Study on Patient- and Caregiver-reported Symptoms and Outcomes With Levodopa/Carbidopa Intestinal Gel for the Treatment of Advanced Parkinson's Disease
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 62
- 主要终点
- Change From Baseline in PDQ-39 Mobility Domain Score at Month 6
研究概览
简要总结
Patient reported outcomes (PRO) have become an important endpoint assessed in clinical trials. It is important to understand the relationship between medication and patients' symptomatology, quality of life and well-being. We predict levodopa/carbidopa intestinal gel (LCIG; Duodopa) will significantly improve quality of life and emotional well-being compared to baseline in patients with advance Parkinson disease (APD) not well controlled with conventional treatment.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with advanced levodopa-responsive Parkinson's disease, with at least, 2 hours "off" time or 2 hours of dyskinesia based on clinician's/investigator's medical judgment
- •Patients with advanced Parkinson's disease with severe motor fluctuations and dyskinesia when combination of conventional PD treatments was unable to satisfactory control symptoms.
- •Patients with advanced Parkinson's disease who meet the criteria for use of Levodopa/Carbidopa Intestinal Gel (LCIG) established in the summary of product characteristics, Spanish Neurology Society (SEN) guidelines, local/regional directories (CCAA) or site's protocols for use of LCIG in common clinical practice.
- •The decision to treat with LCIG is made by the physician prior to any decision to approach the patient to participate in this study.
- •Patients with a cognitive level enough to complete the questionnaires, confirmed by a Mini-mental state examination (MMSE) score of at least 26 points, according to the recommendation of the Movement Disorder Society (MDS) task force on dementia in Parkinson's Disease (PD)
排除标准
- •LCIG's contraindications included in the Summary of Product Characteristics (product label)
结局指标
主要结局
Change From Baseline in PDQ-39 Mobility Domain Score at Month 6
时间窗: Baseline, Month 6 (±15 days)
The PDQ-39 contains 39 items grouped in 8 domains: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Participants are asked to indicate the frequency of each event by selecting one of 5 options (Likert scale): never, occasionally, sometimes, often, always or cannot do at all, scoring 0, 1, 2, 3 or 4, respectively. The mobility domain score was calculated by expressing summed item scores as a percentage score ranging between 0 and 100. The lower scores indicate a better perceived health status and higher scores are associated with more health problems.
Change From Baseline in Parkinson's Disease Questionnaire-39 Item (PDQ-39) Global Score at Month 6
时间窗: Baseline, Month 6 (±15 days)
The PDQ-39 contains 39 items: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Participants are asked to indicate the frequency of each event by selecting one of 5 options (score 0 to 4 on a Likert scale). The global score was calculated by expressing summed item scores as a percentage score ranging between 0 and 100. The lower scores indicate a better perceived health status and higher scores are associated with more health problems.
Change From Baseline in PDQ-39 Cognition Domain Score at Month 6
时间窗: Baseline, Month 6 (±15 days)
The PDQ-39 contains 39 items grouped in 8 domains: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Participants are asked to indicate the frequency of each event by selecting one of 5 options (Likert scale): never, occasionally, sometimes, often, always or cannot do at all, scoring 0, 1, 2, 3 or 4, respectively. The cognition domain score was calculated by expressing summed item scores as a percentage score ranging between 0 and 100. The lower scores indicate a better perceived health status and higher scores are associated with more health problems.
Change From Baseline in PDQ-39 Communication Domain Score at Month 6
时间窗: Baseline, Month 6 (±15 days)
The PDQ-39 contains 39 items grouped in 8 domains: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Participants are asked to indicate the frequency of each event by selecting one of 5 options (Likert scale): never, occasionally, sometimes, often, always or cannot do at all, scoring 0, 1, 2, 3 or 4, respectively. The communication domain score was calculated by expressing summed item scores as a percentage score ranging between 0 and 100. The lower scores indicate a better perceived health status and higher scores are associated with more health problems.
Change From Baseline in PDQ-39 Stigma Domain Score at Month 6
时间窗: Baseline, Month 6 (±15 days)
The PDQ-39 contains 39 items grouped in 8 domains: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Participants are asked to indicate the frequency of each event by selecting one of 5 options (Likert scale): never, occasionally, sometimes, often, always or cannot do at all, scoring 0, 1, 2, 3 or 4, respectively. The stigma domain score was calculated by expressing summed item scores as a percentage score ranging between 0 and 100. The lower scores indicate a better perceived health status and higher scores are associated with more health problems.
Change From Baseline in PDQ-39 Emotional Well-Being Domain Score at Month 6
时间窗: Baseline, Month 6 (±15 days)
The PDQ-39 contains 39 items grouped in 8 domains: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Participants are asked to indicate the frequency of each event by selecting one of 5 options (Likert scale): never, occasionally, sometimes, often, always or cannot do at all, scoring 0, 1, 2, 3 or 4, respectively. The emotional well-being domain score was calculated by expressing summed item scores as a percentage score ranging between 0 and 100. The lower scores indicate a better perceived health status and higher scores are associated with more health problems.
Change From Baseline in PDQ-39 Bodily Discomfort Domain Score at Month 6
时间窗: Baseline, Month 6 (±15 days)
The PDQ-39 contains 39 items grouped in 8 domains: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Participants are asked to indicate the frequency of each event by selecting one of 5 options (Likert scale): never, occasionally, sometimes, often, always or cannot do at all, scoring 0, 1, 2, 3 or 4, respectively. The bodily discomfort domain score was calculated by expressing summed item scores as a percentage score ranging between 0 and 100. The lower scores indicate a better perceived health status and higher scores are associated with more health problems.
Change From Baseline in PDQ-39 Activities of Daily Living Domain Score at Month 6
时间窗: Baseline, Month 6 (±15 days)
The PDQ-39 contains 39 items grouped in 8 domains: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Participants are asked to indicate the frequency of each event by selecting one of 5 options (Likert scale): never, occasionally, sometimes, often, always or cannot do at all, scoring 0, 1, 2, 3 or 4, respectively. The activities of daily living domain score was calculated by expressing summed item scores as a percentage score ranging between 0 and 100. The lower scores indicate a better perceived health status and higher scores are associated with more health problems.
Change From Baseline in PDQ-39 Social Support Domain Score at Month 6
时间窗: Baseline, Month 6 (±15 days)
The PDQ-39 contains 39 items grouped in 8 domains: mobility (items 1-10), activities of daily living (items 11-16), emotional well-being (items 17-22), stigma (items 23-26), social support (items 27-29), cognitions (items 30-33), communication (items 34-36), and bodily discomfort (items 37-39). Participants are asked to indicate the frequency of each event by selecting one of 5 options (Likert scale): never, occasionally, sometimes, often, always or cannot do at all, scoring 0, 1, 2, 3 or 4, respectively. The social support domain score was calculated by expressing summed item scores as a percentage score ranging between 0 and 100. The lower scores indicate a better perceived health status and higher scores are associated with more health problems.
次要结局
- Change From Baseline in the Non-Motor Symptom Scale (NMSS) Global Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in NMSS Urinary Domain Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in NMSS Sexual Function Domain Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in Beck Anxiety Inventory (BAI) at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in NMSS Attention/Memory Domain Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Norris/Bond-Lader Visual Analogue Scale (VAS) Mean Global Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in Beck Depression Inventory, Second Edition (BDI-II) at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in Unified Parkinson's Disease Rating Scale Part III (UPDRS-III): Motor Examination at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in NMSS Sleep/Fatigue Domain Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in NMSS Mood/Cognition Domain Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in NMSS Gastrointestinal Tract Domain Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in NMSS Perceptual Problems/Hallucinations Domain Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Norris/Bond-Lader Visual Analogue Scale (VAS) Calmness/Relaxation Sub-Scale Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in Parkinson Fatigue Scale (PFS-16) at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Score of the Apathy Scale (AS) to Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Scale of Quality of Life of Caregivers (SQLC) Composite Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in NMSS Cardiovascular Domain Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in NMSS Miscellaneous Domain Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Norris/Bond-Lader Visual Analogue Scale (VAS) Alertness/Sedation Sub-Scale Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Treatment Satisfaction With Medicines Questionnaire (SATMED-Q) Convenience of Use at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Norris/Bond-Lader Visual Analogue Scale (VAS) Contented/Discontented Sub-Scale Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Treatment Satisfaction With Medicines Questionnaire (SATMED-Q) Treatment Effectiveness at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Scale of Quality of Life of Caregivers (SQLC) Professional Activities at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Treatment Satisfaction With Medicines Questionnaire (SATMED-Q) Composite Score at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Treatment Satisfaction With Medicines Questionnaire (SATMED-Q) Undesirable Side Effects at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Treatment Satisfaction With Medicines Questionnaire (SATMED-Q) Impact on Daily Living Activities at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Treatment Satisfaction With Medicines Questionnaire (SATMED-Q) Medical Care at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Scale of Quality of Life of Caregivers (SQLC) Caregiving Responsibilities at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in the Caregiver Stress Index (CSI) at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in Goldberg Anxiety and Depression Score (GADS): Depression Sub-Scale for Caregivers at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in WPAI Questionnaire: Mean Percentage of Impairment While Working Due to Caregiving (Presenteeism)(Baseline, Month 6 (±15 days))
- Change From Baseline in the Scale of Quality of Life of Caregivers (SQLC) Social and Leisure Activities at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in Goldberg Anxiety and Depression Score (GADS): Anxiety Sub-Scale for Caregivers at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in Work Productivity and Activity Impairment (WPAI) Questionnaire: Mean Percentage of Work Time Missed (Absenteeism) at Month 6(Baseline, Month 6 (±15 days))
- Correlation Between Caregivers' Quality of Life (SQLC) and Different Questionnaires at Baseline(Baseline)
- Correlation Between Caregivers' Quality of Life (SQLC) and Different Questionnaires at Month 6(Month 6 (±15 days))
- Change From Baseline in the Treatment Satisfaction With Medicines Questionnaire (SATMED-Q) Global Satisfaction at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in Zarit Burden Interview (ZBI) at Month 6(Baseline, Month 6 (±15 days))
- Change From Baseline in WPAI Questionnaire: Mean Percentage of Activity Impairment Due to Caregiving(Baseline, Month 6 (±15 days))
- Change From Baseline in WPAI Questionnaire: Mean Percentage of Overall Work Productivity Impairment (OWPI) Due to Caregiving(Baseline, Month 6 (±15 days))
- Correlation Between Participants' Quality of Life (PDQ-39) and Different Questionnaires at Baseline(Baseline)
- Correlation Between Participants' Quality of Life (PDQ-39) and Different Questionnaires at Month 6(Month 6 (±15 days))