Fig 1.
Mean change from baseline in NSAA total score with domagrozumab compared with placebo (full analysis seta).
CI, confidence interval; NSAA, North Star Ambulatory Assessment. a all patients who were randomized and received at least one dose of study drug.
Fig 2.
Mean change from baseline in NSAA total score by treatment sequence (full analysis seta).
Sequence 1: Domagrozumab dose escalation (5, 20, 40 mg/kg) in first 48 weeks followed by domagrozumab 40 mg/kg in weeks 49–96. Sequence 2: Domagrozumab dose escalation in first 48 weeks followed by placebo in weeks 49–96. Sequence 3: placebo in first 48 weeks followed by domagrozumab dose escalation in weeks 49–96. a all patients who were randomized and received at least one dose of study drug. NSAA, North Star Ambulatory Assessment.
Fig 3.
Proportion of patients who lost function in individual NSAA test items (full analysis seta).
L, left; NSAA, North Star Ambulatory Assessment; R, right. a all patients who were randomized and received at least one dose of study drug.
Fig 4.
Cumulative number of skills (A) gained or (B) lost, through week 49, in domagrozumab- vs. placebo-treated patients.
Data are median [1st and 3rd quartiles]. The stars represent the mean and the circles are the outliers. NSAA, North Star Ambulatory Assessment.
Fig 5.
Cumulative loss of individual NSAA skills over 48 weeks (full analysis set).
Event was defined as a patient dropping from 2 or 1 (able to perform a task) to 0 (unable to perform). This exploratory analysis included transient zero scores that may have increased to a value above zero at a subsequent visit. NSAA, North Star Ambulatory Assessment.
Table 1.
Identified patients with temporary zero value for NSAA total score through week 49.
Table 2.
Mean change from baseline (CFB) in the NSAA total score (from Week 9 to Week 49).
Full analysis and sensitivity analysis, where temporary zero values were changed to “not obtainable”a.