A Long-term Health Economics Study of Intraduodenal Levodopa (Duodopa®) in Routine Care for Patients With Advanced Idiopathic Parkinson's Disease With Severe Motor Fluctuations and Hyper-/Dyskinesia
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 77
- 主要终点
- Direct Monthly Non-medical Costs Per Participant, SEK 2010
研究概览
简要总结
The primary objective of this study is to collect health economic data depicting the initial levels and natural progression over time of resource usage, Parkinson's disease (PD)-related costs, and health related quality of life (HRQoL) for a cohort of advanced PD patients treated with Duodopa (levodopa-carbidopa in an intestinal gel formulation), of which about one-third were Duodopa-naïve prior to the start of the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Advanced idiopathic Parkinson's disease
排除标准
- •Other diseases which might influence compliance or participation in the study
研究组 & 干预措施
Duodopa Naïve
Duodopa-naïve participants titrated to receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: Levodopa-carbidopa intestinal gel (LCIG) (Drug)
Duodopa Naïve
Duodopa-naïve participants titrated to receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: CADD-Legacy® 1400 ambulatory infusion pump (Device)
Duodopa Naïve
Duodopa-naïve participants titrated to receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: percutaneous endoscopic gastrostomy tube (PEG tube) (Device)
Duodopa Naïve
Duodopa-naïve participants titrated to receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: jejunal extension tube (J-tube) (Device)
Duodopa Non-naïve < 2 Years
Duodopa non-naïve participants treated with Duodopa for < 2 years receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: Levodopa-carbidopa intestinal gel (LCIG) (Drug)
Duodopa Non-naïve < 2 Years
Duodopa non-naïve participants treated with Duodopa for < 2 years receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: CADD-Legacy® 1400 ambulatory infusion pump (Device)
Duodopa Non-naïve < 2 Years
Duodopa non-naïve participants treated with Duodopa for < 2 years receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: percutaneous endoscopic gastrostomy tube (PEG tube) (Device)
Duodopa Non-naïve < 2 Years
Duodopa non-naïve participants treated with Duodopa for < 2 years receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: jejunal extension tube (J-tube) (Device)
Duodopa Non-naïve ≥ 2 years
Duodopa non-naïve participants treated with Duodopa for ≥ 2 years receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: Levodopa-carbidopa intestinal gel (LCIG) (Drug)
Duodopa Non-naïve ≥ 2 years
Duodopa non-naïve participants treated with Duodopa for ≥ 2 years receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: CADD-Legacy® 1400 ambulatory infusion pump (Device)
Duodopa Non-naïve ≥ 2 years
Duodopa non-naïve participants treated with Duodopa for ≥ 2 years receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: percutaneous endoscopic gastrostomy tube (PEG tube) (Device)
Duodopa Non-naïve ≥ 2 years
Duodopa non-naïve participants treated with Duodopa for ≥ 2 years receive Duodopa (levodopa/carbidopa intestinal gel) adjusted to an optimal clinical response for each participant, using the portable CADD Legacy Duodopa pump (CE 0473). Treatment is composed of 3 individually adjusted doses: the morning bolus dose (usually 5-10 mL [100-200 mg levodopa]); the continuous maintenance dose (usually 2-6 mL/hour [40-120 mg levodopa/hour]); and extra bolus doses, adjusted individually.
干预措施: jejunal extension tube (J-tube) (Device)
结局指标
主要结局
Direct Monthly Non-medical Costs Per Participant, SEK 2010
时间窗: Baseline (month -3) for Duodopa-naïve participants, then at Month 0, and monthly thereafter until study completion (up to 48 months) for all participants
Direct non-medical costs include nursing home, home help, personal assistance, informal care (from family member or friend) and transportation to inpatient, outpatient visits and nursing home. The average rate for US Dollar (USD) to SEK on 31 December 2010 was 1 USD = 6.734 SEK.
Euro QoL 5 Dimensions Quality of Life Instrument (EQ-5D) Descriptive Systems Summary Index Score at Baseline and Month 12
时间窗: Baseline (Month -3), Month 12
The EQ-5D is a participant-answered questionnaire scoring 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. EQ-5D health states, defined by the EQ-5D descriptive system, are converted into a single summary index by applying a formula that essentially attaches values (also called QOL weights or QOL utilities) to each of the levels in each dimension. EQ-5D Summary Index values range from -0.11 (worst health state) to 1.00 (perfect health state).
Unified Parkinson's Disease Rating Scale (UPDRS) Total Score, and UPDRS Subscores I, II, III, and IV at Baseline and Month 12
时间窗: Baseline (Month -3), Month 12
The UPDRS is an Investigator-used rating tool to follow the longitudinal course of Parkinson's disease. For Parts I-III and Total Score, each question is measured on a 5-point scale of 0 (normal or no disease effect) to 4 (maximum negative effect); for Part IV, questions are measured on a 5- or 2-point scale (0 or 1). Part I score is the sum of answers to 'Mentation, Behavior and Mood' questions, with a score range from 0-16. Part II score is the sum of answers to 'Activities of Daily Living' questions, with a score range from 0-52. Part III score is the sum of answers to 'Motor Examination' questions, with a score range from 0-108. Part IV score is the sum of answers to 'Complications of Therapy' questions, with a score range from 0-23. Total Score is the sum of the responses to the 31 questions (44 answers) that comprise Parts I-III of the scale, with a score range from 0-176. Higher scores are associated with more disability.
Total Monthly Cost Per Participant, in Swedish Crowns (SEK) 2010
时间窗: Baseline (month -3) for Duodopa-naïve participants, then at Month 0, and monthly thereafter until study completion (up to 48 months) for all participants
Total monthly costs include * Direct medical costs (inpatient care, outpatient care, and drug costs \[including Duodopa cost and cost of concomitant anti-PD medication\]). * Direct non-medical costs (nursing home, home help, personal assistance, informal care \[from family member or friend\] and transportation to inpatient, outpatient visits and nursing home). * Indirect costs (sick-leave and early retirement due to PD \[applied to individuals only up to the age of 65 since the main indirect cost item, early retirement due to disability, is only available for individuals 30-64 years old. Sixty-five is also a common retirement age in Sweden\]). The average rate for US Dollar (USD) to SEK on 31 December 2010 was 1 USD = 6.734 SEK.
EQ-5D Visual Analog Scale (VAS) Score at Baseline and Month 12
时间窗: Baseline (Month -3), Month 12
The EQ-5D VAS records the participant's self-rated health on a scale from 0-100 where 100 is the 'best imaginable health state' and 0 is the 'worst imaginable health state.' The scale was normalized to a scale of 0 to 1, with higher values indicating a better health state.
Monthly Drug Costs Per Participant, SEK 2010
时间窗: Baseline (month -3) for Duodopa-naïve participants, then at Month 0, and monthly thereafter until study completion (up to 48 months) for all participants
Drug costs include Duodopa cost and cost of concomitant anti-PD medication. Drug costs are a direct medical cost. The average rate for US Dollar (USD) to SEK on 31 December 2010 was 1 USD = 6.734 SEK.
Monthly Direct Medical Cost (Excluding Drug Costs) Per Participant, SEK 2010
时间窗: Baseline (month -3) for Duodopa-naïve participants, then at Month 0, and monthly thereafter until study completion (up to 48 months) for all participants
Direct medical costs consist of inpatient care, outpatient care (visits to physician, nurse, physiotherapist, occupational therapist, dietitian, speech therapist, counselor, and phone consultations). The average rate for US Dollar (USD) to SEK on 31 December 2010 was 1 USD = 6.734 SEK.
Indirect Monthly Costs Per Participant (Only Applied to Participants Younger Than 65) by Study Month, SEK 2010
时间窗: Baseline (month -3) for Duodopa-naïve participants, then at Month 0, and monthly thereafter until Month 36
Indirect costs consist of sick-leave and early retirement due to PD, are applied to individuals only up to the age of 65 since the main indirect cost item - early retirement due to disability - is only available for individuals 30-64 years old. Sixty-five is also a common retirement age in Sweden. The average rate for US Dollar (USD) to SEK on 31 December 2010 was 1 USD = 6.734 SEK.
次要结局
- PDQ-39 Cognition Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Dyskinetic Time) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Modified Hoehn and Yahr Staging: Worst Stage From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Mini Mental Status Examination (MMSE) Total Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint visit (Month 36 or last visit if discontinued early))
- PDQ-39 Communication Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Off Time) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Modified Hoehn and Yahr Staging: Current Stage From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Schwab and England Scale: Best "On" Period Stage From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MMSE Language Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MADRS Pessimistic Thoughts Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- PDQ-39 Emotional Well Being Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- PDQ-39 Social Support Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MADRS Suicidal Thoughts Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- PDQ-39 Mobility Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- PDQ-39 Activities of Daily Living Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Modified Hoehn and Yahr Staging: Best Stage From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MADRS Reduced Sleep Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MADRS Concentration Difficulties Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- PDQ-39 Bodily Discomfort Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Walking) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (On Time) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Off Magnitude) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MMSE Orientation Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MMSE Registration Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MMSE Attention and Calculation Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MMSE Recall Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MADRS Inner Tension Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MADRS Lassitude Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- PDQ-39 Stigma Subscale Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MADRS Reported Sadness Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MADRS Reduced Appetite Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MADRS Inability to Feel Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Parkinson's Disease Questionnaire-39 (PDQ-39) Summary Index Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Dyskinetic Magnitude) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Montgomery-Åsberg Depression Rating Scale (MADRS) Total Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- MADRS Apparent Sadness Scores From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Cramps) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Satisfied With Function) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Self-assessment) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Free Tapping - Accuracy) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Tapping, Increased Speed - Accuracy) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Drawing Impairment [Wavelet Method]) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Free Tapping - Speed) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Tapping, Random Chase - Speed) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))
- Electronic Diary: Morning and Day Scores (Tapping, Random Chase - Accuracy) From Baseline to Month 36(Baseline (Month -3), Months 0, 3, 6, 9, 12, 18, 24, 30, 36, endpoint (last non-missing value assigned to treatment for the participant))