Gli1 expression by reverse transcription polymerase chain reaction (ΔCT) in whole blood by treatment in phase 1b and phase 2 stage 1
| Level . | Phase 1b dose-escalation phase . | Phase 1b dose expansion phase . | All patients (N = 50) . | ||
|---|---|---|---|---|---|
| Sonidegib 400 mg + ruxolitinib 10 mg (n = 8) . | Sonidegib 400 mg + ruxolitinib 15 mg (n = 10) . | Sonidegib 400 mg + ruxolitinib 20 mg (n = 5) . | Sonidegib 400 mg + ruxolitinib 20 mg (n = 27) . | ||
| Baseline | |||||
| n (%) | 8 (100) | 10 (100) | 5 (100) | 27 (100) | 50 (100) |
| Mean | 6.9 | 6.5 | 6.6 | 6.4 | 6.52 |
| SD (CV%) | 0.80 (11.5) | 0.90 (13.8) | 0.45 (6.8) | 0.93 (14.5) | 0.79 (13.1) |
| Week 25, day 1 (fold-change from baseline) | |||||
| n (%) | 6 (75.0) | 7 (70.0) | 5 (100) | 22 (81.5) | 40 (80) |
| Mean | −0.7 | −0.8 | −1.4 | −0.9 | −0.92 |
| SD (CV%) | 1.41 (−206.4) | 1.55 (−199.8) | 1.77 (−124.7) | 2.08 (−229.6) | 1.68 (−207.8) |
| Week 49, day 1 (fold-change from baseline) | |||||
| n (%) | 4 (50.0) | 3 (30.0) | 4 (80.0) | 14 (51.9) | 25 (50) |
| Mean | −1.0 | −0.2 | −1.3 | −1.7 | −1.34 |
| SD (CV%) | 1.56 (−160.6) | 1.45 (−706.0) | 2.04 (−155.8) | 2.45 (−147.0) | 1.82 (−217.7) |
| End of treatment (fold-change from baseline) | |||||
| n (%) | 4 (50.0) | 8 (80.0) | 1 (20.0) | 22 (81.5) | 35 (70) |
| Mean | −1.0 | −0.2 | −1.0 | −1.4 | −1.07 |
| SD (CV%) | 1.57 (−155.7) | 1.76 (−871.6) | 0 (0.0) | 2.15 (−156.6) | 1.78 (−315.5) |
| Level . | Phase 1b dose-escalation phase . | Phase 1b dose expansion phase . | All patients (N = 50) . | ||
|---|---|---|---|---|---|
| Sonidegib 400 mg + ruxolitinib 10 mg (n = 8) . | Sonidegib 400 mg + ruxolitinib 15 mg (n = 10) . | Sonidegib 400 mg + ruxolitinib 20 mg (n = 5) . | Sonidegib 400 mg + ruxolitinib 20 mg (n = 27) . | ||
| Baseline | |||||
| n (%) | 8 (100) | 10 (100) | 5 (100) | 27 (100) | 50 (100) |
| Mean | 6.9 | 6.5 | 6.6 | 6.4 | 6.52 |
| SD (CV%) | 0.80 (11.5) | 0.90 (13.8) | 0.45 (6.8) | 0.93 (14.5) | 0.79 (13.1) |
| Week 25, day 1 (fold-change from baseline) | |||||
| n (%) | 6 (75.0) | 7 (70.0) | 5 (100) | 22 (81.5) | 40 (80) |
| Mean | −0.7 | −0.8 | −1.4 | −0.9 | −0.92 |
| SD (CV%) | 1.41 (−206.4) | 1.55 (−199.8) | 1.77 (−124.7) | 2.08 (−229.6) | 1.68 (−207.8) |
| Week 49, day 1 (fold-change from baseline) | |||||
| n (%) | 4 (50.0) | 3 (30.0) | 4 (80.0) | 14 (51.9) | 25 (50) |
| Mean | −1.0 | −0.2 | −1.3 | −1.7 | −1.34 |
| SD (CV%) | 1.56 (−160.6) | 1.45 (−706.0) | 2.04 (−155.8) | 2.45 (−147.0) | 1.82 (−217.7) |
| End of treatment (fold-change from baseline) | |||||
| n (%) | 4 (50.0) | 8 (80.0) | 1 (20.0) | 22 (81.5) | 35 (70) |
| Mean | −1.0 | −0.2 | −1.0 | −1.4 | −1.07 |
| SD (CV%) | 1.57 (−155.7) | 1.76 (−871.6) | 0 (0.0) | 2.15 (−156.6) | 1.78 (−315.5) |
CV% means are derived as “SD/mean*100.” ΔCT, a target gene, is the difference between the target gene CT and the average of the control genes CTs. Fold-change is defined as follows: let Diff = (ΔCT)post − (ΔCT)pre; fold-change = 2^(Diff); if Diff ≥0; fold-change = −1*{2^(−1*Diff) }; if Diff <0.
CT, cycle threshold (raw CT results normalized).