Clinical Trial: A Study of Purified Human Antibodies Administered Subcutaneously to Patients With Multifocal Motor Neuropathy (MMN)

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

Official Title: A Multicentre Study of Subcutaneous Immunoglobulin (SCIG) in Patients With Multifocal Motor Neuropathy (MMN)

Brief Summary: The objective of this study is to assess efficacy, safety, and convenience of purified human antibodies administered under the skin in the treatment of MMN patients.

Detailed Summary:
Sponsor: CSL Behring

Current Primary Outcome:

  • Change From Baseline to Week 24 in Muscle Strength [ Time Frame: Baseline to week 24 ]

    The change in Medical Research Council (MRC) score was determined at week 24 compared to baseline using descriptive statistics and nonparametric, two-sided 95% confidence intervals based on the Hodges-Lehmann method. Data for one of the eight subjects was from week 13 as week 24 data were not available.

    The 200-point MRC sum score is the sum of scores for 20 bilateral (left and right side) muscle groups, each rated between 0 (no movement) to 5 (normal movement/power). 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.

  • Mean Overall MRC Score at Baseline and Week 24 [ Time Frame: Baseline and week 24 ]
    The 200-point MRC sum score is the sum of scores for 20 bilateral (left and right side) muscle groups, each rated between 0 (no movement) to 5 (normal movement/power). A higher MRC sum score indicates greater muscle contraction/limb movement.


Original Primary Outcome: Muscle strength of the patients measured using a modified Medical Research Council (MRC) scale [ Time Frame: 24 weeks ]

Current Secondary Outcome:

  • Change From Baseline to Week 24 in Disability [ Time Frame: Baseline to week 24 ]

    The change in disability score was determined at week 24 compared to baseline using descriptive statistics and nonparametric two-sided 95% confidence intervals based on the Hodges-Lehmann method. Data for one of the eight subjects was from week 13 as week 24 data were not available.

    Disability was measured using a modified Guy's Neurological Disability Scale, which comprises subscales for upper and lower limb disability. Both subscales comprise 6 grades, numbered from 0 (no upper limb problem/walking is not affected) to 5 (unable to use either arm for any purposeful movements/usually uses a wheelchair indoors). The disability score is calculated as the sum of both subscales, resulting in a score ranging from 0 to 10. A higher disability score indicates greater disability. Negative values for change in disability score indicate improvement, with a more negative value indicating greater improvement compared with the value at baseline.

  • Mean Disability Score at Baseline and Week 24 [ Time Frame: Baseline and Week 24 ]
    Disability was measured using a modified Guy's Neurological Disability Scale, which comprises subscales for upper and lower limb disability. Both subscales comprise 6 grades, numbered from 0 (no upper limb problem/walking is not affected) to 5 (unable to use either arm for any purposeful movements/usually uses a wheelchair indoors). The disability score is calculated as the sum of both subscales, resulting in a score ranging from 0 to 10. A higher disability score indicates greater disability.
  • Change From Baseline to the Completion Visit in Motor Function [ Time Frame: Baseline to the completion visit (up to week 25) ]

    The change in motor function was determined at the completion visit compared to baseline using descriptive statistics and nonparametric two-sided 95% confidence intervals based on the Hodges-Lehmann method.

    For each patient, four specific tasks were defined according to his/her weakened muscle group. The patient had to grade each of the tasks on a 5-point scale ranging from 0 (normal function) to 4 (not possible). The overall motor function score was calculated as the sum of the 4 grades, resulting in a score ranging from 0 (optimal) to 16 (worst). The baseline motor function score was calculated as the mean of the patient's assessments at Screening and Week 1. Negative values for change in motor function score indicate improvement, with a more negative value indicating greater improvement compared with the value at baseline.

  • Mean Motor Function Score at Screening and Week 25 [ Time Frame: Screening and week 25 ]
    For each patient, four specific tasks were defined according to his/her weakened muscle group. The patient had to grade each of the tasks on a 5-point scale ranging from 0 (normal function) to 4 (not possible). The overall motor function score was calculated as the sum of the 4 grades, resulting in a score ranging from 0 (optimal) to 16 (worst).
  • Health-Related Quality of Life at Baseline and Week 25 [ Time Frame: At baseline and week 25 ]

    Assessed using a questionnaire on patients' satisfaction with current immunoglobulin G (IgG) treatment, treatment at home, and treatment at the hospital/doctor's office. The questions were answered by choosing a number between 1 (extremely good) and 7 (extremely bad).

    Note: No patients received IgG treatment at the hospital/doctor's office at Week 25.

  • Treatment Satisfaction at Baseline and Week 25 [ Time Frame: At baseline and week 25 ]
    Treatment satisfaction was assessed using the Life Quality Index, which comprises 15 items rated on a 7-point scale (1 = worst rating, 7 = best rating) with a possible maximum score of 105. The highest score indicates the highest satisfaction with the impact of treatment on social factors. The 15 items were summarized to 4 scales: treatment interference, therapy-related problems, therapy setting, and treatment costs. The raw scores for these scales were transformed to a score ranging from 0 to 100, with 100 being the best score achievable.
  • Overall Health Status at Baseline and Week 25 [ Time Frame: Baseline and week 25 ]
    Overall Health Status was assessed using a Visual Analogue Scale (VAS). Patients were asked to rate their overall health status by placing a mark on a 100 mm VAS, with 0 being the worst imaginable state and 100 being the best imaginable state.
  • Number of Patients With Adverse Events (AEs) by Severity and Relatedness [ Time Frame: For the duration of the study, up to Week 25 ]

    Included all AEs that occurred during the entire study period.

    Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat wit

    Original Secondary Outcome:

    • Disability, scored using modified Guy's Neurological Disability Scale [ Time Frame: 24 weeks ]
    • Individual Functional Test of four common daily tasks (motor function) [ Time Frame: bi-weekly and 24 weeks ]
    • Quality of Life (QoL), using an adapted QoL questionnaire [ Time Frame: Comparing QoL measured at screening with QoL completion visit in week 25 ]


    Information By: CSL Behring

    Dates:
    Date Received: June 18, 2008
    Date Started: November 2007
    Date Completion:
    Last Updated: June 2, 2013
    Last Verified: June 2013