Background
Results
High-dose PXT3003a | Low-dose PXT3003 | Placebo | ||||
---|---|---|---|---|---|---|
FAS | mFAS | FAS | mFAS | FAS | mFAS | |
(N = 113) | (N = 55) | (N = 109) | (N = 93) | (N = 101) | (N = 87) | |
CMTNS-v2b | 13.68 (3.19) | 13.02 (3.25) | 14.13 (3.04) | 14.04 (2.99) | 13.94 (3.13) | 14.01 (3.27) |
ONLS score | 3.18 (1.11) | 3.05 (1.13) | 3.39 (1.05) | 3.33 (1.05) | 3.20 (1.17) | 3.23 (1.19) |
Age (years) | 39.6 (13.9) | 41.2 (13.6) | 41.0 (12.3) | 40.7 (12.4) | 42.1 (13.2) | 42.3 (13.2) |
BMI (kg/m2) | 25.2 (4.52) | 24.9 (4.53) | 25.6 (4.72) | 25.4 (4.68) | 25.4 (4.94) | 25.2 (4.90) |
Sex (n, [%]) | ||||||
Female | 68 (60.2) | 34 (61.8) | 60 (55.0) | 51 (54.8) | 62 (61.4) | 51 (58.6) |
Male | 45 (39.8) | 21 (38.2) | 49 (45.0) | 42 (45.2) | 39 (38.6) | 36 (41.4) |
Race (n, [%]) | ||||||
White | 110 (97.3) | 53 (96.4) | 107 (98.2) | 91 (97.8) | 100 (99.0) | 86 (98.9) |
Black | 1 (0.9) | 1 (1.8) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
Asian | 2 (1.8) | 1 (1.8) | 2 (1.8) | 2 (2.2) | 1 (1.0) | 1 (1.1) |
Efficacy
Test | High-dose PXT3003 | Low-dose PXT3003 | |
---|---|---|---|
ONLS | Arm score (↓) | − 0.19 (− 0.43, 0.04) | 0.01 (− 0.19, 0.22) |
Leg score (↓) | − 0.18 (− 0.36, 0.01) | − 0.13 (− 0.29, 0.02) | |
QMT | Grip strength (↑) (kg) | − 1.49 (− 3.92, 0.93) | 0.37 (− 1.64, 2.37) |
Dorsiflexion strength (↑) (Newton) | 7.48 (− 3.60, 18.57) | 4.09 (− 5.01, 13.20) | |
Electrophysiology | CMAP (↑) (mV) | − 0.33 (− 1.05, 0.40) | 0.14 (− 0.46, 0.74) |
Motor nerve conduction velocity (↑) (m/s) | 0.08 (− 1.52, 1.67) | − 0.09 (− 1.42, 1.25) | |
Distal motor latency (↓) (ms) | − 0.20 (− 1.58, 1.18) | 0.08 (− 1.07, 1.24) | |
Radial SNAP (↑) (µV) | − 0.11 (− 1.20, 0.99) | − 0.04 (− 0.96, 0.88) | |
Quality of life | Mobility (↓) | − 0.07 (− 0.35, 0.20) | − 0.11 (− 0.34, 0.12) |
Self-care (↓) | − 0.12 (− 0.31, 0.06) | − 0.18 (− 0.34, − 0.03) | |
Usual activities (↓) | − 0.02 (− 0.27, 0.23) | − 0.11 (− 0.32, 0.10) | |
Pain/discomfort (↓) | 0.00 (− 0.25, 0.26) | − 0.12 (− 0.33, 0.09) | |
Anxiety/depression (↓) | − 0.04 (− 0.29, 0.22) | − 0.06 (− 0.28, 0.15) | |
Visual analog scale (↑) (mm) | 2.57 (− 2.36, 7.50) | 4.67 (0.59, 8.75) |
Sensitivity analyses
Safety and tolerability
High-dose PXT3003 | Low-dose PXT3003 | Placebo | p | |
---|---|---|---|---|
N = 113 | N = 109 | N = 101 | ||
Any TEAE | 87 (77.0) | 89 (81.7) | 83 (82.2) | 0.581 |
Treatment-related TEAEs | 38 (33.6%) | 39 (35.8%) | 34 (33.7%) | 0.935 |
Moderate or severe | 5 (4.4%) | 8 (7.3%) | 10 (9.9%) | 0.281 |
TEAE leading to drug withdrawal | 6 (5.3%) | 6 (5.5%) | 6 (5.9%) | 1 |
Treatment-related TEAEs | 2 (1.8%) | 3 (2.8%) | 2 (2.0%) | 0.898 |
Serious TEAEs | 3 (2.7%) | 10 (9.2%) | 5 (5.0%) | 0.108 |
Treatment-related TEAEs | Nil | Nil | Nil | |
Leading to drug withdrawal | Nil | 1 (0.9%) | Nil | |
Deaths | Nil | Nil | Nil |
Eventb | High-dose PXT3003 | Low-dose PXT3003 | Placebo | pc |
---|---|---|---|---|
N = 113 | N = 109 | N = 101 | ||
Nausea | 6 (5.3) | 7 (6.4) | 3 (3.0) | 0.502 |
Diarrhea | 6 (5.3) | 3 (2.8) | 5 (5.0) | 0.620 |
Headache | 6 (5.3) | 6 (5.5) | 3 (3.0) | 0.639 |
Dry mouth | 3 (2.7) | 2 (1.8) | 4 (4.0) | 0.642 |
Dizziness | 3 (2.7) | 4 (3.7) | 1 (1.0) | 0.496 |
Muscle spasms | 3 (2.7) | nil | 3 (3.0) | 0.223 |
Nasopharyngitis | 3 (2.7) | 4 (3.7) | 5 (5.0) | 0.662 |
Dyspepsia | 2 (1.8) | 1 (0.9) | 2 (2.0) | 0.868 |
Fatigue | 1 (0.9) | 6 (5.5) | 4 (4.0) | 0.132 |
Somnolence | 1 (0.9) | 5 (4.6) | 1 (1.0) | 0.185 |
Constipation | 1 (0.9) | 3 (2.8) | 1 (1.0) | 0.536 |
Arthralgia | 1 (0.9) | 3 (2.8) | 2 (2.0) | 0.608 |
Asthenia | 1 (0.9) | 1 (0.9) | 2 (2.0) | 0.693 |
Tinnitus | 1 (0.9) | 1 (0.9) | 2 (2.0) | 0.693 |
Abdominal pain | 1 (0.9) | Nil | 2 (2.0) | 0.311 |
Weight increased | Nil | Nil | 3 (3.0) | 0.030 |