Table 2.
Key Secondary Efficacy End Points, with Adjustment for Multiplicity.*
| End Point | Trial L1 | Trial L2 | ||||
|---|---|---|---|---|---|---|
| Placebo (N = 127) | Relugolix Combination Therapy (N = 128) | Delayed Relugolix Combination Therapy (N = 132) | Placebo (N = 129) | Relugolix Combination Therapy (N = 125) | Delayed Relugolix Combination Therapy (N = 127) | |
| Amenorrhea over last 35 days of treatment period — no. (%) | 7 (6) | 67 (52) | 76 (58) | 4 (3) | 63 (50) | 63 (50) |
| Difference vs. placebo (95% CI) — percentage points | 47 (37 to 56) | 47 (38 to 57) | ||||
| P value vs. placebo | <0.001 | <0.001 | ||||
| Percent change from baseline to wk 24 in menstrual blood-loss volume | −23.2±4.6 | −84.3±4.7 | −88.2±4.6 | −15.1±5.5 | −84.3±5.5 | −89.4±5.7 |
| Difference vs. placebo (95% CI) — percentage points | −61.1 (−73.5 to −48.6) | −69.2 (−84.1 to −54.3) | ||||
| P value vs. placebo | <0.001 | <0.001 | ||||
| Change from baseline to wk 24 in Bleeding and Pelvic Discomfort scale score | −16.1±2.8 | −45.0±2.9 | −51.3±2.9 | −18.3±2.9 | −51.7±2.9 | −48.9±3.0 |
| Difference vs. placebo (95% CI) | −28.9 (−36.3 to −21.5) | 33.4 (−41.2 to −25.5) | ||||
| P value vs. placebo | <0.001 | <0.001 | ||||
| Participants with anemia at baseline and an increase in hemoglobin level of >2 g/dl at wk 24 — no. (%)† | 5/23 (22) | 15/30 (50) | 18/32 (56) | 2/37 (5) | 19/31 (61) | 18/31 (58) |
| Difference vs. placebo (95% CI) — percentage points | 28 (4 to 53) | 56 (37 to 75) | ||||
| P value vs. placebo | 0.04 | <0.001 | ||||
| Maximum numerical rating scale score ≤1 over last 35 days of treatment period among participants in pain-evaluation subgroup — no. (%)‡ | 7/69 (10) | 25/58 (43) | 27/65 (42) | 14/82 (17) | 32/68 (47) | 24/58 (41) |
| Difference vs. placebo (95% CI) — percentage points | 33 (18 to 48) | 30 (16 to 44) | ||||
| P value vs. placebo | <0.001 | <0.001 | ||||
| Percent change from baseline to wk 24 in volume of primary uterine fibroid | −0.3±5.4 | −12.4±5.6 | −22.7±5.5 | −7.4±5.9 | −17.4±5.9 | −30.2±6.3 |
| Difference vs. placebo (95% CI) — percentage points | −12.1 (−26.3 to 2.0) | −10.0 (−25.8 to 5.8) | ||||
| P value vs. placebo§ | 0.09 | 0.22 | ||||
| Percent change from baseline to wk 24 in uterine volume | 2.2±3.0 | −12.9±3.1 | −17.9±3.0 | −1.5±3.4 | −13.8±3.4 | −17.7±3.5 |
| Difference vs. placebo (95% CI) — percentage points | −15.1 (−23.0 to −7.3) | −12.2 (−21.3 to −3.2) | ||||
| P value vs. placebo | <0.001 | 0.008 | ||||
Plus–minus values are least-squares means ±SD. In trial L1, the first four key secondary end points were tested sequentially in the order listed, and the remaining three secondary end points were to be tested with the use of the Hochberg step-up procedure. In trial L2, the first, second, third, and fifth secondary end points were tested sequentially, followed by testing of the other three key secondary end points (fourth, sixth, and seventh) with the use of the Hochberg procedure. CI denotes confidence interval.
The percentages of participants with a response were calculated in the subgroup of participants with anemia (hemoglobin level ≤10.5 g per deciliter) at baseline and who had hemoglobin data reported at week 24. This end point was tested fourth in trial L1 and fifth in trial L2.
The percentages of participants with a response were calculated in the subgroup of participants who had pain ratings that could be evaluated (maximum numerical rating scale score of ≥4 at baseline, with ≥28 days [80% of the last 35 days of the treatment period] of pain scores recorded in an electronic diary). This end point was tested fifth in trial L1 and fourth in trial L2.
The P value for the comparison in the percentage change from baseline to week 24 in the volume of the primary uterine fibroid did not meet the cutoff for statistical significance according to the Hochberg procedure.