Table 2.
Adverse Events in the Safety Population.*
| Category | Nirogacestat (N = 69) | Placebo (N = 72) |
|---|---|---|
| Adverse event, any grade — no. (%) | 69 (100) | 69 (96) |
| Advert event, grade ≥3 — no. (%) | 38 (55) | 12 (17) |
| Adverse event leading to early discontinuation of nirogacestat or placebo, any grade — no. (%) | 14 (20) | 1 (1) |
| Adverse event leading to death — no. (%) | 0 | 1 (1)† |
| Adverse events reported in ≥15% of patients in either group, any grade — no. (%) | ||
| Diarrhea | 58 (84) | 25 (35) |
| Nausea | 37 (54) | 28 (39) |
| Fatigue | 35 (51) | 26 (36) |
| Hypophosphatemia | 29 (42) | 5 (7) |
| Maculopapular rash | 22 (32) | 4 (6) |
| Stomatitis | 20 (29) | 3 (4) |
| Headache | 20 (29) | 11 (15) |
| Dermatitis acneiform | 15 (22) | 0 |
| Vomiting | 14 (20) | 14 (19) |
| Rash | 13 (19) | 5 (7) |
| Hot flush | 13 (19) | 4 (6) |
| Alopecia | 13 (19) | 1 (1) |
| Alanine aminotransferase level increased | 12 (17) | 6 (8) |
| Covid-19 | 12 (17) | 12 (17) |
| Weight gain | 11 (16) | 5 (7) |
| Cough | 11 (16) | 3 (4) |
| Abdominal pain | 11 (16) | 9 (12) |
| Aspartate aminotransferase level increased | 11 (16) | 8 (11) |
| Dyspnea | 11 (16) | 4 (6) |
| Decreased appetite | 11 (16) | 8 (11) |
| Dry skin | 11 (16) | 5 (7) |
| Tumor pain | 5 (7) | 13 (18) |
| Ovarian dysfunction‡ | ||
| In the safety population — no. (%) | 27 (39) | 0 |
| In women of childbearing potential in the safety population — no./total no. (%) | 27/36 (75) | 0/37 |
*
Shown are adverse events that emerged or worsened from the time of the first dose of nirogacestat or placebo through 30 days after the last dose. Covid-19 denotes coronavirus disease 2019.
†
The patient died from sepsis.
‡
Ovarian dysfunction includes the Medical Dictionary for Regulatory Activities, version 24.0, preferred terms amenorrhea, premature menopause, menopause, and ovarian failure.