Study of prevalence, clinical, electrophysiological characteristics, treatment response and quality of life in Orthostatic tremors/myoclonus- A longitudinal follow up study.
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 120
- 试验地点
- 2
- 主要终点
- Change in Orthostatic Tremor 10-item (OT-10) scale score — a validated measure of tremor severity and standing-related functional impairment.
研究概览
简要总结
LOTS is a multi-centre, prospective cohort study that builds upon an initial cohort of 58 retrospectively characterised patients identified at a tertiary care centre in India. Additional incident cases will be recruited across national and international sites during a two-year enrolment window. Participants will undergo assessments at baseline, 6 months, and annually for five years. Primary outcomes include the OT-10 scale and a novel Disease Burden Score (DBS). Secondary outcomes encompass quality of life, functional capacity, and treatment response. Exploratory analyses will include neurophysiology, imaging, and biomarker data where available. Latent Class Analysis (LCA) will identify subgroups with distinct trajectories, while mixed-effects models will assess longitudinal changes and multivariable regression will identify predictors of progression and treatment response. Potential limitations include attrition during long-term follow-up and variability in access to specialised electrophysiological assessments across centres.
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 18.00 Year(s) 至 80.00 Year(s)（—）
- 性别
- All
入选标准
- •Age between 18 and 80 years Male and female patients from all participating centers with a confirmed diagnosis of Orthostatic Tremor (OT) based on clinical and EMG findings Willingness to participate and provide written informed consent Ability to comply with study assessments and follow-up visits.
排除标准
- •Major psychiatric disorders that interfere with assessments Uncontrolled cardiovascular, metabolic, or systemic diseases Co-existing neurological disorders (e.g., Parkinson’s disease, peripheral neuropathy) Pregnant or lactating women Inability to provide informed consent or adhere to follow-up protocol.
结局指标
主要结局
Change in Orthostatic Tremor 10-item (OT-10) scale score — a validated measure of tremor severity and standing-related functional impairment.
时间窗: OT-10 scores will be recorded at baseline, 6 months, and annually at Years 1, 2, 3, 4 and 5.
次要结局
- Quality of Life (QoL)
- Fall Frequency(Recorded continuously & summarised at 6 months & annually at Years 1 to 5)
- Signs & Symptoms(Baseline, 6 months, & annually at Years 1 to 5)
- EMG Burst Frequency & Regularity(Baseline & annually where available (up to Year 5))
- Electrophysiological Classification(Baseline & annually up to Year 5)
- Diagnostic Reclassification(At each follow-up visit (6 months & annually up to Year 5))
- LCA-Derived Subtypes(Derived from data collected at baseline, 6 months, & each annual visit (Years 1–5))
- LCA Class-Specific Trajectories(Analysed from longitudinal data collected at baseline, 6 months, & annually at Years 1–5)
- Treatment Response Rate(Baseline & each follow-up visit (6 months & annually to Year 5))
- Predictors of Treatment Response(Baseline, 6 months, & annually at Years 1 to 5)
- Time to Clinical Improvement or Deterioration(Continuously monitored during follow-up; summarised at 6 months & annually at Years 1 to 5)
- Adverse Events(Recorded at every follow-up visit (6 months & annually at Years 1 to 5))
- Neuroimaging Correlates(Baseline (where available) & annually up to Year 5)
- Presence of Movement Disorder-Associated Variants(Baseline genetic sampling & once during follow-up (Year 3–5 as applicable))
- Genotype-Phenotype Correlations(Analysed from data collected at baseline & final follow-up (Year 5))
- Genetics & Treatment Response(Baseline genotyping correlated with treatment outcomes at Years 1–5)
- Oscillator Type Classification (Central vs Peripheral vs Mixed)(Baseline & annually where electrophysiological data are available (up to Year 5))
- Imaging Correlates of Oscillator Type(Baseline & annually where imaging data are available (up to Year 5))
- Oscillator Class & LCA Subtype Concordance(Derived from integrated datasets collected at baseline, 6 months, & annually at Years 1–5)
- Oscillator Type & Treatment Response(Baseline & each follow-up visit (6 months & annually up to Year 5))
- Patient Global Impression of Change (PGIC)(Baseline, 6 months, & annually at Years 1–5)
- Caregiver Burden Score(Baseline, 6 months, & annually at Years 1–5)
- Wearable-Based Tremor & Gait Monitoring(Baseline pilot measurement & annually where available (up to Year 5))
- Cost of Illness Assessment(Baseline & annually up to Year 5)
- Bayesian Modeling of Latent Class Transitions(Modelled using data obtained at baseline, 6 months, & annually up to Year 5)
研究者
Dr Anish Mehta
Ramaiah Medical College and Hospitals, Ramaiah University of Applied Sciences