Clinical trial NCT04672733: Hizentra® in Inflammatory Neuropathies - pHeNIx Study
Official title: Hizentra® in Inflammatory Neuropathies - pHeNIx Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Chronic Inflammatory Demyelinating Polyneuropathy
Keywords: CIDP
IgPro20
Hizentra
Subcutaneous Immunoglobulins
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2020-12-17
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: French cohort of patients with CIDP

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.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* Concomitant participation in an interventional clinical study

Primary outcomes:
measure: Length of time of Continuation of treatment
timeFrame: up to 12 months
Secondary outcomes:
measure: The time between the last dose of IVIg and starting Hizentra®
timeFrame: At Baseline
measure: The total dose of the last course of IVIg
timeFrame: At Baseline
measure: The interval between courses of IVIg
timeFrame: At Baseline
measure: The total dose of the first course of Hizentra
timeFrame: At Baseline
measure: The number of days of the first course of Hizentra
timeFrame: At Baseline
measure: Number of Self-administrations or administrations by a state-registered nurse
timeFrame: Up to 12 months
measure: The daily dose of Hizentra
timeFrame: Up to 12 months
measure: The daily volume of Hizentra
timeFrame: Up to 12 months
measure: Duration of the infusion
timeFrame: Up to 12 months
measure: Number of infusion sites
timeFrame: Up to 12 months
measure: Number of Patients completing the Patient Reported Outcome (PRO) tests at home
timeFrame: Up to 12 months
measure: Rasch-built Overall Disability Scale (RODS) incapacity scale score by patient
timeFrame: Up to 12 months
measure: 10-metre walking test score by patient
timeFrame: Up to 12 months
measure: Rasch-built Overall Disability Scale (RODS) incapacity scale score by doctor
timeFrame: Up to 12 months
measure: 10-metre walking test score by doctor
timeFrame: Up to 12 months
measure: Time since the diagnosis of CIDP
timeFrame: At baseline
measure: EuroQol-5D (EQ-5D) quality of life score
timeFrame: Up to 12 months
measure: Pictorial Representation of Illness and Self Measure (PRISM) score
timeFrame: Up to 12 months
Other outcomes:
Locations:
city: Amiens
country: France
facility: CHU Amiens Picardie 1
city: Angers
country: France
facility: CHU Angers
city: Aubagne
country: France
facility: Hôpital privé de La Casamance
city: Bayonne
country: France
facility: CH Bayonne
city: Bordeaux
country: France
facility: Hôpital Pellegrin
city: Brest
country: France
facility: CHRU Brest
city: Créteil
country: France
facility: Hôpital Henri Mondor
city: Le Kremlin-Bicêtre
country: France
facility: Hôpital Bicêtre
city: Libourne
country: France
facility: CH Libourne
city: Lille
country: France
facility: Hôpital Roger Salengro, CHU Lille
city: Limoges
country: France
facility: Hôpital Dupuytren
city: Lyon
country: France
facility: Hôpital Pierre Wertheimer,HCL
city: Marseille
country: France
facility: Hôpital de la Timone
city: Montpellier
country: France
facility: CHU Montpellier
city: Nancy
country: France
facility: CHRU Nancy
city: Nantes
country: France
facility: CHU Nantes
city: Nice
country: France
facility: Hôpital Pasteur
city: Paris
country: France
facility: Hôpital Lariboisière
city: Paris
country: France
facility: La Pitié-Salpêtrière
city: Poitiers
country: France
facility: CHU Poitiers
city: Rennes
country: France
facility: Centre hospitalier privé Saint Grégoire
city: Rouen
country: France
facility: Hôpital Charles Nicolle
city: Saint-Étienne
country: France
facility: CHU Saint-Etienne
city: Strasbourg
country: France
facility: CHU de Strasbourg
city: Toulouse
country: France
facility: CHU Toulouse
city: Tours
country: France
facility: CHR Tours
city: Valence
country: France
facility: CH de Valence
Study officials:
affiliation: CSL Behring SA
name: Study Director
role: STUDY_DIRECTOR
References: