Subcutaneous Immunoglobulin Therapy Effectiveness Monitoring in CIDP Patients Using Smart Devices
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 35
- 试验地点
- 4
- 主要终点
- Change in Inflammatory Rasch-built Overall Disability Scale (I-RODS)
研究概览
简要总结
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare autoimmune disease that affects the peripheral nerves. It causes progressive weakness and sensory loss in the arms and legs, which can severely limit daily activities. Many patients need long-term treatment with immunoglobulins, either through intravenous infusions (IVIG) or subcutaneous injections (fSCIG).
The S.T.E.P.S. study aims to explore how digital health technologies-specifically smartwatches-can help monitor the disease course and treatment effects in CIDP patients who use fSCIG at home. Current clinical tests are useful but sometimes miss small changes in strength or function. Wearables may provide a more detailed and continuous picture of patients' health between clinic visits.
Study Goals:
The main goal is to find out whether smartwatch data (such as step count and physical activity) reflect disease severity and treatment response when compared to standard clinical scores like the Inflammatory Neuropathy Cause and Treatment (INCAT) scale and the Inflammatory Rasch-built Overall Disability Scale (I-RODS).
Secondary goals include:
Assessing how smartwatch data relate to patients' quality of life and sleep patterns.
Comparing smartwatch results with other clinical scores such as muscle strength (MRC sum score) and grip strength.
Evaluating how well patients can use the smartwatch over the long term during home treatment.
An additional exploratory goal is to see whether smartwatch data can detect early signs of worsening disease before symptoms appear.
Study Design:
This is a 12-month observational study with five main clinic visits (at 0, 3, 6, 9, and 12 months). After enrolling, participants will begin or continue subcutaneous immunoglobulin therapy as decided by their treating physician. Each visit includes standard clinical assessments and questionnaires.
Participants will receive a smartwatch at the start of the study to continuously track their activity and sleep patterns. A follow-up phone call one week later will check that the device is working properly.
Duration:
Recruitment will last about 6 months, and each participant will be followed for 12 months.
Why This Matters:
By combining established clinical measures with continuous digital monitoring, the S.T.E.P.S. study may help improve understanding of disease activity and treatment response in CIDP. This could lead to more personalized therapy schedules and better long-term care for patients.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed typical or possible typical CIDP according to the 2021 EAN/PNS criteria
- •Age ≥18 years
- •Ability to use a smartwatch as decided by the investigator
- •switched from IVIG to fSCIG within the prior 6 months or plan to switch during study recruitment phase
- •on investigator-confirmed stable IVIG therapy pre-switch
排除标准
- •Age < 18 years
- •Inability to operate smartwatch or smartphone device
- •current pregnancy and breastfeeding status
- •CIDP variants according to the 2021 EAN/PNS criteria
研究组 & 干预措施
CIDP patients
CIDP patients (≥18 years) with typical or possible typical CIDP (2021 EAN/PNS criteria), Switched from IVIG to fSCIG within 6 months or during recruitment, Must be able to use smartwatch + smartphone, Exclude: CIDP variants, pregnancy, breastfeeding, inability to use devices
结局指标
主要结局
Change in Inflammatory Rasch-built Overall Disability Scale (I-RODS)
时间窗: Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12)
24-item scale, with each item representing a common, daily activity minimum value: 0 points, maximum value: 48 points, best result: 48 points
Change in Inflammatory Neuropathy Cause and Treatment (INCAT) disability score
时间窗: Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12)
minimum value: 0 points, maximum value: 10 points, best result: 0 points
Longitudinal development of activity parameter: duration of moderate activity (seconds) defined by Withings
时间窗: 365 days
Examples for moderate activity: Walking fast, Cleaning (vacuuming, washing windows, etc.), Playing doubles tennis or badminton, Taking a bike ride, Gardening
Change in Inflammatory Rasch-built Overall Disability Scale (I-RODS)
时间窗: Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12)
24-item scale, with each item representing a common, daily activity minimum value: 0 points, maximum value: 48 points, best result: 48 points
Change in Inflammatory Neuropathy Cause and Treatment (INCAT) disability score
时间窗: Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12)
minimum value: 0 points, maximum value: 10 points, best result: 0 points
Longitudinal development of activity parameter: step count
时间窗: 365 days
Longitudinal development of activity parameter: duration of moderate activity (seconds) defined by Withings
时间窗: 365 days
Examples for moderate activity: Walking fast, Cleaning (vacuuming, washing windows, etc.), Playing doubles tennis or badminton, Taking a bike ride, Gardening
次要结局
- Longitudinal development of activity parameter: sum of all active time (seconds)(365 days)
- Longitudinal development of cardiovascular parameter: maximal heartrate(365 days)
- Change in Grip strength(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Change in Medical Research Council (MRC)-Sumscore(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Wearing time of smartwatch (daily)(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Longitudinal development of activity parameter: approximate calories burned(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Change in Subjective occurrence of end-of-dose phenomena/wearing off(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Change in Quality of life (QoL)(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Change in Pittsburgh Sleep Quality Index (PSQI)(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Change in Blood analysis (levels of sNfl, serum proteomics)(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Questionnaire about smartwatch usage(End of study visit (after 12 months, V5))
- Longitudinal development of sleep parameter: time awake (seconds)(365 days)
- Longitudinal development of sleep parameter: number of times user woke up(365 days)
- Longitudinal development of sleep parameter: total time asleep (seconds)(365 days)
- Longitudinal development of sleep parameter: WASO(365 days)
- Longitudinal development of sleep parameter: time spent in bed before falling asleep (seconds)(365 days)
- Longitudinal development of sleep parameter: time awake after first falling asleep (seconds)(365 days)
- Longitudinal development of sleep parameter: Withings Sleep score(365 days)
- Longitudinal development of cardiovascular parameter: minimum heartrate(365)
- Change in Grip strength(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Change in Medical Research Council (MRC)-Sumscore(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Longitudinal development of activity parameter: approximate distance traveled (meter)(365 days)
- Longitudinal development of activity parameter: duration of soft activity (seconds) defined by Withings(365 days)
- Longitudinal development of activity parameter: duration of intense activity (seconds) defined by Withings(365 days)
- Longitudinal development of activity parameter: sum of all active time (seconds)(365 days)
- Wearing time of smartwatch (daily)(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Longitudinal development of activity parameter: approximate calories burned(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Change in Subjective occurrence of end-of-dose phenomena/wearing off(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Change in Quality of life (QoL)(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Change in Pittsburgh Sleep Quality Index (PSQI)(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Change in Blood analysis (levels of sNfl, serum proteomics)(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
- Questionnaire about smartwatch usage(End of study visit (after 12 months, V5))
- Longitudinal development of sleep parameter: time awake (seconds)(365 days)
- Longitudinal development of sleep parameter: number of times user woke up(365 days)
- Longitudinal development of sleep parameter: time to sleep (seconds)(365 days)
- Longitudinal development of sleep parameter: total time in bed (seconds)(365 days)
- Longitudinal development of sleep parameter: total time asleep (seconds)(365 days)
- Longitudinal development of sleep parameter: WASO(365 days)
- Longitudinal development of sleep parameter: time spent in bed before falling asleep (seconds)(365 days)
- Longitudinal development of cardiovascular parameter: average heartrate(365 days)
- Longitudinal development of sleep parameter: time awake after first falling asleep (seconds)(365 days)
- Longitudinal development of sleep parameter: Withings Sleep score(365 days)
- Longitudinal development of cardiovascular parameter: maximal heartrate(365 days)
- Longitudinal development of cardiovascular parameter: minimum heartrate(365)
- Longitudinal development of cardiovascular parameter: time in light heartrate zone (seconds)(365 days)
- Longitudinal development of cardiovascular parameter: time in moderate heartrate zone (seconds)(365 days)
- Longitudinal development of cardiovascular parameter: time in intense heartrate zone (seconds)(365 days)
- Longitudinal development of cardiovascular parameter: time in maximal heartrate zone (seconds)(365 days)
- Number and time of Irregular 1-channel ECGs (according to Withings algorithm)(365 days)
- Change in Fatigue Severity Scale (FSS)(Baseline up to 12 months, 5 visits (months 0, 3, 6, 9, and 12))
研究者
Dr. med. Marc Günter Pawlitzki
Study coordinator / PI
Heinrich-Heine University, Duesseldorf