A single-arm study to demonstrate the efficacy and safety of Privigen in the treatment of subjects with chronic inflammatory demyelinating polyneuropathy (CIDP)

Phase 1

• Conditions: Chronic Inflammatory Demyelinating Polyneuropathy (CIDP); MedDRA version: 13.1Level: PTClassification code 10061811Term: Demyelinating polyneuropathySystem Organ Class: 10029205 - Nervous system disorders

• Registration Number: EUCTR2009-017672-24-BE
• Lead Sponsor: CSL Behring GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-07-07
• Last Update Posted: 21 August 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Subjects meeting all of the following inclusion criteria may be enrolled into the study:
• 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
OR
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
- 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).
• 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.
- IVIG-untreated subjects need an actual diagnosis including electrophysiology.
- IVIG-pretreated subjects need a historic diagnosis.
• Age =18 years.
• Male or female.
• Written informed consent for study participation obtained before undergoing any
study specific procedures.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Subjects meeting any of the following exclusion criteria must not be enrolled into the study:
• 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
radicolopathy, 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-a 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 & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified