Table 1.
Demographics and Baseline Characteristics (ITT population)
| Zilucoplan 0·3mg/kg N=12 |
Placebo N=15 |
All Participants N=27 |
||
|---|---|---|---|---|
| Age (years) | ||||
| Mean (SD) | 56·9 (9·0) | 52·8 (13·6) | 54·6 (11·8) | |
| 95% CI | 51·2, 62·7 | 45·2, 60·4 | ||
| Sex | ||||
| F | n (%) | 6 (50·0) | 7 (46·7) | 13 (48·1) |
| M | n (%) | 6 (50·0) | 8 (53·3) | 14 (51·9) |
| Weight (kg) | ||||
| Mean (SD) | 81·1 (17·5) | 91·5 (27·1) | 86·9 (23·5) | |
| 95% CI | 70·0, 92·3 | 76·5, 106·5 | ||
| Countries | ||||
| France | n (%) | 2 (16·7) | 2 (13·3) | 4(14·8) |
| United Kingdom | n (%) | 2 (16·7) | 2 (13·3) | 4 (14·8) |
| Netherlands | n (%) | 0 | 1(6·7) | 1 (3·7) |
| United States | n (%) | 8 (66·7) | 10 (66·7) | 18 (66·7) |
| Race | ||||
| Black or African American | n (%) | 1 (6·7) | 3 (25·0) | 4 (14·8) |
| White | n (%) | 10 (66·7) | 7 (58·3) | 17 (63·0) |
| Unknown | n (%) | 1 (6·7) | 0 | 1 (3·7) |
| Missing | n (%) | 3 (20·0) | 2 (16·7) | 5 (18·5) |
| Months since Initial Diagnosis * | n | 12 | 14 | 26 |
| Mean (SD) | 35·6 (35·2) | 21·5 (24·1) | 28·0 (30·0) | |
| 95% CI | 13·3, 58·0 | 7·6, 35·5 | ||
| Age at Initial IMNM Diagnosis (years) † | n | 12 | 14 | 26 |
| Mean (SD) | 54·2 (9·8) | 52·2 (13·9) | 53·1 (12·0) | |
| HMGCR/SRP Antibodies | ||||
| Positive/Negative | n (%) | 10 (83·3) | 11 (73·3) | 21 (77·8) |
| Negative/Positive | n (%) | 2 (16·7) | 4 (26·7) | 6 (22·2) |
| Muscle Biopsy performed | ||||
| Y | n (%) | 11 (91·7) | 15 (100) | 26 (96.3) |
| Complement C5b-9 or C9 staining performed | n (%) | 4 (33·3) | 5 (33·3) | 9 (33·.3) |
| Positive | n (%) | 3 (25.0) | 5 (33.3) | 8 (29·6) |
| Negative | n (%) | 1 (8.3) | 0 | 1 (3·7) |
| IMNM Treatment history | ||||
| Any medication received for IMNM | n (%) | 11 (91·7) | 14 (93·3) | 25 (92·6) |
| Prednisone | n (%) | 11 (91·7) | 11 (73·3) | 22 (81·5) |
| Methotrexate | n (%) | 8 (66·7) | 7 (46.7) | 15 (55.6) |
| Azathioprine | n (%) | 3 (25·0) | 3 (20·0) | 6 (22·2) |
| Mycophenolate mofetil | n (%) | 2 (16·7) | 3 (20·0) | 5 (18·5) |
| Cyclophosphamide | n (%) | 1 (8·3) | 1 (6·7) | 2 (7·4) |
| Cyclosporine | n (%) | 0 | 0 | 0 |
| Tacrolimus | n (%) | 0 | 0 | 0 |
| Rituximab | n (%) | 3 (25·0) | 6 (40·0) | 9 (33·3) |
| Plasma Exchange | n (%) | 2 (16·7) | 3 (20·0) | 5 (18·5) |
| Intravenous immunoglobulin | n (%) | 9 (75·0) | 10 (66·7) | 19 (70·4) |
| Other | n (%) | 1 (8·3) | 3 (20·0) | 4 (14·8) |
CI=confidence interval, HMGCR=3-hydroxy-3-methyl-glutaryl-coenzyme A reductase, IMNM=Immune mediated necrotizing myopathy, SD= standard deviation, SRP=signal recognition particle.
*
Months since initial diagnosis was calculated as: (Date of Randomization – Date of Initial IMNM Diagnosis + 1)/30.5.
‡
Age at initial diagnosis was calculated as: Year of Initial IMNM Diagnosis - Year of Birth.