MIKIE: A randomized, double-blind, regimen-controlled, phase II, multicenter study to assess the efficacy and safety of two different vismodegib regimens in patients with multiple basal cell carcinomas.
Bibliographic record
Abstract
TPS9121 Background: Vismodegib, a Hedgehog signaling pathway inhibitor, is FDA and EMA approved for patients (pts) with advanced basal cell carcinoma (BCC) who are not candidates for surgery or radiotherapy. While vismodegib reduces BCC tumor burden and blocks growth of new BCCs in pts with basal cell carcinoma nevus syndrome (BCCNS), further studies are required to optimize the therapeutic index of long-term (chronic) therapy. The MIKIE study (NCT01815840) assesses the efficacy and safety of 2 intermittent vismodegib dosing regimens in pts with multiple BCCs. Methods: MIKIE is a randomized, double-blind, regimen-controlled phase II study, in adults with multiple BCCs (including BCCNS) ≥18 years with ≥1 histopathologically confirmed BCC and ≥6 clinically evident BCCs (at least three ≥5 mm in diameter). 200 pts are planned to be randomized (1:1) to regimen A or B and stratified by BCCNS status, geographic region, and immunosuppression status. In regimen A, pts receive 3 courses of vismodegib (150 mg/d) for 12 weeks followed by placebo for 8 weeks, followed by a final 12-week course of vismodegib. In regimen B, pts receive vismodegib (150 mg/d) for 24 weeks, followed by 3 alternating courses of placebo (8 weeks) followed by vismodegib (8 weeks). Total duration of treatment will be 72 weeks in both arms, with 52 weeks of follow up. Major inclusion criteria include ECOG performance status 0–2; adequate hematopoietic, hepatic, and renal function; and signed written informed consent. Major exclusion criteria include inoperable metastatic or locally advanced BCC. Primary objective includes the percentage reduction from baseline in the number of clinically evident BCCs after 72 weeks of treatment in the 2 regimens. Secondary objectives include the percentage reduction in overall size of 3 largest BCCs, the proportion of pts with a 50% reduction in BCCs, the number of pts with new BCCs, recurrence rate, and safety and tolerability (including dropout rate due to tolerability). Skindex-16 quality of life and pharmacokinetic assessments will be conducted at selected sites. The first patient was enrolled in March 2013; the study is ongoing. 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 | 0.006 | 0.003 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 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 | 0.001 |
| 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".