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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02027701




Registration number
NCT02027701
Ethics application status
Date submitted
3/01/2014
Date registered
6/01/2014
Date last updated
2/10/2018

Titles & IDs
Public title
Extension Study of Maintenance Treatment With Subcutaneous Immunoglobulin (IgPro20) for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Scientific title
Multicenter, Open-label Extension Study to Investigate the Long-term Safety and Efficacy of IgPro20 in Maintenance Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) in Subjects Completing Study IgPro20_3003
Secondary ID [1] 0 0
2013-004157-24
Secondary ID [2] 0 0
IgPro20_3004
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) 0 0
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders
Neurological 0 0 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - IgPro20

Experimental: IgPro20 - 20% liquid formulation (200 mg/mL) of human normal immunoglobulin for SC use administered SC weekly: 0.2 g/kg bw (low-dose IgPro20) for up to 48 weeks. Subjects who experience CIDP relapse on 0.2 g/kg IgPro20 will have an increase to 0.4 g/kg IgPro20 immediately and will continue on high-dose until they have completed a total of 48 weeks of IgPro20 treatment.


Other interventions: IgPro20


Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Adverse Events (AEs) Per Infusion
Timepoint [1] 0 0
Up to 49 weeks
Secondary outcome [1] 0 0
Time to First CIDP Relapse - Time to first CIDP relapse based on adjusted INCAT score, using the Kaplan-Meier estimator. Relapse is defined as an increase of at least 1 INCAT score point (except for the increase from 0 to 1 in the upper limb score only).
Timepoint [1] 0 0
Up to 49 weeks
Secondary outcome [2] 0 0
Change From Baseline in CIDP Total Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Score - The INCAT score is a 10-point scale that covers the functionality of legs and arms, and has been successfully used to measure treatment effects in various CIDP studies. Scores for arm disability range from 0 ("No upper limb problems") to 5 ("Inability to use either arm for any purposeful movement"), and scores for leg disability range from 0 ("Walking not affected") to 5 ("Restricted to wheelchair, unable to stand and walk a few steps with help"). The INCAT (total) score is the sum of these 2 scores and ranges from 0 to 10. For the "adjusted" INCAT score, changes in the function of the upper limbs from 0 (normal) to 1 (minor symptoms) or from 1 to 0 were not recorded as deterioration or improvement because these changes are not considered clinically significant.
Timepoint [2] 0 0
Baseline and up to 49 weeks
Secondary outcome [3] 0 0
Change From Baseline in Medical Research Council (MRC) Score - An adapted version of the MRC sum score as published by Kleyweg and the RMC trial group was used. With the MRC sum score, the following 8 bilateral muscle pairs were assessed, and individual muscle scores as well as the sum score documented: Shoulder abduction; Elbow flexion; Wrist extension; Index finger abduction; Hip flexion; Knee extension; Foot dorsiflexion; Great toe dorsiflexion. The MRC sum score ranges from 0 (paralysis) to 80 (normal strength) points.
Timepoint [3] 0 0
Baseline and up to 49 weeks
Secondary outcome [4] 0 0
Change From Baseline in Rasch-built Overall Disability Scale (R-ODS) - The R-ODS is a recently published outcome measure that captures activity and social participation in subjects with Guillain-Barré Syndrome, CIDP, and monoclonal gammopathy of uncertain significance. The 24-item questionnaire covers a wide range of tasks of daily life that are each to be rated as "impossible to perform", "able to perform with difficulty", or "easy to perform" (scale of 0 - 2 points respectively). Items are sorted in order of increasing difficulty to perform, based on data from subjects with peripheral neuropathies (chronic inflammatory demyelinating polyneuropathy, Guillain-Barré Syndrome, or monoclonal gammopathy of uncertain significance) and subjects recruited at the university outpatient clinics of Rotterdam and Maastricht.
Timepoint [4] 0 0
Baseline and up to 49 weeks
Secondary outcome [5] 0 0
Change From Baseline in Mean Grip Strength - The hand-held Vigorimeter from Martin (Tuttlingen, Germany) is a device that measures the strength of small muscles in the hand, ie, grip strength. The subject squeezes a rubber bulb lying between the palm of the hand and the thumb and index fingers. The pressure is recorded via a rubber tube on a nanometer and expressed in kilopascal (kPa). At each assessment, the subject squeezes 3 times with each hand.
Timepoint [5] 0 0
Baseline and up to 49 weeks
Secondary outcome [6] 0 0
Percentage of Subjects With Adverse Events (AEs)
Timepoint [6] 0 0
Up to 49 weeks
Secondary outcome [7] 0 0
Number of AEs by Severity Per Infusion
Timepoint [7] 0 0
Up to 49 weeks
Secondary outcome [8] 0 0
Percentage of Subjects With AEs by Severity
Timepoint [8] 0 0
Up to 49 weeks
Secondary outcome [9] 0 0
Number of Causally Related AEs Per Infusion
Timepoint [9] 0 0
Up to 49 weeks
Secondary outcome [10] 0 0
Percentage of Subjects With Causally Related AEs
Timepoint [10] 0 0
Up to 49 weeks
Secondary outcome [11] 0 0
Number of Serious AEs Per Infusion
Timepoint [11] 0 0
Up to 49 weeks
Secondary outcome [12] 0 0
Percentage of Subjects With Serious AEs
Timepoint [12] 0 0
Up to 49 weeks

Eligibility
Key inclusion criteria
- Subjects having completed the pivotal study IgPro20_3003 (SC Week 25) or successfully
rescued from a CIDP relapse during the SC Treatment Period of pivotal study
IgPro20_3003 (NCT01545076).

- Written informed consent for study participation obtained before undergoing any
study-specific procedures.
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Subject is unable to directly transition from study IgPro20_3003.

- New medical condition and/or social behavior (ie, alcohol, drug, or medication abuse)
during participation in study IgPro20_3003 that in the judgment of the investigator
could increase risk to the subject, interfere with the evaluation of investigational
medicinal product, and/or conduct of the study.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Site Reference 0360017 - Woolloongabba
Recruitment hospital [2] 0 0
Site Reference 0360011 - Fitzroy
Recruitment postcode(s) [1] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [2] 0 0
3065 - Fitzroy
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Kansas
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
North Carolina
Country [6] 0 0
Canada
State/province [6] 0 0
Ontario
Country [7] 0 0
Canada
State/province [7] 0 0
Quebec
Country [8] 0 0
Czechia
State/province [8] 0 0
Hradec Kralove
Country [9] 0 0
France
State/province [9] 0 0
Nice Cedex 1
Country [10] 0 0
Germany
State/province [10] 0 0
Nordrhein-Westfalen
Country [11] 0 0
Germany
State/province [11] 0 0
Berlin
Country [12] 0 0
Germany
State/province [12] 0 0
Bochum
Country [13] 0 0
Germany
State/province [13] 0 0
Essen
Country [14] 0 0
Germany
State/province [14] 0 0
Hannover
Country [15] 0 0
Germany
State/province [15] 0 0
Leipzig
Country [16] 0 0
Germany
State/province [16] 0 0
Potsdam
Country [17] 0 0
Germany
State/province [17] 0 0
Wurzburg
Country [18] 0 0
Italy
State/province [18] 0 0
Milano
Country [19] 0 0
Japan
State/province [19] 0 0
Saitama
Country [20] 0 0
Japan
State/province [20] 0 0
Yamaguchi
Country [21] 0 0
Japan
State/province [21] 0 0
Chiba
Country [22] 0 0
Japan
State/province [22] 0 0
Kanagawa
Country [23] 0 0
Japan
State/province [23] 0 0
Nagoya
Country [24] 0 0
Japan
State/province [24] 0 0
Tokyo
Country [25] 0 0
Netherlands
State/province [25] 0 0
Amsterdam
Country [26] 0 0
Spain
State/province [26] 0 0
Barcelona
Country [27] 0 0
United Kingdom
State/province [27] 0 0
London
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
CSL Behring
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study is an extension study to the pivotal study IgPro20_3003 (NCT01545076). The purpose
of this extension study is to investigate the long-term treatment of CIDP with IgPro20, with
regard to safety and efficacy.

Subjects who have completed subcutaneous (SC) Week 25 or were successfully rescued from a
CIDP relapse during the SC Treatment Period of pivotal study IgPro20_3003 (NCT01545076) will
have the option to receive open-label low-dose IgPro20 (0.2 g/kg bodyweight [bw]) weekly for
up to 48 weeks. Subjects relapsing on low-dose IgPro20 will either return to high-dose
IgPro20 (0.4 g/kg) immediately or be discontinued, depending on investigator's judgment.
Subjects returning to high-dose IgPro20 will continue on high-dose until they have completed
a total of 48 weeks of IgPro20 treatment. If subjects do not successfully recover from CIDP
relapse within 4 weeks, they will be withdrawn.

The treatment duration will be up to 48 weeks, followed by a completion visit (week 49).
Trial website
https://clinicaltrials.gov/show/NCT02027701
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Prof. Dr. Ivo N. van Schaik
Address 0 0
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications