Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) and Treatment With Subcutaneous Immunoglobulin (IgPro20)

NCT ID: NCT01545076

Last Updated: 2018-07-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 208 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-03-31
• Study Completion Date: 2016-09-30

Brief Summary

This is a prospective, multicenter, randomized, double-blind, placebo-controlled, parallel-group 3-arm study to investigate 2 different doses of subcutaneous (SC) IgPro20 compared with placebo for maintenance treatment of patients with CIDP.

Patients who received at lease 1 dose of intravenous immunoglobulin (IVIG) within 8 weeks before screening will be assessed during 4 separate study periods. Patients first undergo a Screening Period, followed by an IgG Dependency Test Period of up to 12 weeks to test for ongoing need of IgG. Those patients experiencing CIDP relapse during this test period will be administered a standardized IVIG regimen during an IVIG Re-stabilization Period. Patients with improved and maintained adjusted inflammatory neuropathy cause and treatment scale (INCAT) in the IVIG Re-stabilization Period will continue to the SC Treatment Period of the study. Patients entering the 24 week SC Treatment Period will be randomized to receive weekly infusions of 1 of 2 IgPro20 doses (0.2 or 0.4 g/kg body weight) or placebo.

The overall study duration is up to 52 weeks. Clinical outcomes will be assessed by the Inflammatory Neuropathy Cause and Treatment (INCAT) score, maximum grip strength, the Medical Research Council (MRC) sum score, the Rasch-built Overall Disability Scale (R-ODS), and electrophysiological evaluations.

Conditions

Chronic Inflammatory Demyelinating Polyneuropathy; Polyradiculoneuropathy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

IgPro20 low dose

Group Type: EXPERIMENTAL

IgPro20 (low dose)

Intervention Type: BIOLOGICAL

20% liquid formulation (200 mg/mL) of human normal immunoglobulin for subcutaneous use administered weekly during the SC treatment period of the study according to the randomization:

0.2 g/kg body weight (low dose arm)

IgPro10

Intervention Type: BIOLOGICAL

10% Immunoglobulin G (IgG) liquid formulation of human normal immunoglobulin (Privigen®) administered intravenously during Restabilization Period of the study and/or as Rescue Therapy during SC Treatment Period for subjects with a CIDP relapse.

IgPro20 high dose

Group Type: EXPERIMENTAL

IgPro10

Intervention Type: BIOLOGICAL

10% Immunoglobulin G (IgG) liquid formulation of human normal immunoglobulin (Privigen®) administered intravenously during Restabilization Period of the study and/or as Rescue Therapy during SC Treatment Period for subjects with a CIDP relapse.

IgPro20 (high dose)

Intervention Type: BIOLOGICAL

20% liquid formulation (200 mg/mL) of human normal immunoglobulin for subcutaneous use administered weekly during the SC treatment period of the study according to the randomization:

0.4 g/kg body weight (high dose arm)

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: BIOLOGICAL

2% human albumin administered by weekly SC infusions during the SC treatment period of the study.

IgPro10

Intervention Type: BIOLOGICAL

10% Immunoglobulin G (IgG) liquid formulation of human normal immunoglobulin (Privigen®) administered intravenously during Restabilization Period of the study and/or as Rescue Therapy during SC Treatment Period for subjects with a CIDP relapse.

Interventions

IgPro20 (low dose)

20% liquid formulation (200 mg/mL) of human normal immunoglobulin for subcutaneous use administered weekly during the SC treatment period of the study according to the randomization:

0.2 g/kg body weight (low dose arm)

Intervention Type: BIOLOGICAL

Placebo

2% human albumin administered by weekly SC infusions during the SC treatment period of the study.

Intervention Type: BIOLOGICAL

IgPro10

10% Immunoglobulin G (IgG) liquid formulation of human normal immunoglobulin (Privigen®) administered intravenously during Restabilization Period of the study and/or as Rescue Therapy during SC Treatment Period for subjects with a CIDP relapse.

Intervention Type: BIOLOGICAL

IgPro20 (high dose)

20% liquid formulation (200 mg/mL) of human normal immunoglobulin for subcutaneous use administered weekly during the SC treatment period of the study according to the randomization:

0.4 g/kg body weight (high dose arm)

Intervention Type: BIOLOGICAL

Other Intervention Names

Hizentra; Hizentra

Eligibility Criteria

Inclusion Criteria

• Definite or probable CIDP according to the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) criteria 2010.
• An IVIG treatment during the last 8 weeks prior to enrollment.
• Age ≥18 years.
• Written informed consent for study participation obtained before undergoing any study-specific procedures.

Exclusion Criteria

• Any polyneuropathy of other causes
• Any other disease (mainly neurological or chronic orthopedic) that has caused neurological symptoms or may interfere with treatment or outcome assessments
• Severe diseases and conditions that are likely to interfere with evaluation of the study product or satisfactory conduct of the study
• History of thrombotic episodes within the 2 years prior to enrolment
• Known allergic or other severe reactions to blood products including intolerability to previous IVIG

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ICON Clinical Research

INDUSTRY

Sponsor Role: collaborator

CSL Behring

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Prof. Dr. Ivo N. van Schaik

Role: PRINCIPAL_INVESTIGATOR

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Locations

Site reference 8400181

Birmingham, Alabama, United States

Site reference 8400173

Phoenix, Arizona, United States

Site reference 8400172

Phoenix, Arizona, United States

Site reference 8400167

Los Angeles, California, United States

Site reference 8400077

Centennial, Colorado, United States

Site Reference 8400352

Washington D.C., District of Columbia, United States

Site reference 8400214

Miami, Florida, United States

Site reference 8400162

Chicago, Illinois, United States

Site Reference 8400247

Chicago, Illinois, United States

Site Reference 8400215

Indianapolis, Indiana, United States

Site reference 8400166

Kansas City, Kansas, United States

Site Reference 8400347

New Brunswick, New Jersey, United States

Site reference 8400169

New York, New York, United States

Site reference 8400179

New York, New York, United States

Site reference 8400182

Charlotte, North Carolina, United States

Site Reference 8400346

Durham, North Carolina, United States

Site reference 8400178

Columbus, Ohio, United States

Site reference 8400217

Oklahoma City, Oklahoma, United States

Site Reference 8400177

Nashville, Tennessee, United States

Site reference 8400164

Houston, Texas, United States

Site Reference 8400268

Charlottesville, Virginia, United States

Site Reference 8400340

Seattle, Washington, United States

Site Reference 0360017

Herston, Queensland, Australia

Site reference 0360011

Fitzroy, Victoria, Australia

Site reference 0360008

Southport, , Australia

Site reference 0560003

Leuven, , Belgium

Site Reference 1240048

Vancouver, British Columbia, Canada

Site Reference 1240051

Montreal, Quebec, Canada

Site reference 1240006

Edmonton, , Canada

Site reference 1240007

Québec, , Canada

Site reference 1240009

Toronto, , Canada

Site reference 2030002

Hradec Králové, , Czechia

Site reference 2030009

Hradec Králové, , Czechia

Site reference 2030003

Prague, , Czechia

Site Reference 2330002

Tallinn, , Estonia

Site Reference 2330003

Tallinn, , Estonia

Site reference 2460002

Helsinki, , Finland

Site reference 2500024

Clermont-Ferrand, , France

Site reference 2500013

Marseille, , France

Site reference 2500022

Nice, , France

Site reference 2500019

Pessac, , France

Site reference 2760048

Berlin, , Germany

Site reference 2760069

Berlin, , Germany

Site reference 2760072

Berlin, , Germany

Site reference 2760049

Bochum, , Germany

Site reference 2760080

Cologne, , Germany

Site reference 2760075

Düsseldorf, , Germany

Site Reference 2760094

Essen, , Germany

Site reference 2760052

Essen, , Germany

Site reference 2760036

Göttingen, , Germany

Site reference 2760053

Göttingen, , Germany

Site reference 2760054

Hanover, , Germany

Site Reference 2760113

Ibbenbueren, , Germany

Site reference 2760055

Leipzig, , Germany

Site reference 2760047

Potsdam, , Germany

Site reference 2760039

Würzburg, , Germany

Site reference 3760005

Haifa, , Israel

Site reference 3760002

Tel Aviv, , Israel

Site reference 3800026

Chieti, , Italy

Site reference 3800027

Florence, , Italy

Site reference 3800028

Genova, , Italy

Site reference 3800031

Milan, , Italy

Site reference 3800035

Roma, , Italy

Site reference 3800036

Roma, , Italy

Site reference 3800030

Rozzano, , Italy

Site reference 3800037

Torino, , Italy

Site Reference 3920040

Aomori, , Japan

Site Reference 3920042

Aomori, , Japan

Site reference 3920038

Chiba, , Japan

Site reference 3920061

Kanagawa, , Japan

Site reference 3920045

Matsumoto, , Japan

Site reference 3920058

Osaka, , Japan

Site reference 3920037

Saitama, , Japan

Site reference 3920034

Tokushima, , Japan

Site Reference 3920065

Tokyo, , Japan

Site Reference 3920062

Tokyo, , Japan

Site reference 3920032

Tokyo, , Japan

Site reference 3920035

Yamaguchi, , Japan

Site reference 5280001

Amsterdam, , Netherlands

Site reference 5280005

Maastricht, , Netherlands

Site reference 5280004

Utrecht, , Netherlands

Site Reference 6160058

Gdansk, , Poland

Site Reference 6160060

Lodz, , Poland

Site Reference 6160055

Lublin, , Poland

Site reference 7240010

Barcelona, , Spain

Site reference 7240011

Barcelona, , Spain

Site reference 7240013

Madrid, , Spain

Site reference 7240014

Madrid, , Spain

Site reference 7240016

Seville, , Spain

Site reference 8260019

London, , United Kingdom

Site Reference 8260032

Manchester, , United Kingdom

Countries

United States; Australia; Belgium; Canada; Czechia; Estonia; Finland; France; Germany; Israel; Italy; Japan; Netherlands; Poland; Spain; United Kingdom

References

van Schaik IN, Bril V, van Geloven N, Hartung HP, Lewis RA, Sobue G, Lawo JP, Praus M, Mielke O, Durn BL, Cornblath DR, Merkies ISJ; PATH study group. Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (PATH): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2018 Jan;17(1):35-46. doi: 10.1016/S1474-4422(17)30378-2. Epub 2017 Nov 6.

Reference Type: DERIVED

PMID: 29122523 (View on PubMed)

van Schaik IN, van Geloven N, Bril V, Hartung HP, Lewis RA, Sobue G, Lawo JP, Mielke O, Cornblath DR, Merkies IS; PATH study group. Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (The PATH Study): study protocol for a randomized controlled trial. Trials. 2016 Jul 25;17(1):345. doi: 10.1186/s13063-016-1466-2.

Reference Type: DERIVED

PMID: 27455854 (View on PubMed)

Other Identifiers

IgPro20_3003

Identifier Type: -

Identifier Source: org_study_id