Hizentra® in Inflammatory Neuropathies - pHeNIx Study
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy
- Registration Number
- NCT04672733
- Lead Sponsor
- CSL Behring
- Brief Summary
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).
- Detailed Description
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 100
- 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
- Concomitant participation in an interventional clinical study
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Length of time of Continuation of treatment 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®
- Secondary Outcome Measures
Name Time Method 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 10-metre walking test score by patient 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
Trial Locations
- Locations (27)
CHU Amiens Picardie 1
🇫🇷Amiens, France
CHU Angers
🇫🇷Angers, France
Hôpital privé de La Casamance
🇫🇷Aubagne, France
CH Bayonne
🇫🇷Bayonne, France
Hôpital Pellegrin
🇫🇷Bordeaux, France
CHRU Brest
🇫🇷Brest, France
Hôpital Henri Mondor
🇫🇷Créteil, France
Hôpital Bicêtre
🇫🇷Le Kremlin-Bicêtre, France
CH Libourne
🇫🇷Libourne, France
Hôpital Roger Salengro, CHU Lille
🇫🇷Lille, France
Hôpital Dupuytren
🇫🇷Limoges, France
Hôpital Pierre Wertheimer,HCL
🇫🇷Lyon, France
Hôpital de la Timone
🇫🇷Marseille, France
CHU Montpellier
🇫🇷Montpellier, France
CHRU Nancy
🇫🇷Nancy, France
CHU Nantes
🇫🇷Nantes, France
Hôpital Pasteur
🇫🇷Nice, France
Hôpital Lariboisière
🇫🇷Paris, France
La Pitié-Salpêtrière
🇫🇷Paris, France
CHU Poitiers
🇫🇷Poitiers, France
Centre hospitalier privé Saint Grégoire
🇫🇷Rennes, France
Hôpital Charles Nicolle
🇫🇷Rouen, France
CHU Saint-Etienne
🇫🇷Saint-Étienne, France
CHU de Strasbourg
🇫🇷Strasbourg, France
CHU Toulouse
🇫🇷Toulouse, France
CHR Tours
🇫🇷Tours, France
CH de Valence
🇫🇷Valence, France