Disease
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Multiple myeloma |
Stage of Disease/Treatment
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Metastatic/advanced |
Prior Therapy
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More than two prior regimens |
Type of Study
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Phase II, single arm |
Primary Endpoint
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Overall response rate |
Secondary Endpoints
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Complete response rate, progression‐free survival, overall survival, tolerability |
Additional Details of Endpoints or Study Design
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The statistical design incorporated a two‐stage Simon minimax design where the first stage would enroll 13 patients, and if one response occurred, the study would proceed to the second stage for a total enrollment of 27 patients. The study would be considered a success if four total responses occurred (14.7%), with an alpha of 5% and a power of 80%. |
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Response Definition: The 2014 International Myeloma Working Group (IMWG) response criteria for multiple myeloma was used. In brief, response criteria are as follows: |
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Stringent complete response (CR): CR with normalization of serum‐free light chains |
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CR: No detectable monoclonal protein by serum protein electrophoresis and immunofixation |
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Very good partial response (PR): 90% improvement in M‐protein |
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PR: 50% improvement in M‐protein |
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Minimal response: 25% improvement in M‐protein |
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Stable disease (SD): Neither PR nor progressive disease (PD) |
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PD: 25% increase in M‐protein |
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In terms of ORR, a PR or better is required. In cases with light chain–only disease, the difference between involved and uninvolved light chains is used to gauge response. |
Investigator's Analysis
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Study prematurely terminated because of the COVID‐19 pandemic, with only modest activity in a small number of patients. |