AEs through 30 days after end of trial
| . | Leniolisib (n = 21) . | Placebo (n = 10) . | Total (N = 31) . |
|---|---|---|---|
| Overall AE incidence∗,† | |||
| All, nE, nS (%) | 92, 18 (85.7) | 40, 9 (90.0) | 132, 27 (87.1) |
| Grade 1, nE, nS (%) | 65, 15 (71.4) | 26, 8 (80.0) | 91, 23 (74.2) |
| Grade 2, nE, nS (%) | 19, 9 (42.9) | 10, 5 (50.0) | 29, 14 (45.2) |
| Grade 3, nE, nS (%) | 3, 2 (9.5) | 4, 3 (30.0) | 7, 5 (16.1) |
| Grade 4, nE, nS (%) | 3, 2 (9.5) | 0, 0 (0.0) | 3, 2 (6.5) |
| Grade 5, nE, nS (%) | 0, 0 (0.0) | 0, 0 (0.0) | 0, 0 (0) |
| Study drug–related, nE, nS (%) | 6, 5 (23.8) | 8, 3 (30.0) | 14, 8 (25.8) |
| Serious, nE, nS (%) | 5, 3 (14.3) | 3, 2 (20.0) | 8, 5 (16.1) |
| Incidence of AEs by preferred term‡ | |||
| Headache, nS (%) | 5 (23.8) | 2 (20.0) | 7 (22.6) |
| Nausea, nS (%) | 1 (4.8) | 3 (30.0) | 4 (12.9) |
| Sinusitis, nS (%) | 4 (19.0) | 0 (0.0) | 4 (12.9) |
| Upper respiratory tract infection, nS (%) | 2 (9.5) | 2 (20.0) | 4 (12.9) |
| Fatigue, nS (%) | 2 (9.5) | 1 (10.0) | 3 (9.7) |
| Abdominal discomfort, nS (%) | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Alopecia, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Asthenia, nS (%) | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Back pain, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Diarrhea, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Dizziness, nS (%) | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Eczema, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Neck pain, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Pyrexia, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Urinary tract infection, nS (%) | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Vomiting | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Weight increased | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Incidence of treatment-related AEs as reported by study investigators | |||
| Alopecia, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Abdominal pain, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Aphthous ulcer, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Dyspnea, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Fatigue, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Headache, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Taste disorder, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Vasculitis, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Vertigo, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Vomiting, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Weight increased, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Incidence of serious AEs by preferred term§ | |||
| Alcohol poisoning, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Coma, nS (%)‖ | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Dyspnea, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Failure to thrive, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Lipase increased, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Lymphadenopathy, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Mastoiditis, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Urinary tract infection, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| . | Leniolisib (n = 21) . | Placebo (n = 10) . | Total (N = 31) . |
|---|---|---|---|
| Overall AE incidence∗,† | |||
| All, nE, nS (%) | 92, 18 (85.7) | 40, 9 (90.0) | 132, 27 (87.1) |
| Grade 1, nE, nS (%) | 65, 15 (71.4) | 26, 8 (80.0) | 91, 23 (74.2) |
| Grade 2, nE, nS (%) | 19, 9 (42.9) | 10, 5 (50.0) | 29, 14 (45.2) |
| Grade 3, nE, nS (%) | 3, 2 (9.5) | 4, 3 (30.0) | 7, 5 (16.1) |
| Grade 4, nE, nS (%) | 3, 2 (9.5) | 0, 0 (0.0) | 3, 2 (6.5) |
| Grade 5, nE, nS (%) | 0, 0 (0.0) | 0, 0 (0.0) | 0, 0 (0) |
| Study drug–related, nE, nS (%) | 6, 5 (23.8) | 8, 3 (30.0) | 14, 8 (25.8) |
| Serious, nE, nS (%) | 5, 3 (14.3) | 3, 2 (20.0) | 8, 5 (16.1) |
| Incidence of AEs by preferred term‡ | |||
| Headache, nS (%) | 5 (23.8) | 2 (20.0) | 7 (22.6) |
| Nausea, nS (%) | 1 (4.8) | 3 (30.0) | 4 (12.9) |
| Sinusitis, nS (%) | 4 (19.0) | 0 (0.0) | 4 (12.9) |
| Upper respiratory tract infection, nS (%) | 2 (9.5) | 2 (20.0) | 4 (12.9) |
| Fatigue, nS (%) | 2 (9.5) | 1 (10.0) | 3 (9.7) |
| Abdominal discomfort, nS (%) | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Alopecia, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Asthenia, nS (%) | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Back pain, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Diarrhea, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Dizziness, nS (%) | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Eczema, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Neck pain, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Pyrexia, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Urinary tract infection, nS (%) | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Vomiting | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Weight increased | 1 (4.8) | 1 (10.0) | 2 (6.5) |
| Incidence of treatment-related AEs as reported by study investigators | |||
| Alopecia, nS (%) | 2 (9.5) | 0 (0.0) | 2 (6.5) |
| Abdominal pain, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Aphthous ulcer, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Dyspnea, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Fatigue, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Headache, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Taste disorder, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Vasculitis, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Vertigo, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Vomiting, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Weight increased, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Incidence of serious AEs by preferred term§ | |||
| Alcohol poisoning, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Coma, nS (%)‖ | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Dyspnea, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Failure to thrive, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Lipase increased, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Lymphadenopathy, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
| Mastoiditis, nS (%) | 1 (4.8) | 0 (0.0) | 1 (3.2) |
| Urinary tract infection, nS (%) | 0 (0.0) | 1 (10.0) | 1 (3.2) |
nE, number of events in the category; nS, number of patients with at least 1 AE in the category; % is based on the number of patients.
Common Toxicity Criteria were used to determine AE grade. If grading did not exist for an AE, the following definitions were used: 1, mild; 2, moderate; 3, severe; 4, life-threatening; 5, death.
For AEs in ≥2 patients in total patients. A patient with multiple occurrences of an AE is counted only once in the AE category. Only AEs occurring at or after first drug intake are included.
A patient with multiple serious AEs with the same preferred term is counted only once for that preferred term.
The coma was a consequence of the alcohol poisoning.