of the TETRAS-ADL subscale and modified items 6 - 7 of the TETRAS-PS. The TETRAS-ADL subscale is a patient-rated scale administered by a trained interviewer that assesses the impact of tremor on day-to-day functioning, such as eating, drinking, dressing, and other fine motor skills. The TETRAS-PS is a clinical rating scale that quantifies tremor in the head, face voice, limbs and trunk. Items 6 (drawing an Archimedes spiral using left and right hands) and 7 (handwriting) of the TETRAS-PS evaluate the impact of upper limb tremor on performance. Each item from the modified subscales ranges from 0 - 3, with 0 representing normal or slightly abnormal and 3 representing severely abnormal. The sum of the 14 items provides the TETRAS composite outcome score, which ranges from 0 - 42, with higher scores representing more severe ET.
次要结局
- Percentage of Participants Who Improved (≥ 1-Point Improvement) From Baseline to Week 12 on the Clinical Global Impression- Severity Scale (CGI-S)(Change from baseline to week 12)
- Proportion of Participants Reported as Much Improved on the Clinical Global Impression of Change (CGI-C) at Week 12(Week 12)
- Proportion of Participants Reported as Much Improved on the Patient Global Impression of Change (PGI-C) at Week 12(Change from baseline to week 12)
- Change From Baseline to Week 12 on the TETRAS-ADL Subscale as Summarized by Each Dose of JZP385 and Placebo(Change from baseline to week 12)
- Change From Baseline to Week 12 on the TETRAS-PS Subscale as Summarized by Each Dose of JZP385 and Placebo(Change from baseline to week 12)
- Change From Baseline to Week 12 on the TETRAS Total Score, as Summarized by Each Dose of JZP385 and Placebo.(Change from baseline to week 12)
- Change From Baseline to Week 12 on the Upper Limb Score (Item 4) of the TETRAS-PS as Summarized by Each Dose of JZP385 and Placebo(Change from baseline to week 12)
- Change From Baseline to Week 12 on the Quality of Life in Essential Tremor Questionnaire (QUEST) as Summarized by Each Dose of JZP385 and Placebo(Change from baseline to week 12)
- Change From Baseline to Week 12 on the Essential Tremor Embarrassment Assessment (ETEA) Score A as Summarized by Each Dose of JZP385 and Placebo(Change from baseline to week 12)
- Change From Baseline to Week 12 on the Essential Tremor Embarrassment Assessment (ETEA) Score B as Summarized by Each Dose of JZP385 and Placebo(Change from baseline to week 12)
研究者
研究点 (73)
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