Observational, Real-world, Digital Biomarker, and Integrated Treatment Outcomes in Chronic Inflammatory Demyelinating Polyneuropathy/Polyradiculoneuropathy (CIDP)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Sanofi
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- I-RODS score at baseline
研究概览
简要总结
This study is an observational, ambispective, descriptive, non-interventional study of people with a chronic inflammatory demyelinating polyneuropathy/polyradiculoneuropathy (CIDP) diagnosis in the United States with residual impairment, disability, or neurological deficits after at least three months of treatment with standard of care therapy. The study is expected to last two years. Enrollment is expected to continue for one year. Depending on when the participant is enrolled, a participant can be followed for between one and two years, through the end of study, approximately two years after the study starts.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
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
- •Participants are eligible to be included in the study only if all of the following criteria apply:
- •Neurologist-confirmed diagnosis of CIDP found in the medical record, with the last neurologist visit prior to enrollment containing no information that suggests this diagnosis was reversed
- •Active use of at least one of the following CIDP treatments for three months or longer, with no evidence of discontinuation of this therapy as of the last neurologist visit prior to enrollment
- •immunoglobulin
- •corticosteroids, with the exception of prednisone (or equivalent) monotherapy at 10mg or less per day
- •plasma exchange
- •efgartigimod alfa
- •azathioprine
- •mycophenolate mofetil
- •cyclosporine
- •rituximab
- •methotrexate
- •Signed informed consent
- •Residual impairment, disability, or neurological deficits at enrollment, as defined by a raw I-RODS score of 44 or below
排除标准
- •Participants are excluded from the study if any of the following criteria apply:
- •Evidence of participation in any interventional clinical trial with an investigational drug at the time of enrollment
- •Hyperreflexia (increased reflexes) recorded in the medical record during a neurological exam the year before enrollment and after CIDP diagnosis
- •Aged under 18 at the time of enrollment
- •The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
结局指标
主要结局
I-RODS score at baseline
时间窗: Baseline
The inflammatory Rasch-built overall disability scale (I-RODS) is a 24-item patient-reported questionnaire that captures disease impact on daily tasks with a score ranging from 0 (maximum disability) to 48 (no disability). It contains 24 items across an activity domain and a social participation domain. Each item is rated as "0": Not possible to perform; "1": possible, but with some difficulty, or "2" possible, without any difficulty.
Variations of I-RODS score over the study duration
时间窗: From Baseline up to End of Study (2 years)
Difference in I-RODS between baseline and: 6 months after enrollment, 12 months after enrollment, end of study. The inflammatory Rasch-built overall disability scale (I-RODS) is a 24-item patient-reported questionnaire that captures disease impact on daily tasks with a score ranging from 0 (maximum disability) to 48 (no disability). It contains 24 items across an activity domain and a social participation domain. Each item is rated as "0": Not possible to perform; "1": possible, but with some difficulty, or "2" possible, without any difficulty.
Variations of I-RODS score after treatment change
时间窗: From baseline up to End of Study (approx. 2 years)
Difference in I-RODS tests between the most recent assessments prior to and subsequent to a change in treatment class. The inflammatory Rasch-built overall disability scale (I-RODS) is a 24-item patient-reported questionnaire that captures disease impact on daily tasks with a score ranging from 0 (maximum disability) to 48 (no disability). It contains 24 items across an activity domain and a social participation domain. Each item is rated as "0": Not possible to perform; "1": possible, but with some difficulty, or "2" possible, without any difficulty.
Annualized I-RODS response rate
时间窗: Up to End of Study (approx. 2 years)
Percent of patients who experienced inflammatory Rasch-built overall disability scale (I-RODS) response over a given time period, normalized to 365 days
Annualized I-RODS relapse rate
时间窗: Up to End of Study (approx. 2 years)
Percent of patients who experienced inflammatory Rasch-built overall disability scale (I-RODS) relapse over a given time period, normalized to 365 days
次要结局
- Frequency of select comorbidities at baseline and over the study duration(From Baseline up to End of Study (approx. 2 years))
- Adjusted INCAT (aINCAT) score at baseline and its variations over the study duration and after treatment change(Baseline and throughout the study (up to 2 years))
- Annualized aINCAT response rate(Up to End of Study (approx. 2 years))
- Annualized aINCAT relapse rate(Up to End of Study (approx. 2 years))
- Interobserver variability of remote INCAT assessment(Up to End of Study (approx. 2 years))
- Incidence of select comorbidities at baseline and over the study duration(From Baseline up to End of Study (approx. 2 years))
- Laboratory measurements at baseline and over study duration(From Baseline up to End of Study (approx. 2 years))
- CAP-PRI score at baseline and its variations over the study duration and after treatment change(From Baseline to End of Study (approx 2 years))