A Global Real-World Study to Assess HyQvia Use and Outcomes in Patients With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) Switching to HyQvia
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 30
- 试验地点
- 15
- 主要终点
- Treatment Preference for CIDP Therapy as Measured by Novel Treatment Preference Questionnaires
研究概览
简要总结
The main aims of this study are to understand why adults with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) chose a certain treatment, why they changed to HyQvia from another therapy, how satisfied they are with HyQvia and their previous treatment, how their work productivity and activity is impacted and learn about their CIDP signs and symptoms. Other aims are to collect information on any medical problems or side effects during the treatment with HyQvia, learn how effective treatment of CIDP with HyQvia is and understand details on the use of HyQvia in standard clinical routine as well on the need for healthcare intervention (such as emergency room visits or hospital visits or stays).
During the study, data will be collected from medical records already available, interviews with participants at study start and study completion and via questionnaires completed by participants.
Participants will be treated as per the doctor's or the clinic's routine.
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
- •Participants aged 18 years or older.
- •Investigator-confirmed documented diagnosis of CIDP of any type (e.g., typical, multifocal, focal, motor, sensory, or distal) according to the revised European Academy of Neurology and Peripheral Nerve Society (EAN/PNS) 2021 guidelines on the diagnosis and treatment of CIDP.
- •Participants must currently have a stable disease course (as per physician judgment) for at least 12 weeks.
- •Is considered a new user or current user of HyQvia-
- •New users (Cohort A) are participants on a CIDP treatment as of the date of enrollment, who intend to switch to HyQvia within 6 weeks after enrollment.
- •Current users (Cohort B) are participants on HyQvia as of the date of enrollment, who switched to HyQvia within the 6 weeks preceding the date of enrollment (the 6-week window may be extended depending on the recruitment progress).
- •Has provided written informed consent (for the main study).
- •Participant is willing to comply with the protocol requirement of non-standard of care (non-SOC) assessments (e.g., adjusted inflammatory neuropathy cause and treatment (INCAT) assessment).
- •Participants must be able to communicate fluently in their local language (if they participate in the optional qualitative participant interview [sub-study].
排除标准
- •Had any HyQvia use, defined as
- •any record of HyQvia use prior to enrollment for new users of HyQvia (Cohort A), and
- •any record of HyQvia use more than 6 weeks preceding the enrollment date (the 6-week window may be extended depending on the recruitment progress) for current users of HyQvia (Cohort B).
- •Without any prior treatment for CIDP.
- •Pregnant or breastfeeding.
- •Participants with known hypersensitivity to any component of HyQvia.
- •Has participated in an interventional clinical study within 30 days prior to enrollment or was scheduled to participate in an interventional clinical study during this study.
- •Has had major surgery within 12 weeks prior to enrollment, or has surgery planned during the time the participant is expected to participate in the study. Note: Participants with planned surgical procedures to be conducted under local anesthesia may participate. Kyphoplasty or vertebroplasty are not considered major surgeries.
- •Received induction treatment for CIDP during the past 12 weeks.
- •Participant is identified by the investigator as being unable or unwilling to cooperate with the study procedures.
- •(Cohort A) only: Participants who do not initiate HyQvia treatment within 6 weeks after enrollment.
结局指标
主要结局
Treatment Preference for CIDP Therapy as Measured by Novel Treatment Preference Questionnaires
时间窗: Baseline up to 12 months (HyQvia or study discontinuation)
The novel treatment preference questionnaires measure participant's treatment preferences for switching to HyQvia from other subcutaneous immunoglobulin/intravenous immunoglobulin (SCIG/IVIG) treatments for CIDP. It includes overall preference for switching from prior therapy to HyQvia, reason for switching from prior therapy type (like other SCIG, IVIG, corticosteroids, or plasma exchange) to HyQvia for the maintenance treatment of CIDP, current/future preference for HyQvia, reason for HyQvia or study discontinuation, and details on previous treatment setting/components for other subcutaneous immunoglobulin (SCIG) or intravenous immunoglobulin (IVIG) user only. Users will be presented a maximum of 8 questions at baseline, 2 questions at Month 12, 2 questions if HyQvia is discontinued, and 4 questions if the study is discontinued. The questionnaires will be self-administered.
Treatment Satisfaction With HyQvia as Measured by the Treatment Satisfaction Questionnaire for Medication (TSQM) Short Form-9
时间窗: Baseline up to 12 months
The TSQM is a 9-item questionnaire which assesses domains that include effectiveness, convenience, and global satisfaction. The instrument is self-administered. Response options fall on a 5- or 7-point Likert/Likert-type scale with scores ranging from 0 to 100 and higher scores indicating a higher level of treatment satisfaction.
CIDP Signs, Symptoms, and Impacts, as Measured by the Inflammatory Rasch-built Overall Disability Scale (I-RODS)
时间窗: Baseline up to 12 months (HyQvia or study discontinuation)
The I-RODS is a linearly weighted scale developed to capture activity and social participation limitations in participants with Guillain-Barré syndrome and CIDP. This 24-item questionnaire assesses domains that include activity and social participation. Response options include a categorical rating scale where 0 is "Not possible to perform," 1 is "Possible with some difficulty," and 2 is "Possible, without any difficulty," with a lower score indicating more severe activity and social participation limitations.
Productivity as Measured by the Work Productivity and Activity Impairment (WPAI)
时间窗: Baseline up to 12 months
The WPAI is a 6-item questionnaire that assesses domains that include absenteeism (missing work), presenteeism (impaired productivity at work), overall work performance (combined absenteeism and presenteeism), and non-work activities (activity impairment. Outcomes as measured by the WPAI are expressed as impairment percentages i.e., 0% (best) to 100% (worst), with higher numbers indicating greater impairment and less productivity, i.e., worse outcomes.
Health-Related Quality of Life (HRQoL) as Measured by the Chronic Acquired Polyneuropathy Patient Reported Index (CAP-PRI)
时间窗: Baseline up to 12 months
The CAP-PRI is a disease-specific HRQoL measure designed to assess diabetic polyneuropathy. This 15-item questionnaire assesses various life domains including physical and social functioning, pain, and emotional well-being. Items will be scored 0 (not at all), 1 (a little bit), or 2 (a lot). Individual scores within the CAP-PRI may be summed to a total score ranging from 0 to 30 that indicates the level of disease impairment, with higher scores representing higher degrees of impairment.
次要结局
- Change from Baseline in Medical Research Council (MRC) Score(Baseline up to 12 months)
- Number of Participants With Serious Adverse Events (SAEs) and All Non-serious Adverse Events (AEs) Causally or Temporally Relationship to HyQvia Treatment(Baseline up to 12 months)
- Change From Baseline in Hand Grip Strength(Baseline up to 12 months)
- Treatment Utilization of HyQvia(Baseline, Month 3, Month 6, Month 9, and Month 12 (as available))
- Number of Participants With Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Increase of >=1 point Relative to the Baseline(Baseline up to 12 months)
- Change From Baseline in Adjusted INCAT Disability Score(Baseline up to 12 months)
- Change From Baseline in I-RODS Centile Score(Baseline up to 12 months)
- Healthcare Resource Utilization (HCRU)(Baseline, Month 3, Month 6, Month 9, and Month 12 (as available))