Table 2.
Changes in Anthropometric and Metabolic Parameters Compared With Baseline After 52 Weeks of Setmelanotide Treatment: Pivotal Patients With BBS and Alström Syndrome (FAS; n=36)
| ≥12 years old (n=31) | ||||
|---|---|---|---|---|
|
| ||||
| ATB | Week 52 | Change from ATB | % change from ATB | |
| Body weight, kg | 117·0 (29·3) | 111·1 (31·0) | −5·9 (9·3) [−9·3, −25] p=0·0007 | −5·2 (7·9) [−8·1, −2·3] p=0·0005 |
| Achieved >10% lossa, % | — | 32·3 [16·7, 51·4] p=0·0006 | — | — |
| Maximal hunger scoreb | 7·3 (2·0) | 5·0 (2·4) | −2·3 (2·0) [−3·3, −1·2] p=0·0002 | −30·9 (24·7) [−44·1, −17·7] p<0·0001 |
| Achieved >25% reduction, %a,b | — | 62·5 [35·4, 84·8] p<0·0001 | — | — |
|
| ||||
| All ages, ≥6 years old (n=36) | ||||
|
| ||||
| ATB | Week 52 | Change from ATB | % change from ATB | |
|
| ||||
| Waist circumference, cmc | 118·1 (18·1) | 113·2 (24·8) | −5·1 (10·9) [−9·3, −0·8] | −4·7 (9·2) [−8·3, −1·2] |
| Body fat, kgd | 51·9 (21·0) | 43·3 (16·2) | −4·6 (12·0) [−10·2, 1·3] | −8·9 (26·1) [−21·5, 3·6] |
| Lean muscle, kgd | 57·8 (14·4) | 56·2 (12·6) | −0·7 (4·0) [−2·7, 1·2] | −1·0 (7·0) [−4·4, 2·4] |
| Lipids, mmol/Le | ||||
| Total cholesterol | 4·4 (1·0) | 3·9 (0·9) | −0·3 (0·4) | −7·0 (10·2) [−11·1, −2·8] |
| HDL cholesterol | 1·1 (0·2) | 1·1 (0·2) | 0·1 (0·1) | 4·3 (11·6) [−0·4, 9·0] |
| LDL cholesterol | 3·0 (1·0) | 2·3 (0·9) | −0·2 (0·4) | −8·8 (16·2) [−15·3, −2·3] |
| Triglycerides | 2·0 (1·4) | 1·4 (0·8) | −0·2 (0·6) | −10·7 (32·0) [−23·6, 2·2] |
Data are the mean (SD), mean [95% CI], or mean (SD) [95% CI] unless otherwise specified. ATB, active treatment baseline (defined as last measurement before the first dose of setmelanotide; ie, Week 0 for setmelanotide group and Week 14 for placebo group); BBS, Bardet-Biedl syndrome; CI, confidence interval; FAS, full analysis set; HDL, high-density lipoprotein; LDL, low-density lipoprotein; SD, standard deviation.
Multiple imputation model used to impute measurements for patients with <52 weeks of treatment.
Estimated proportion on the basis of qualifying patients in the FAS (ie, ≥12 years old without cognitive impairment; n=16).
Patients with measurements at ATB (n=36) and Week 52 (n=28).
Patients with measurements at ATB (n=33), Week 52 (n=20), and both (n=19).
Patients with measurements at ATB (n=36) and Week 52 (n=26).