Hizentra® in Inflammatory Neuropathies - pHeNIx Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- CSL Behring
- 入组人数
- 100
- 试验地点
- 27
- 主要终点
- Length of time of Continuation of treatment
研究概览
简要总结
The pHeNIx study, a national multicentre prospective non-interventional study, should help to describe the conditions of use for Hizentra® and the methods for switching from the IV to SC route in everyday practice, together with the tolerability and efficacy of treatment, which is monitored using a patient application (PRO: Patient-Reported Outcomes).
详细描述
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is a neurological and rare type of autoimmune disorder. Intravenous immunoglobulin (IVIg) is the first-line treatment for CIDP which has been proven to be effective. For several years, published cases have suggested that the Sub-Cutaneous Ig (SCIg) may be an alternative treatment to IVIg in the treatment of CIDP. Compared to IVIg treatment, the SCIg can achieve more stable plasma IgG concentrations, suggesting a potential reduction in the dose exhaustion effect at the end of the cycle, but also fewer systemic effects. SC administration also enables more straightforward treatment to be given for ambulatory patients.
Based on the PATH study (NCT01545076), a double blind placebo-controlled, randomised, prospective, international multicentre phase III study, Hizentra® obtained an extension of its marketing authorization for the CIDP indication as maintenance treatment after stabilisation with IVIg.
However, in the "real-life" situation, the literature is still based at present on small series of patient or short-term follow-up periods.
However, the methods for switching from the IV to the SC route and the characteristics of patients receiving this treatment are not known. In addition, SCIg administration remote from a specialist centre without assistance from a health professional no longer enables a more regular assessment of the patient in terms of tolerability and efficacy.
The pHeNIx study, a national multicentre prospective non-interventional study, should help to describe the conditions of use for Hizentra® and the methods for switching from the IV to SC route in everyday practice, together with the tolerability and efficacy of treatment, which is monitored using a patient application (PRO: Patient-Reported Outcomes). The study duration is estimated to be 36 months in view of: a 24-month inclusion period and a 12-month follow-up period.
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
- •Adult patient (aged ≥18 years)
- •Patients suffering from CIDP according to EAN/PNS 2021 criteria
- •Planned switch from IVIg to Hizentra®
- •Patient treated with at least 3 courses of IV immunoglobulin and deemed by the investigator to be dependent on immunoglobulins
- •Patient deemed to be stable, with no change in their treatment for the disease during the 3 months prior to inclusion
- •Patients who have a smartphone, a tablet or a computer
- •Patients who have been informed verbally and in writing of the purposes of the study
排除标准
- •Concomitant participation in an interventional clinical study
结局指标
主要结局
Length of time of Continuation of treatment
时间窗: up to 12 months
Non-continuation is defined by: * an increase in the INCAT score of over one point measured in a consultation despite a bolus dose of IVIg and/or after increasing the dose of Hizentra® * stopping treatment with Hizentra®
次要结局
- 10-metre walking test score by patient(Up to 12 months)
- The time between the last dose of IVIg and starting Hizentra®(At Baseline)
- The total dose of the first course of Hizentra(At Baseline)
- Number of infusion sites(Up to 12 months)
- Number of Patients completing the Patient Reported Outcome (PRO) tests at home(Up to 12 months)
- Duration of the infusion(Up to 12 months)
- The total dose of the last course of IVIg(At Baseline)
- The daily volume of Hizentra(Up to 12 months)
- The interval between courses of IVIg(At Baseline)
- Number of Self-administrations or administrations by a state-registered nurse(Up to 12 months)
- The number of days of the first course of Hizentra(At Baseline)
- The daily dose of Hizentra(Up to 12 months)
- Rasch-built Overall Disability Scale (RODS) incapacity scale score by patient(Up to 12 months)
- Time since the diagnosis of CIDP(At baseline)
- EuroQol-5D (EQ-5D) quality of life score(Up to 12 months)
- Pictorial Representation of Illness and Self Measure (PRISM) score(Up to 12 months)
- Rasch-built Overall Disability Scale (RODS) incapacity scale score by doctor(Up to 12 months)
- 10-metre walking test score by doctor(Up to 12 months)