Table 2.
Summary of efficacy outcome measures at baseline and Day 28 in the AXA1125 and placebo treatment groups.
| Parameter | AXA1125 33.9 g BID (n = 21) |
Placebo BID (n = 20) |
||||
|---|---|---|---|---|---|---|
| Baseline | Day 28 | Δ | Baseline | Day 28 | Δ | |
| τPCr, sec | ||||||
| Mean (SD) | 96.8 (34.6) | 118.0 (68.1) | 21.2 (52.2) | 87.9 (36.3) | 91.9 (43.2) | 4.0 (43.0)† |
| Median (Q1,Q3) | 87.7 (70.9, 119.0) | 111.0 (70.7, 146.2) | 4.5 (−12.0, 34.2) | 75.2 (63.0, 102.0) | 84.8 (55.8, 118.3) | −3.7 (−23.1, 47.7)L |
| 6-MWT distance, m | ||||||
| Mean (SD) | 511.0 (117.1) | 536.6 (91.8) | 25.57 (54.0) | 540.5 (106.5) | 565.8 (20.6) | 25.3 (12.1)† |
| Median (Q1,Q3) | 500 (427.0, 576.0) | 500 (480.0, 604.0) | 23.0 (0.0, 39.0) | 559 (455.0, 651.50) | 559.0 (506.0, 636.0) | 10 (−3.0, 36.5) |
| 6-MWT distance, observed:predicted, % | ||||||
| Mean (SD) | 82.4 (19.6) | 86.7 (17.3) | 4.3 (8.5) | 86.8 (3.9) | 90.8 (14.1) | 4.0 (8.6)† |
| Median (Q1,Q3) | (83.4 (66.8, 100.0) | 80.0 (76.0, 94.3) | 4.3 (0.0, 7.1) | 85.5 (73.4, 97.7) | 89.0 (82.1, 100.6) | 1.7 (−0.5, 5.4) |
| Peak serum lactate post-6MWT, mmol/L | ||||||
| Mean (SD) | 1.30 (1.1) | 1.00 (0.98) | −0.30 (1.0) | 1.64 (1.1) | 1.65 (1.0) | 0.01 (0.6)† |
| Median (Q1,Q3) | 1.4 (0.7, 1.6) | 0.8 (0.0, 1.5) | −0.1 (−0.90, 0.20) | 1.75 (0.75, 2.2) | 1.5 (1.00, 2.00) | 0.0 (−0.6, 0.45) |
| CFQ-11 Total fatigue (Likert) scorea | ||||||
| Mean (SD) | 26.2 (3.59) | 21.0 (5.51) | −5.25 (5.49) | 28.05 (2.96) | 26.45 (3.78) | −2.25 (2.92)∗∗ |
| Median (Q1, Q3) | 26.0 (24.0, 28.0) | 22 (17.3) | −4.0 (−8.5, −1.0) | 28 (25.5, 31.0) | 26.5 (24.3) | −1.5 (−4.0, 0) |
| CFQ-11 Physical fatigue (Likert) scorea | ||||||
| Mean (SD) | 17.3 (2.54) | 13.6 (3.93) | −3.67 (0.75) | 19.0 (1.70) | 17.7 (2.87) | −1.30 (3.05)∗∗ |
| Median (Q1,Q3) | 17.0 (16.0, 19.0) | 13.0 (11.0, 16.0) | −3.0 (−6.0, −1.0) | 19.5 (18.0, 21.0) | 18.0 (16.0, 20.0) | −1.0 (−3.0,-0.50) |
| CFQ-11 Mental fatigue (Likert) scorea | ||||||
| Mean (SD) | 8.95 (1.56) | 7.38 (2.09) | −1.57 (1.99) | 9.10 (1.97) | 8.80 (1.80) | −0.30 (1.13)∗∗ |
| Median (Q1,Q3) | 8.00 (8.0, 10.0) | 8.00 (6.0, 9.0) | −2.0 (−3.0, 0.00) | 8.5 (8.0, 11.0) | 8.0 (8.0, 10.0) | 0.0 (−1.0, 0.5) |
| CFQ-11 Physical fatigue category, % patientsb | ||||||
| Normal | 0 | 3 (14.3) | 0 | 0 | ||
| Mild | 2 (9.5) | 12 (57.1) | 1 (5.0) | 4 (20.0) | ||
| Moderate-severe | 19 (90.5) | 6 (28.6) | 19 (95.0) | 16 (80.0) | ||
| CFQ-11 Mental fatigue category, n (%) patientsc | ||||||
| Normal | 0 | 0 | 0 | 0 | ||
| Mild | 2 (9.5) | 9 (42.9) | 4 (20.0) | 4 (20.0) | ||
| Moderate-severe | 19 (90.5) | 12 (57.1) | 16 (80.0) | 16 (80.0) | ||
BID, twice daily; CFQ-11, Chalder Fatigue Questionnaire, 11-item; SE, standard error; 6-MWT, 6-min walk test; τPCr, phosphocreatine recovery rate time constant. †P > 0.05, ∗P ≤ 0.05, ∗∗P ≤ 0.01, ∗∗∗P ≤ 0.001.
a
Pre-exercise fatigue.
b
Physical fatigue categories: normal = Likert score 0–9; mild = Likert score 10–15; moderate-severe = Likert score ≥16.
c
Mental fatigue categories: normal = Likert score 0–3; mild = Likert score 4–7; moderate-severe = Likert score ≥8. Comparison of least squares mean change from baseline at Day 28: AXA1125 vs placebo (ANCOVA model).