Efficacy and Safety Study of I10E in Treatment of Patients With CIDP (PRISM)

An International, Multicentre, Efficacy and Safety Study of I10E in Initial and Maintenance Treatment of Patients With Chronic Inflammatory Demyelinating Polyradiculoneuropathy.

Primary objective:

To assess the efficacy of I10E in improving the disability of patients with CIDP.

Secondary objective:

To assess the safety of I10E in patients with CIDP.

Study Overview

• Status: Completed
• Conditions: Chronic Inflammatory Demyelinating Polyradiculoneuropathy
• Intervention / Treatment: Drug: I10E

• Study Type: Interventional
• Enrollment (Actual): 44
• Phase: Phase 3

Contacts and Locations

Study Locations

• France
  • Bordeaux, France
    • CHU de Bordeaux - Hôpital Pellegrin
  • Dijon, France
    • Hôpital général du CHU de Dijon
  • Nice, France
    • CHU de Nice - Hopital L'Archet
  • Paris, France
    • CHU Paris - Hôpital Pitié Salpétrière
  • Rennes, France
    • Hopital Pontchaillou
  • Saint Etienne, France
    • CHU de Saint Etienne - Hôpital Nord
  • Strasbourg, France
    • Hôpital de HAUTEPIERRE
• Italy
  • Genova, Italy
    • IRRCS Azienda Ospedaliera Universitaria
  • Milano, Italy
    • IRCCS - Istituto Clinico Humanitas
  • Milano, Italy
    • IRRCS Istituto Nazionale Neurologico Besta
  • Milano, Italy
    • Ospedale San Raffaele IRCCS
  • Padova, Italy
    • Azienda Ospedaliere Universitaria di Padova
  • Roma, Italy
    • Universita Cattolica del Sacro Cuore
  • Torino, Italy
    • Azienda Ospedaliere Universitaria san Giovanni
• Spain
  • Barcelona, Spain
    • Hospital de La Santa Creu I Sant Pau
  • Madrid, Spain, 28223
    • Hospital Quiron Madrid
  • Madrid, Spain
    • Hospital General Universitario Gregorio
  • Santiago de Compostela, Spain
    • Hospital Clinico Universitario de Santiago
  • Seville, Spain
    • Hospital Universitario Virgen del Rocío
  • Valencia, Spain
    • Hospital Universitario i Politècnico La Fe
• Tunisia
  • Manouba, Tunisia
    • Hôpital Razi, La Manouba
  • Monastir, Tunisia
    • Hôpital Fattouma Bourguiba
  • Sfax, Tunisia
    • Hôpital Habib Bourguiba
  • Sousse, Tunisia
    • Hôpital Sahloul
  • Tunis, Tunisia
    • Hopital Militaire de Tunis
• Turkey
  • Ankara, Turkey
    • Ankara university medical school Neurology
  • Ankara, Turkey
    • Hacettepe University medical School Neurology
  • Bursa, Turkey
    • Uludag University Medical School Neurology
  • Istanbul, Turkey
    • Istanbul UniversityCerrahpasa Medical School Neurology
  • Istanbul, Turkey
    • Marmara Universitesi Egitim Ve Arastirma Hastanesi
• United Kingdom
  • London, United Kingdom, SW17OQT
    • St Georges
  • Southampton, United Kingdom
    • Southampton General Hospital
  • Stratford-upon-Avon, United Kingdom
    • University Hospital of North Straffordshire

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. Male or female patient aged 18 years or more
2. Definite or probable CIDP according to the European Federation of Neurological Societies (EFNS)/Peripheral Nerve Society (PNS) guidelines 2010 clinical and neurophysiological criteria Pure motor CIDP, provided that a diagnosis of multifocal motor neuropathy has been ruled out CIDP associated with monoclonal gammopathy of undetermined significance (MGUS), provided that anti-MAG antibodies titer is lower than the used technique's negativity threshold (1000 BTU for Bühlmann ELISA technique) Lewis-Sumner syndrome
3. Score of at least 2 on the adjusted INCAT disability scale

4. Patient who either :

   1. has never been previously treated with Ig (Ig-naive patient) Or
   2. was previously treated with Ig but is in clinical relapse following treatment withdrawal. In the latter case, the last Ig course shall have been administered no less than 3 months prior to screening

Exclusion Criteria:

1. History of IgA deficiency, unless the absence of anti-IgA antibodies has been documented
2. History of cardiac insufficiency (New York Heart Association [NYHA] III/IV), uncontrolled cardiac arrhythmia, unstable ischemic heart disease, or uncontrolled hypertension
3. History of venous thrombo-embolic disease, myocardial infarction or, cerebrovascular accident
4. Risk factor for blood hyperviscosity such as cryoglobulinemia or haematologic malignancy with monoclonal gammopathy
5. Body mass Index (BMI) ≥40 kg/m²
6. Glomerular filtration rate <80 mL/min/1.73m² measured according to the Modified Diet in Renal Disease (MDRD) calculation
7. Any other ongoing disease that may cause chronic peripheral neuropathy, such as toxin exposure, dietary deficiency, uncontrolled diabetes, hyperthyroidism, cancer, systemic lupus erythematosus or other connective tissue diseases, infection with HIV, hepatitis B virus (HBV) or hepatitis C virus (HCV), Lyme disease, multiple myeloma, Waldenström's macroglobulinaemia, amyloid, and hereditary neuropathy
8. Woman with positive results on a urine pregnancy test or breastfeeding woman or woman of childbearing potential without an effective contraception.
9. Any other serious medical condition that would interfere with the clinical assessment of CIDP or use of I10E or prevent the patient from complying with the protocol requirements
10. Increasing dosage or introduction of a corticotherapy within the last 3 months prior to screening, with oral or systemic corticosteroids at a dose higher than 10 mg daily prednisolone or equivalent. Topical corticosteroids are permitted
11. Treatment within 12 months prior to screening with immunomodulatory or immunosuppressant agents (including but not limited to cyclophosphamide, cyclosporine, interferon-alfa, interferon-beta1a, anti-CD20, alemtuzumab, aziathioprine, etanercept, mycophenolate mofetil, methotrexate and haemopoetic stem cell transplantation)
12. Plasma exchange, blood products or derivatives administered within the last 3 months prior to screening
13. Administration of another investigational product within the last month prior to screening

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: I10E Arm	Drug: I10E; Patients who meet all eligibility criteria will receive one dose of IMP at 2g/kg over 2-5 days followed by 7 doses of IMP at 1g/kg over 1-2 day(s), every 3 weeks. Duration of treatment period: 21 weeks +/- 7 days.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy Endpoint: Responder Rate at End of Study	Responders were defined as patients with a decrease ≥1 point in the adjusted INCAT disability score compared to baseline. Adjusted INCAT disability score can vary from 0 (normal) to 9 (maximal disability). If a patient was treated with a not-allowed treatment during the study period, then all adjusted INCAT disability score measured after the intake of these not-allowed treatments were censored. If the score at EoS visit was missing, then the Last Observation Carried Forward (LOCF) approach was applied to replace this missing value.	24 weeks after first treament injection

Collaborators and Investigators

• Sponsor: Laboratoire français de Fractionnement et de Biotechnologies
• Investigators: Principal Investigator: Eduardo NOBILE-ORAZIO, MD, IRCCS Instituto Clinico Humanitas, Milano, Italy

Study record dates

Study Major Dates

• Study Start: May 1, 2015
• Primary Completion (Actual): September 29, 2017
• Study Completion (Actual): September 29, 2017

Study Registration Dates

• First Submitted: November 13, 2014
• First Submitted That Met QC Criteria: November 17, 2014
• First Posted (Estimate): November 18, 2014

Study Record Updates

• Last Update Posted (Actual): January 27, 2021
• Last Update Submitted That Met QC Criteria: January 5, 2021
• Last Verified: January 1, 2021

More Information

Terms related to this study

• Keywords: Peripheral neuropathy; Rare disease; Disimmune disease; Neurology Chronic disease
• Additional Relevant MeSH Terms: Nervous System Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Neuromuscular Diseases; Peripheral Nervous System Diseases; Polyneuropathies; Polyradiculoneuropathy; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
• Other Study ID Numbers: I10E-1302