A Multicentre, Randomised, Double-blind, Placebo-controlled, Parallel-group Trial to Evaluate the Efficacy and Safety of BP1.4979 in Adult Patients With Essential Tremor
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- Bioprojet
- 入组人数
- 50
- 试验地点
- 14
- 主要终点
- Performance Subscale of The Essential Tremor Rating Assessment Scale (TETRAS-P)
研究概览
简要总结
The goal of this clinical trial is to learn if drug BP1.4979 works to treat essential tremors in adults. It will also learn about the safety of drug BP1.4979. The main questions it aims to answer are:
- Does the drug BP1.4979 reduce tremors in individuals with essential tremor?
- Do participants have a good safety and tolerability of drug BP1.4979?
Researchers will compare drug BP1.4979 to a placebo (a look-alike substance that contains no drug) to see if drug BP1.4979 works to treat essential tremors.
Participants will:
- Take the study drug (active or a placebo) twice daily over a 4-week period
- Visit the clinic 5 times for health checkups and questionnaires completion over a period of approximately 7 to 10 weeks
- Complete a diary weekly to assess the impact of essential tremor on daily life
详细描述
Essential tremor (ET) is the most common form of movement-related tremor, affecting approximately 1% of the global population and up to 5% of individuals over 60 years old. This condition significantly impacts quality of life, as it often makes daily tasks difficult to perform.
Current treatments, mainly beta-blockers (e.g., propranolol), do not work for everyone and often cause bothersome side effects.
The exact causes of ET remain poorly understood, suggesting that it may be a syndrome (a group of related disorders) rather than a single disease. Brain imaging studies show that certain parts of the brain responsible for movement do not function normally, but it is not yet clear whether this is a disease that worsens over time.
Researchers believe that the brain chemical dopamine plays a key role in ET. One of dopamine receptors could be an interesting target for treatment. Drugs acting on this receptor have already shown positive results in other conditions, such as Parkinson's disease.
The objective of this trial is to develop a more effective treatment for ET, addressing an unmet medical need and improving the quality of life of affected patients.
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 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female ≥18-85 years old
- •Confirmed diagnosis of ET, characterized by meeting the following criteria: (a) the presence of a bilateral upper limb action tremor that occurs in isolation; (b) a minimum duration of 3 years; and (c) the tremor may or may not be present in other areas such as the voice, or lower limbs
- •ET characterized by a TETRAS-P score of at least 1.5 in either the forward posture, wing beating posture, or finger-to-nose movement of at least one upper extremity
- •Female patient: post-menopausal woman having at least 12 months of natural amenorrhea without any alternative medical cause, or woman of childbearing potential using a highly effective method of contraception for the duration of the trial and for 1 month after stopping the investigational medication
排除标准
- •Severe tremor, defined as patient with ET characterized by a TETRAS-P score of ≥ 3 in either the forward posture, wing beating posture, or finger-to-nose movement of at least one upper extremity, or tremor of trunk
- •Isolated head tremor not accompanied with tremor of any other body part
- •Medical history or clinical evidence of any other conditions, whether medical, neurological, or psychiatric, that could potentially explain or contribute to the presence of tremors
- •Patient who takes a medication which may induce tremor
- •Direct or indirect injury or trauma to the nervous system within 3 months before the onset of tremor
- •Concomitant treatment with more than three drugs to treat ET
- •Any prior procedures for the treatment of ET such as deep brain stimulation, brain lesioning, or magnetic resonance (MR)-guided procedures, including MR-guided focused ultrasound
研究组 & 干预措施
BP1.4979
One tablet of BP1.4979 taken twice daily for 4 weeks, approximately 8 to 10 hours apart
干预措施: BP1.4979 (Drug)
Placebo
One tablet of placebo taken twice daily for 4 weeks, approximately 8 to 10 hours apart
干预措施: Placebo (Drug)
结局指标
主要结局
Performance Subscale of The Essential Tremor Rating Assessment Scale (TETRAS-P)
时间窗: 4 weeks
Change in the total score of the Performance Subscale of The Essential Tremor Rating Assessment Scale (TETRAS-P) after 4 weeks of treatment.
次要结局
未报告次要终点