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Safety and Efficacy of Avonex in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

Primary Purpose

Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Interferon Beta-1a
Sponsored by
Biogen
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Chronic Inflammatory Demyelinating Polyradiculoneuropathy focused on measuring Chronic Inflammatory Demyelinating Polyradiculoneuropathy, CIDP, IVIg, AVONEX

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Written informed consent prior to any testing under this protocol Must be between 18 and 75 years of age Have a diagnosis of CIDP as determined by a board-certified or board-eligible neurologist, or equivalent, for at least 6 months, including fulfilling modified INCAT neurophysiological criteria for CIDP,CIDP motor deficit responsive to IVIg and alternative EP data that justifies subject inclusion, and/or supportive pathologic or laboratory data that supports the diagnosis of CIDP Documentation in the medical record prior to screening that the CIDP had been associated with loss of muscle strength, such that the MRC sum score was less than or equal to 58. Documentation in the medical record that the patient benefited fom IVIg treatment (patient had a 2-point change or equivalent in 60-point MRC sum score) Tested for IgM monoclonal gammopathy and found to have tested negative for IgM monoclonal gammopathy or if positive for IgM monoclonal gammopathy, then are MAG antibody-negative and proven to be IVI g responsive per protocol. Must have been clinically stable while on a constant regimen of IVIg (every 2-weeks, 3-weeks, 4-weeks or 5-weeks) in the 3 months prior to screening. Exclusion Criteria*: Associated systemic disorder that might cause neuropathy. History of, or abnormal laboratory results indicative of any significant major disease or known drug hypersensitivity that, in the opinion of the investigator, would preclude the administration if IFN-beta or participation in this study. Subjects with diabetes mellitus will not be eligible, unless they satisfy both of the following requirements: a) their diabetes is well-controlled, with no retinopathy or nephropathy, having been identified during the ongoing care of their diabetes; and b) they have a normal sensory nerve action potential (SNAP) amplitude recorded in the sural nerve on at least one side of the body identified during electrophysiology (EP) testing documented in their medical record. Abnormal screening or baseline blood tests that the investigator deems clinically significant History of a seizure disorder prior to baseline (Visit 1, Week 0). History of suicidal ideation within 3 months prior to Baseline Visit (Week 0) or an episode of severe depression within 3 months prior to Baseline Visit (Week 0). Pure motor syndrome fulfilling criteria for multifocal motor neuropathy with conduction block. Pure sensory CIDP, or any other variant of CIDP without motor involvement Serious local infection or systemic infection within the 6 months prior to Screening. Use of IFN-beta at any time, use of plasma exchange, plasmapheresis, or any other immunosuppressant (with the exception of oral or non-systemic corticosteroids) within 6 months prior to Screening. History of intolerance to acetaminophen (paracetamol), ibuprofen, naproxen, and/or aspirin that would preclude use of at least one of these during the study. For female subjects, unless postmenopausal or surgically sterile, unwillingness to practice effective contraception, as defined by the Investigator, during the study. Female subjects considering becoming pregnant while in the study Female subjects who are currently pregnant or breast-feeding. This list is not exhaustive and there may be additional exclusions

Sites / Locations

  • Phoenix Neurological Associates, Ltd.
  • Neuromuscular Research Center
  • University of Florida, Jacksonville
  • University of Miami
  • University of Kansas
  • Louisiana State University
  • Harvard University/MGH
  • Tufts University/ St. Elizabeths
  • University of Minnesota
  • Weill Medical College of Cornell University
  • Raleigh Neurology Associates
  • University of Texas Southwestern
  • University of Utah
  • Liverpool Hospital
  • Institute of Clinical Neurosciences
  • St. Vincent's Hospital
  • Royal Melbourne Hospital
  • London Health Sciences Center
  • Montreal Neurological Hospital
  • Guy's Hospital/Dept. of Neuroimmunology

Outcomes

Primary Outcome Measures

The primary endpoint for the efficacy analyses is the total IVIg dose (g/Kg) administered after Visit 5 and through Visit 9 (Week 32, End of Study).

Secondary Outcome Measures

The time to disease progression.
Percentage reduction in IVIg dose (g/Kg).
The number of days between Visit 5 and either disease progression or Visit 9
(Week 32, End of Study).
The proportion of subjects with disease progression at Visit 9 (Week 32, End of Study).
The change in MRC sum score from baseline to the time of IVIg withdrawal.
Change from baseline to Visit 5 and to Visit 9 (Week 32, End of Study) in a composite score of maximal conduction velocity.

Full Information

First Posted
December 15, 2004
Last Updated
March 4, 2010
Sponsor
Biogen
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1. Study Identification

Unique Protocol Identification Number
NCT00099489
Brief Title
Safety and Efficacy of Avonex in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
Official Title
A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Determine the Safety and Efficacy of AVONEX When Used in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
Study Type
Interventional

2. Study Status

Record Verification Date
March 2010
Overall Recruitment Status
Completed
Study Start Date
February 2004 (undefined)
Primary Completion Date
February 2006 (Actual)
Study Completion Date
February 2006 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Biogen

4. Oversight

5. Study Description

Brief Summary
The purpose of this study is to determine whether AVONEX (Interferon Beta-1a), when compared to placebo, reduces the total dose of IVIg that is administered after Visit 5 and through Visit 9 (Week 32, End of Study).
Detailed Description
Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) is an acquired peripheral neuropathy of unknown origin. The etiology is not well understood but is presumed to be immunological. Evidence for this comes from observed similarities to Guillain-Barre syndrome and from the favorable response with immunomodulatory treatments. CIDP is a peripheral nervous system demyelinating neuropathy that is sometimes a corollary disorder to the central nervous system demyelination of multiple sclerosis (MS. The precise mechanisms underlying the pathogenesis are uncertain, but a number of those mechanisms support a potential role for immunomodulatory treatments such as interferon beta (e.g., Biogen Idec Inc.'s AVONEX). The rationale for the use of AVONEX in CIDP derives from observations on the pathogenesis of the condition and its similarities to MS, the mechanism of action of AVONEX, clinical trials that have been performed in CIDP that support a role for IFN-beta, and the unmet need that currently exists because of availability and safety issues with existing therapies. This Phase 2b study is a dose-ranging study designed to provide scientific evidence regarding the safety and efficacy of AVONEX in CIDP. In addition, the study aims to demonstrate the responsiveness and clinical relevance of changes in the MRC sum score and ODSS in CIDP patients.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Keywords
Chronic Inflammatory Demyelinating Polyradiculoneuropathy, CIDP, IVIg, AVONEX

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
Double
Allocation
Randomized
Enrollment
67 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Interferon Beta-1a
Primary Outcome Measure Information:
Title
The primary endpoint for the efficacy analyses is the total IVIg dose (g/Kg) administered after Visit 5 and through Visit 9 (Week 32, End of Study).
Secondary Outcome Measure Information:
Title
The time to disease progression.
Title
Percentage reduction in IVIg dose (g/Kg).
Title
The number of days between Visit 5 and either disease progression or Visit 9
Title
(Week 32, End of Study).
Title
The proportion of subjects with disease progression at Visit 9 (Week 32, End of Study).
Title
The change in MRC sum score from baseline to the time of IVIg withdrawal.
Title
Change from baseline to Visit 5 and to Visit 9 (Week 32, End of Study) in a composite score of maximal conduction velocity.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Written informed consent prior to any testing under this protocol Must be between 18 and 75 years of age Have a diagnosis of CIDP as determined by a board-certified or board-eligible neurologist, or equivalent, for at least 6 months, including fulfilling modified INCAT neurophysiological criteria for CIDP,CIDP motor deficit responsive to IVIg and alternative EP data that justifies subject inclusion, and/or supportive pathologic or laboratory data that supports the diagnosis of CIDP Documentation in the medical record prior to screening that the CIDP had been associated with loss of muscle strength, such that the MRC sum score was less than or equal to 58. Documentation in the medical record that the patient benefited fom IVIg treatment (patient had a 2-point change or equivalent in 60-point MRC sum score) Tested for IgM monoclonal gammopathy and found to have tested negative for IgM monoclonal gammopathy or if positive for IgM monoclonal gammopathy, then are MAG antibody-negative and proven to be IVI g responsive per protocol. Must have been clinically stable while on a constant regimen of IVIg (every 2-weeks, 3-weeks, 4-weeks or 5-weeks) in the 3 months prior to screening. Exclusion Criteria*: Associated systemic disorder that might cause neuropathy. History of, or abnormal laboratory results indicative of any significant major disease or known drug hypersensitivity that, in the opinion of the investigator, would preclude the administration if IFN-beta or participation in this study. Subjects with diabetes mellitus will not be eligible, unless they satisfy both of the following requirements: a) their diabetes is well-controlled, with no retinopathy or nephropathy, having been identified during the ongoing care of their diabetes; and b) they have a normal sensory nerve action potential (SNAP) amplitude recorded in the sural nerve on at least one side of the body identified during electrophysiology (EP) testing documented in their medical record. Abnormal screening or baseline blood tests that the investigator deems clinically significant History of a seizure disorder prior to baseline (Visit 1, Week 0). History of suicidal ideation within 3 months prior to Baseline Visit (Week 0) or an episode of severe depression within 3 months prior to Baseline Visit (Week 0). Pure motor syndrome fulfilling criteria for multifocal motor neuropathy with conduction block. Pure sensory CIDP, or any other variant of CIDP without motor involvement Serious local infection or systemic infection within the 6 months prior to Screening. Use of IFN-beta at any time, use of plasma exchange, plasmapheresis, or any other immunosuppressant (with the exception of oral or non-systemic corticosteroids) within 6 months prior to Screening. History of intolerance to acetaminophen (paracetamol), ibuprofen, naproxen, and/or aspirin that would preclude use of at least one of these during the study. For female subjects, unless postmenopausal or surgically sterile, unwillingness to practice effective contraception, as defined by the Investigator, during the study. Female subjects considering becoming pregnant while in the study Female subjects who are currently pregnant or breast-feeding. This list is not exhaustive and there may be additional exclusions
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Allan Ropper, MD
Organizational Affiliation
Tufts University School of Medicine, St. Elizabeth's Medical Center
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Kate Dawson, MD
Organizational Affiliation
Biogen
Official's Role
Study Director
Facility Information:
Facility Name
Phoenix Neurological Associates, Ltd.
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85006
Country
United States
Facility Name
Neuromuscular Research Center
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85258
Country
United States
Facility Name
University of Florida, Jacksonville
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32209
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
University of Kansas
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Louisiana State University
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Facility Name
Harvard University/MGH
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Tufts University/ St. Elizabeths
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02135
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Weill Medical College of Cornell University
City
New York
State/Province
New York
ZIP/Postal Code
10022
Country
United States
Facility Name
Raleigh Neurology Associates
City
Raleigh
State/Province
North Carolina
ZIP/Postal Code
27607
Country
United States
Facility Name
University of Texas Southwestern
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390-8897
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Liverpool Hospital
City
Liverpool
State/Province
New South Wales
ZIP/Postal Code
2170
Country
Australia
Facility Name
Institute of Clinical Neurosciences
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2006
Country
Australia
Facility Name
St. Vincent's Hospital
City
Fitzroy
State/Province
Victoria
ZIP/Postal Code
3065
Country
Australia
Facility Name
Royal Melbourne Hospital
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
London Health Sciences Center
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5A5
Country
Canada
Facility Name
Montreal Neurological Hospital
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H3A 2B4
Country
Canada
Facility Name
Guy's Hospital/Dept. of Neuroimmunology
City
London
ZIP/Postal Code
SE1 1UL
Country
United Kingdom

12. IPD Sharing Statement

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Safety and Efficacy of Avonex in Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

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