Study of Efficacy and Safety of Privigen in Subjects With Chronic Inflammatory Demyelinating Polyneuropathy

Condition(s) targeted: Chronic Inflammatory Demyelinating Polyneuropathy

Intervention: 10% liquid formulation of human immunoglobulin (Biological)

Phase: Phase 3

Status: Completed

Sponsored by: CSL Behring

Official(s) and/or principal investigator(s):
Program Director Clinical R&D, Study Director, Affiliation: CSL Behring

The objective of this study is to demonstrate the efficacy and safety of Privigen in subjects with CIDP.

Official title: A Single-arm Study to Demonstrate the Efficacy and Safety of Privigen in the Treatment of Subjects With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Responder Rate

Secondary outcome:

Change in Adjusted INCAT Score

Change in Maximum Grip Strength

Change in Medical Research Council Sum Scale (MRC)

Immunoglobulin G (IgG) Level

Frequency of Adverse Events (AEs)

Severity of AEs Per Infusion

Severity of AEs Per Subject

Relatedness of AEs Per Infusion

Relatedness of AEs Per Subject

Mean Change in Systolic and Diastolic Blood Pressure During Infusion

Mean Change in Pulse Rate During Infusion

Mean Change in Body Temperature During Infusion

Number of Subjects With Normal/Abnormal Not Clinically Significant (ANCS) Value at Baseline Changing to Abnormal Clinically Significant (ACS) Value at Completion Visit in Routine Laboratory Parameters.

Minimum age: 18 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

Inclusion Criteria: IVIG-untreated subjects:

- Either subjects with newly diagnosed CIDP (developing over at least 2 months) or

subjects with an IVIG treatment interruption for at least 1 year with a progressive disease (deteriorating in the last 2 months) prior to enrolment.

- Actual diagnosis (including electrophysiology) of CIDP with progressive or relapsing

dysfunction from motor and sensory or symmetric motor nerve only in at least 1 limb resulting from neuropathy. Criteria for definite or probable CIDP according to EFNS/PNS guideline.

- Age ≥18 years.

- Male or female.

- Written informed consent for study participation obtained before undergoing any study

specific procedures. IVIG-pretreated subjects:

- Being treated regularly with IVIG on a fixed cycle length of 2 to 6 weeks ± 5 days in

the last 6 months, on a fixed dosage of ± 20 % in the last 6 months and deteriorating by at least 1 INCAT score point during the Washout Period of up to 10 weeks (except for an increase from 0 to 1 solely due to upper limb score).

- Historic diagnosis of CIDP with progressive or relapsing dysfunction from motor and

sensory or symmetric motor nerve only in at least 1 limb resulting from neuropathy. Criteria for definite or probable CIDP according to EFNS/PNS guideline.

- Age ≥18 years.

- Male or female.

- Written informed consent for study participation obtained before undergoing any study

specific procedures. Exclusion Criteria:

- A motor syndrome that fulfils criteria for multifocal motor neuropathy (MMN) with

conduction block (i. e., upper limb motor weakness without sensory deficit and with a 50% decrease in action potential amplitude or area on proximal compared with distal stimulation in motor nerves).

- CIDP with monoclonal gammopathy of uncertain significance (CIDP-MGUS) with anti-MGUS

antibodies and patients with distal acquired demyelinating symmetric (DADS)neuropathy.

- Any disease (mainly neurological or chronic orthopedic) that may cause symptoms or

may interfere with treatment or outcome assessments with the INCAT (e. g., diphtheria, drug or toxin exposure and diabetes mellitus likely to have caused the neuropathy, IgM paraproteinemia, familial neuropathy, borreliosis with radiculopathy, post-polio-syndrome,M. Parkinson, stroke).

- Current malignancy.

- History of cardiac insufficiency (New York Heart Association [NYHA] III/IV),

cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease, congestive heart failure or severe hypertension.

- History of thrombotic episodes (deep vein thrombosis, myocardial infarction,

cerebrovascular accident).

- Migraine associated with IVIG infusion in the last 3 months prior to enrolment.

- Known allergic or other severe reactions to blood products including intolerability

to previous IVIG (i. e. severe headache, hypersensitivity, intravascular hemolysis).

- Subjects with serum IgA level less than 50% of the lower normal limit.

- Known hyperprolinemia.

- Any condition (including alcohol, drug or medication abuse) that is likely to

interfere with evaluation of the study product or satisfactory conduct of the study.

- Plasma exchange 3 months prior to enrolment.

- Treatment with immunomodulatory agents others than steroids, methotrexate or

azathioprine (e. g. interferon, TNF-α inhibitors) within 6 months before enrolment.

- Treatment with rituximab in the 12 months before enrolment.

- Abnormal laboratory parameters: creatinine > 1. 5 times the upper normal limit (UNL),

lactate dehydrogenase (LDH) > 1. 5 times the UNL, C-reactive protein (CRP) > 1. 5 times the UNL, hemoglobin (Hb) < 10 g/dL.

- Ongoing HIV, hepatitis C and hepatitis B infection.

- Participation in another clinical study (or use of another investigational medicinal

product [IMP]) within 3 months prior to enrolment

- Not able to comply with study procedures and treatment regimen.

- Employee at the study site, or spouse/partner or relative of any study staff (e. g.,

investigator, sub-investigators, or study nurse).

- Pregnancy or nursing mother.

- Intention to become pregnant during the course of the study.

- Female subjects of childbearing potential either not using, or not willing to use, a

medically reliable method of contraception for the entire duration of the study, or not sexually abstinent for the entire duration of the study, or not surgically sterile.

Study Site, Bruxelles, Belgium

Study Site, Edegem, Belgium

Study Site, Gent, Belgium

Study Site, Leuven, Belgium

Study Site, Helsinki, Finland

Study Site, Turku, Finland

Study Site, Vaasa, Finland

Study Site, Limoges, France

Study Site, Lyon, France

Study Site, Marseille, France

Study Site, Montpellier, France

Study Site, Paris, France

Study Site, Berlin, Germany

Study Site, Feldberger Seenlandschaft, Germany

Study Site, Göttingen, Germany

Study Site, Itzehoe, Germany

Study Site, Prien, Germany

Study Site, Schwedt, Germany

Study Site, Würzburg, Germany

Study Site, Krakow, Poland

Study Site, Lublin, Poland

Study Site, Wroclaw, Poland

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Starting date: November 2010
Last updated: August 27, 2013