Mikie: A randomized, double-blinded, regimen-controlled, phase 2 study to assess the efficacy and safety of two different vismodegib (VISMO) regimens in patients (pts) with multiple basal cell carcinomas (BCCs).
Bibliographic record
Abstract
9509 Background: VISMO, a first-in-class Hedgehog pathway inhibitor, is approved for use in adult pts with advanced BCC. Pts with multiple BCCs, including BCC nevus syndrome (BCCNS), require VISMO treatment for extended periods of time. MIKIE (NCT01815840) was designed to assess the efficacy and safety of 2 long-term intermittent VISMO dosing regimens in pts with multiple BCCs. Methods: Adult pts with multiple BCCs (including BCCNS) with ≥ 1 histopathologically confirmed BCC and ≥ 6 clinically evident BCCs were randomized 1:1 to Regimen A (VISMO 150 mg/d × 12 wks, then 3 alternating courses of placebo (P) × 8 wks followed by VISMO × 12 wks) or B (VISMO 150 mg/d × 24 wks, then 3 alternating courses of P × 8 wks followed by VISMO × 8 wks). Primary objective was % reduction from baseline (BL) in number of BCCs at wk 73 (end of treatment). Results: At primary analysis cut-off (Aug 27, 2015), 229 pts were randomized (A: n = 116; B: n = 113). Mean % reduction in number of BCC lesions at wk 73 (ITT population) was 62.1% (Regimen A) vs 54.0% (Regimen B). Mean % reduction from BL in total size of 3 target lesions was 82.9% (Regimen A) vs 68.8% (Regimen B), with a higher proportion of pts in Regimen A (65.5%) experiencing ≥ 50% reduction in total number of BCCs (target and nontarget) vs pts in Regimen B (50.4%). Majority of pts in both regimens (A: 98.3%; B: 97.3%) reported a treatment-emergent adverse event (AE); most common were muscle spasms (A: 71.3%; B: 82.3%), dysgeusia (A: 65.2%; B: 66.4%), and alopecia (A: 62.6%; B: 64.6%). Majority of AEs in both regimens were grade 1-2. Treatment discontinuation rate was 44.0% (A) vs 50.4% (B), with AEs the main reason for discontinuation (A: 19.8%; B: 26.5%). Clinically meaningful improvement ( ≥ 10-point change from BL) in all Skindex-16 QoL domains was noted for both regimens. Conclusions: Both treatment regimens were efficacious, with treatment interruptions not appearing to affect efficacy in either regimen. Regimen A was associated with numerically better outcomes in terms of efficacy and discontinuation rate. The safety profiles of both regimens were similar and consistent with previous clinical experience. Clinical trial information: NCT01815840.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.005 | 0.003 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.003 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".