Clinical Trial: Study of the Effectiveness of Intravenous Immune Globulin (10%) for the Treatment of Multifocal Motor Neuropathy

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

Official Title: A Randomized, Double-Blind, Placebo Controlled, Cross-over Study of the Effectiveness of Immune Globulin Intravenous (Human), 10% (IGIV, 10%) for the Treatment of Multifocal Moto

Brief Summary: The purpose of the study is to evaluate the efficacy (effect on grip strength and disability) and safety/tolerability of IGIV, 10% in subjects with Multifocal Motor Neuropathy.

Detailed Summary:
Sponsor: Baxalta US Inc.

Current Primary Outcome:

  • Grip Strength in the More Affected Hand [ Time Frame: Week 0, then at Last infusion cycle for each study part (Day 8 of last treatment cycle for 2-week interval or Day 15 of last treatment cycle for 3 or 4 -week interval), then at the end of study visit ]

    The grip strength was measured using a DynEx digital dynamometer. The result of grip strength was recorded to a resolution of 0.1 kg.

    Each grip strength test consisted of 3 maximal repeated contractions (trials). Each participant will perform 2 sessions of grip strength testing. After a 10-minute break, the testing session will be repeated for a total of 6 grip repetitions per hand.

  • Mean Relative Change in Grip Strength in the More Affected Hand [ Time Frame: Baseline and last infusion cycle during the two study cross-over periods, approximately weeks 13 and 24; and weeks 37 and 48 (i.e. baseline and end of Study Parts 2 and 4) ]

    Relative Change is defined as 100 * (End of the Cross-Over Period - baseline of Cross-Over Period) divided by baseline of Cross-Over Period.

    The grip strength was measured using a DynEx digital dynamometer. The result of grip strength was recorded to a resolution of 0.1 kg. For statistical analysis, the mean of (usually three) trials for cross-over sessions 1 and 2 was computed and the mean of the sessions was used in the analysis as the result of the grip strength measurement. Only if no grip strength testing could be performed the results were considered as missing.

  • Co-Primary Endpoint: Guy's Neurologic Disabi

    Original Primary Outcome:

    • Grip strength in the more affected hand (primary endpoint) [ Time Frame: End of blinded cross-over period ]
    • Upper limb subsection of the Guy's Neurologic Disability Scale (co primary endpoint) [ Time Frame: End of blinded cross-over period ]


    Current Secondary Outcome:

    • Percentage of Participants With at Least a 30% Decline in Relative Grip Strength in the More Affected Hand (Measured Using a DynEx Digital Dynamometer) [ Time Frame: Baseline and last infusion cycle during the two study cross-over periods, approximately weeks 13 and 24; and weeks 37 and 48 (i.e. baseline and end of Study Parts 2 and 4) ]

      Relative grip strength change is defined as 100 * (End of the Cross-Over Period - baseline of Cross-Over Period) divided by baseline of Cross-Over Period.

      The grip strength was measured using a DynEx digital dynamometer. The result of grip strength was recorded to a resolution of 0.1 kg. For statistical analysis, the mean of (usually three) trials for cross-over sessions 1 and 2 was computed and the mean of the sessions was used in the analysis as the result of the grip strength measurement. Only if no grip strength testing could be performed the results were considered as missing.

    • Grip Strength in the Less Affected Hand [ Time Frame: Week 0, then at Last infusion cycle for each study part (Day 8 of last treatment cycle for 2-week interval or Day 15 of last treatment cycle for 3 or 4 -week interval), then at the end of study visit ]

      The grip strength was measured using a DynEx digital dynamometer. The result of grip strength was recorded to a resolution of 0.1 kg.

      Each grip strength test consisted of 3 maximal repeated contractions (trials). Each participant will perform 2 sessions of grip strength testing. After a 10-minute break, the testing session will be repeated for a total of 6 grip repetitions per hand.

    • Mean Relative Change in Grip Strength in the Less Affected Hand [ Time Frame: Baseline and last infusion cycle during the two study cross-over periods, approximately weeks 13 and 24; and weeks 37 and 48 (i.e. baseline and end of Study Parts 2 and 4) ]

      Relative Change is defined as 100 * (End of the Cross-Over Period - Baseline of Cross-Over Period) divided by baseline of Cross-Over Period.

      The grip strength was measured using a DynEx digital dynamometer. The result of grip strength was recorded to a resolution of 0.1 kg. For statistical analysis, the mean of (usually three) trials for cross-over sessions 1 and 2 was computed and the mean of the sessions was used in the analysis as the result of the grip strength measurement. Only if no grip strength testing could be performed the results were considered as missing.

    • Proportion of Participants That Were Accelerated Forward Into the Next Stabilization Phase (ie Switched to Open-Label IGIV, 10%) [ Time Frame: During the two study cross-over periods, approximately weeks 13-24 and weeks 37-48 (i.e. Study Parts 2 and 4) ]
      Participants were permitted to switch from blinded treatment with placebo or IGIV, 10% to open label IGIV, 10% if they and investigator agreed that deterioration had occurred to the extent that the participant had unacceptable difficulty carrying out daily activities involving the affected muscles, or decline in grip strength of ≥50% in the more affected hand had occurred.
    • Patient Global Impression of Change [ Time Frame: Last infusion cycle for each study part (Day 8 of last treatment cycle for 2-week interval or Day 15 of last treatment cycle for 3 or 4 -week interval), then at the end of study visit ]

      Patient Global Impression of Change was measured on an ordinal scale of 1-7, higher scores representing greater perceived deterioration since the previous efficacy assessment (ranging from (1) very much improved to very much worse (7)).

      1. Very much improved
      2. Much improved
      3. Minimally improved
      4. No change
      5. Minimally worse
      6. Much worse
      7. Very much worse
    • Overall Disability Sum Score [ Time Frame: Week 0, then at Last infusion cycle for each study part (Day 8 of last treatment cycle for 2-week interval or Day 15 of last treatment cycle for 3 or 4 -week interval), then at the end of study visit ]

      The overall disability sum scale (based on Merkies et al., 2002) is a patient questionnaire that measures disability.

      Overall disability sum score = arm disability scale (range 0-5) + leg disability scale (range 0-7); Overall Range: 0 (no signs of disability) to 12 (maximum disability).

    • Overall Disability Sum Score - Standardized [ Time Frame: Week 0, then at Last infusion cycle for each study part (Day 8 of last treatment cycle for 2-week interval or Day 15 of last treatment cycle for 3 or 4 -week interval), then at the end of study visit ]

      The overall disability sum scale (bas

      Original Secondary Outcome:

      Information By: Baxalta US Inc.

      Dates:
      Date Received: April 23, 2008
      Date Started: August 2008
      Date Completion:
      Last Updated: June 26, 2015
      Last Verified: January 2013