Characteristics of clinical trials in AL amyloidosis
| Variable . | Trials, n (%), except when indicated . |
|---|---|
| Disease setting | |
| Newly diagnosed | 12 (37.5) |
| Previously treated | 17 (53.1) |
| Mixed | 3 (9.4) |
| Trial sponsor | |
| Investigator-sponsored | 17 (53.1) |
| Industry-sponsored | 8 (25.0) |
| Cooperative group | 7 (21.9) |
| Trial phase | |
| 1 | 7 (21.9) |
| 2 | 14 (43.8) |
| 1/2 | 6 (18.8) |
| 3 | 5 (15.6) |
| Randomized | 11 (34.4) |
| Estimated sample size, median (range) | 45 (12-416) patients |
| Trial location | |
| United States | 17 (53.1) |
| Ex-US | 10 (31.3) |
| Both United States and ex-US | 5 (15.6) |
| Primary end point | |
| Safety | 13 (40.6) |
| OS | 4 (12.5) |
| Hematologic response rate | 13 (40.6) |
| HRQoL measured | 12 (37.5) |
| Nature of investigational agent | |
| Clone-directed | 27 (84.4) |
| Fibril-directed | 4 (12.5) |
| Other | 1 (3.1) |
| Biomarker-selected∗ | 5 (15.6) |
| dFLC cutoff for inclusion, mg/dL | |
| 2 | 8 (25.0) |
| 4 | 2 (6.3) |
| 4.5 | 1 (3.1) |
| 5 | 16 (50.0) |
| 18 | 2 (6.3) |
| No cutoff | 2 (6.3) |
| Not available | 1 (3.1) |
| Upper limit of NT-proBNP for exclusion, pg/mL | |
| 1800 | 2 (6.3) |
| 5000 | 2 (6.3) |
| 7500 | 1 (3.1) |
| 8500 | 17 (53.1) |
| No upper limit | 5 (15.6) |
| Not available | 5 (15.6) |
| NYHA class for exclusion† | |
| III or higher | 10 (55.6) |
| IIIb or higher | 6 (33.3) |
| IV | 2 (11.1) |
| Lower limit of ANC for exclusion‡ | |
| 1000/cc | 21 (91.3) |
| 1500/cc | 2 (8.7) |
| Treatment duration§ | |
| Fixed-duration | 26 (83.9) |
| Treatment until progression | 5 (16.1) |
| Variable . | Trials, n (%), except when indicated . |
|---|---|
| Disease setting | |
| Newly diagnosed | 12 (37.5) |
| Previously treated | 17 (53.1) |
| Mixed | 3 (9.4) |
| Trial sponsor | |
| Investigator-sponsored | 17 (53.1) |
| Industry-sponsored | 8 (25.0) |
| Cooperative group | 7 (21.9) |
| Trial phase | |
| 1 | 7 (21.9) |
| 2 | 14 (43.8) |
| 1/2 | 6 (18.8) |
| 3 | 5 (15.6) |
| Randomized | 11 (34.4) |
| Estimated sample size, median (range) | 45 (12-416) patients |
| Trial location | |
| United States | 17 (53.1) |
| Ex-US | 10 (31.3) |
| Both United States and ex-US | 5 (15.6) |
| Primary end point | |
| Safety | 13 (40.6) |
| OS | 4 (12.5) |
| Hematologic response rate | 13 (40.6) |
| HRQoL measured | 12 (37.5) |
| Nature of investigational agent | |
| Clone-directed | 27 (84.4) |
| Fibril-directed | 4 (12.5) |
| Other | 1 (3.1) |
| Biomarker-selected∗ | 5 (15.6) |
| dFLC cutoff for inclusion, mg/dL | |
| 2 | 8 (25.0) |
| 4 | 2 (6.3) |
| 4.5 | 1 (3.1) |
| 5 | 16 (50.0) |
| 18 | 2 (6.3) |
| No cutoff | 2 (6.3) |
| Not available | 1 (3.1) |
| Upper limit of NT-proBNP for exclusion, pg/mL | |
| 1800 | 2 (6.3) |
| 5000 | 2 (6.3) |
| 7500 | 1 (3.1) |
| 8500 | 17 (53.1) |
| No upper limit | 5 (15.6) |
| Not available | 5 (15.6) |
| NYHA class for exclusion† | |
| III or higher | 10 (55.6) |
| IIIb or higher | 6 (33.3) |
| IV | 2 (11.1) |
| Lower limit of ANC for exclusion‡ | |
| 1000/cc | 21 (91.3) |
| 1500/cc | 2 (8.7) |
| Treatment duration§ | |
| Fixed-duration | 26 (83.9) |
| Treatment until progression | 5 (16.1) |