Hizentra® in Inflammatory Neuropathies - pHeNIx Study (pHeNIx)

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).

Study Overview

• Status: Recruiting
• Conditions: Chronic Inflammatory Demyelinating Polyneuropathy
• Intervention / Treatment: Biological: Hizentra

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.

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: Trial Registration Coordinator; Phone Number: +1 610-878-4697; Email: clinicaltrials@cslbehring.com

Study Locations

• France
  • Amiens, France, 80054
    • Not yet recruiting
    • CHU Amiens Picardie 1
  • Angers, France
    • Recruiting
    • Chu Angers
  • Aubagne, France
    • Recruiting
    • Hopital Privé de La Casamance
  • Bayonne, France
    • Recruiting
    • CH Bayonne
  • Bordeaux, France
    • Recruiting
    • Hopital Pellegrin
  • Brest, France
    • Recruiting
    • CHRU Brest
  • Créteil, France
    • Recruiting
    • Hôpital Henri Mondor
  • Le Kremlin-Bicêtre, France
    • Recruiting
    • Hôpital Bicêtre
  • Libourne, France
    • Recruiting
    • CH Libourne
  • Lille, France
    • Recruiting
    • Hopital Roger Salengro, CHU Lille
  • Limoges, France
    • Recruiting
    • Hopital DUPUYTREN
  • Lyon, France
    • Recruiting
    • Hôpital Pierre Wertheimer,HCL
  • Marseille, France
    • Recruiting
    • Hôpital de la Timone
  • Montpellier, France
    • Recruiting
    • CHU Montpellier
  • Nancy, France
    • Recruiting
    • CHRU Nancy
  • Nantes, France
    • Recruiting
    • Chu Nantes
  • Nice, France
    • Recruiting
    • Hôpital Pasteur
  • Paris, France
    • Recruiting
    • Hôpital Lariboisière
  • Paris, France
    • Recruiting
    • La Pitié-Salpétrière
  • Poitiers, France, 86000
    • Recruiting
    • CHU Poitiers
  • Rennes, France, 35760
    • Recruiting
    • Centre Hospitalier Privé Saint Grégoire
  • Rouen, France
    • Recruiting
    • Hôpital Charles Nicolle
  • Saint-Étienne, France
    • Recruiting
    • Chu Saint-Etienne
  • Strasbourg, France, 67091
    • Not yet recruiting
    • CHU de Strasbourg
  • Toulouse, France
    • Not yet recruiting
    • Chu Toulouse
  • Tours, France
    • Recruiting
    • CHR Tours
  • Valence, France, 26000
    • Recruiting
    • CH de Valence

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

French cohort of patients with CIDP

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Length of time of Continuation of treatment	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®	up to 12 months

Secondary Outcome Measures

Outcome Measure	Time Frame
The time between the last dose of IVIg and starting Hizentra®	At Baseline
The total dose of the last course of IVIg	At Baseline
The interval between courses of IVIg	At Baseline
The total dose of the first course of Hizentra	At Baseline
The number of days of the first course of Hizentra	At Baseline
Number of Self-administrations or administrations by a state-registered nurse	Up to 12 months
The daily dose of Hizentra	Up to 12 months
The daily volume of Hizentra	Up to 12 months
Duration of the infusion	Up to 12 months
Number of infusion sites	Up to 12 months
Number of Patients completing the Patient Reported Outcome (PRO) tests at home	Up to 12 months
Rasch-built Overall Disability Scale (RODS) incapacity scale score by patient	Up to 12 months
10-metre walking test score by patient	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
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

Collaborators and Investigators

• Sponsor: CSL Behring
• Investigators: Study Director: Study Director, CSL Behring SA

Study record dates

Study Major Dates

• Study Start (Actual): June 10, 2022
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: December 11, 2020
• First Submitted That Met QC Criteria: December 11, 2020
• First Posted (Actual): December 17, 2020

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 6, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: CIDP; IgPro20; Hizentra; Subcutaneous Immunoglobulins
• Additional Relevant MeSH Terms: Nervous System Diseases; Pathologic Processes; Neuromuscular Diseases; Chronic Disease; Disease Attributes; Autoimmune Diseases; Immune System Diseases; Peripheral Nervous System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Polyradiculoneuropathy; Polyneuropathies; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
• Other Study ID Numbers: pHeNIx study; 2019-A01803-54 (Other Identifier: ANSM)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No