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

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.

Time frame: Baseline to week 24

Population: The Intention-to-Treat (ITT) data set comprised all patients treated with the study drug who had at least one post-baseline measurement for muscle strength.

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.

Time frame: Baseline and week 24

Population: The ITT data set comprised all patients treated with the study drug who had at least one post-baseline measurement for muscle strength.

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.

Time frame: Baseline to the completion visit (up to week 25)

Population: The analysis population comprised the subjects in the ITT data set (ie, all patients treated with the study drug) who had at least one post-baseline value (and if necessary a baseline value).

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.

Time frame: Baseline to week 24

Population: The analysis population comprised the subjects in the ITT data set (ie, all patients treated with the study drug) who had at least one post-baseline value (and if necessary a baseline value).

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.

Time frame: At baseline and week 25

Population: The analysis population comprised the subjects in the ITT data set (ie, all patients treated with the study drug) who had at least one post-baseline value (and if necessary a baseline value).

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.

Time frame: Baseline and Week 24

Population: The analysis population comprised the subjects in the ITT data set (ie, all patients treated with the study drug) who had at least one post-baseline value (and if necessary a baseline value).

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).

Time frame: Screening and week 25

Population: The analysis population comprised the subjects in the ITT data set (ie, all patients treated with the study drug) who had at least one post-baseline value (and if necessary a baseline value).

Included all AEs that occurred during the entire study period. Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.

Time frame: For the duration of the study, up to Week 25

Population: The safety data set (SDS) comprised all treated patients.

Laboratory parameters included hematology, serum chemistry, and urinalysis parameters.

Time frame: Baseline to Week 25

Population: The SDS comprised all treated patients.

Vital signs included heart rate, systolic blood pressure, diastolic blood pressure, and body temperature.

Time frame: Baseline to Week 25

Population: The SDS comprised all treated patients.

All AEs arising from local/injection site reactions.

Time frame: For the duration of the study, up to Week 25

Population: The SDS comprised all treated patients.

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.

Time frame: Baseline and week 25

Population: The analysis population comprised the subjects in the ITT data set (ie, all patients treated with the study drug) who had at least one post-baseline value (and if necessary a baseline value).

The rate was the number of AEs over the number of infusions administered. Included all AEs that occurred during the entire study period. Mild AE: Did not interfere with routine activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.

Time frame: For the duration of the study, up to Week 25

Population: The SDS comprised all treated patients.

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.

Time frame: At baseline and week 25

Population: The analysis population comprised the subjects in the ITT data set (ie, all patients treated with the study drug) who had at least one post-baseline value (and if necessary a baseline value).