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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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)

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

Detailed Summary:
Sponsor: CSL Behring

Current Primary Outcome: Responder Rate [ Time Frame: 25 weeks ]

Percentage of responders based on the adjusted Inflammatory Neuropathy Cause and Treatment Scale (INCAT) score.

Responders were defined as those subjects who: 1) demonstrated a "clinically meaningful improvement" between baseline and Week 25, or 2) who were discontinued from the study for any reason after the start of IgPro10 treatment but with "clinically meaningful improvement" at the last study visit.

"Clinically meaningful improvement" was a decrease of at least 1 adjusted INCAT score point excluding an improvement of one point in the total score if this improvement was only due to a decrease in the upper limb score of 1 to 0.



Original Primary Outcome: Responder Rate [ Time Frame: 25 weeks ]

Percentage of responders based on the Inflammatory Neuropathy Cause and Treatment Scale (INCAT) score


Current Secondary Outcome:

  • Change in Adjusted INCAT Score [ Time Frame: Up to 34 weeks ]

    The change in INCAT score was determined at the completion visit compared to baseline and to the last measurement under the previous IVIG treatment using a non-parametric analysis to calculate the Hodges-Lehmann point estimate and the corresponding Tukey confidence interval on an exploratory basis.

    The INCAT disability score ranges from 0 to 10 and is the sum of arm and leg disability each rated between 0 and 5 (where arm = 0 indicates 'no upper limb problems' and arm = 5 indicates 'inability to use either arm for any purposeful movement', and leg = 0 indicates 'walking not affected', and leg = 5 indicates 'restricted to wheelchair, unable to stand and walk a few steps with help'). Thus, a higher INCAT disability score indicates greater disability. Negative values for change in INCAT score indicate improvement, with a more negative value indicating greater improvement compared with the value at baseline.

  • Change in Maximum Grip Strength [ Time Frame: Up to 34 weeks ]
    Change in maximum grip strength of the dominant hand. A non-parametric analysis was used to calculate the Hodges-Lehmann point estimate and the corresponding Tukey confidence interval on an exploratory basis. Positive values for change in maximum grip strength indicate improvement.
  • Change in Medical Research Council Sum Scale (MRC) [ Time Frame: Up to 34 weeks ]

    The change in MRC sum score was determined at the completion visit compared to baseline and to the last measurement under the previous IVIG treatment using a non-parametric analysis to calculate the Hodges-Lehmann point estimate and the corresponding Tukey confidence interval on an exploratory basis.

    The 80-point MRC sum score is the sum of scores for eight bilateral (left and right side) muscle groups, each rated between 0 (no visible contraction) to 5 (normal movement). A higher MRC sum score indicates greater muscle contraction/limb movement. Positive values for change in MRC sum score indicate improvement, with a more positive value indicating greater muscle contraction/ limb movement compared with the value at baseline.

  • Immunoglobulin G (IgG) Level [ Time Frame: At baseline and at Weeks 7, 13 and 19 (levels determined immediately before and after IVIG infusion), and at completion visit (Week 25) ]
  • Frequency of Adverse Events (AEs) [ Time Frame: For the duration of the study, up to 34 weeks ]
    Overall rate of AEs per infusion.
  • Severity of AEs Per Infusion [ Time Frame: For the duration of the study, up to 34 weeks ]

    The severity of each AE was to be graded by the investigator as follows:

    • Mild: Symptoms were easily tolerated and there was no interference with daily activities.
    • Moderate: Discomfort enough to cause some interference with daily activities.
    • Severe: Incapacitating with inability to work or do usual activity.
  • Severity of AEs Per Subject [ Time Frame: 34 weeks ]

    The severity of each AE was to be graded by the investigator as follows:

    • Mild: Symptoms were easily tolerated and there was no interference with daily activities.
    • Moderate: Discomfort enough to cause some interference with daily activities.
    • Severe: Incapacitating with inability to work or do usual activity.
  • Relatedness of AEs Per Infusion [ Time Frame: For the duration of the study, up to 34 weeks ]
    The causal relationship of an AE to the study drug was to be assessed and assigned by the investigator.
  • Relatedness of AEs Per Subject [ Time Frame: For the duration of the study, up to 34 weeks ]
    The causal relationship of an AE to the study drug was to be assessed and assigned by the investigator.
  • Mean Change in Systolic and Diastolic Blood Pressure During Infusion [ Time Frame: At Days 1 to 5 and at Weeks 4, 7, 10, 13, 16, 19 and 22. ]
    Systolic and diastolic blood pressure (BP) were measured before the start of IgPro10 infusion, at 30 minutes and 1 hour after the start of infusion, then every hour until the end of infusion and at 1 hour after the end of infusion. Mean changes from the pre-infusion value to each of the post-infusion values were calculated for each infusion, and the mean value and standard deviation (SD) of these individual mean changes is reported.
  • Mean Change in Pulse Rate During Infusion [ Time Frame: At Days 1 to 5 an

    Original Secondary Outcome:

    • Change in INCAT score [ Time Frame: 25 weeks ]
      Change in the INCAT score at completion visit compared to baseline and to last measurement under the previous IVIG treatment.
    • Change in maximal grip strength [ Time Frame: 25 weeks ]
      Change in maximum grip strength at completion visit compared to baseline and to last measurement under the previous IVIG treatment
    • Change in Medical Research Council Sum Scale (MRC) [ Time Frame: 25 weeks ]
      Change in MRC sum score at completion visit compared to baseline and to last measurement under the previous IVIG treatment.
    • Frequency of Adverse Events (AEs) [ Time Frame: 34 weeks ]
    • IgG level [ Time Frame: 25 weeks ]
      IgG levels at baseline and at Weeks 7, 13 and 19 (levels determined immediately before and after IVIG infusion), and at completion visit (Week 25)
    • Severity of AEs Per Infusion [ Time Frame: 34 weeks ]
    • Severity of AEs Per Subject [ Time Frame: 34 weeks ]
    • Relatedness of AEs Per Infusion [ Time Frame: 34 weeks ]
    • Relatedness of AEs Per Subject [ Time Frame: 34 weeks ]


    Information By: CSL Behring

    Dates:
    Date Received: August 18, 2010
    Date Started: November 2010
    Date Completion:
    Last Updated: August 27, 2013
    Last Verified: March 2013