{"classes":[{"categories":[{"measurements":[{"groupId":"OG000","lowerLimit":"0.0","upperLimit":"60.2","value":"0.0"},{"groupId":"OG001","lowerLimit":"0.0","upperLimit":"41.0","value":"0.0"},{"groupId":"OG002","lowerLimit":"22.3","upperLimit":"95.7","value":"66.7"},{"groupId":"OG003","lowerLimit":"0.4","upperLimit":"64.1","value":"16.7"},{"groupId":"OG004","lowerLimit":"33.7","upperLimit":"64.2","value":"48.9"},{"groupId":"OG005","lowerLimit":"5.0","upperLimit":"53.8","value":"23.1"}]}]}],"denoms":[{"counts":[{"groupId":"OG000","value":"4"},{"groupId":"OG001","value":"7"},{"groupId":"OG002","value":"6"},{"groupId":"OG003","value":"6"},{"groupId":"OG004","value":"45"},{"groupId":"OG005","value":"13"}],"units":"Participants"}],"description":"The CBRR was defined as the percentage of patients with a clinical benefit response (CBR), defined as the first occurrence of confirmed disease response including MR or better (i.e, MR, PR, VGPR, CR, or sCR). Starting on completion of Cycle 2, response was assessed according to the IMWG criteria based on the Investigator's assessment for all patients at every cycle during the treatment period. MR was defined as ≥25% but \\<49% reduction of serum M-protein and reduction in 24 hour urine M-protein by 50 to 89%, which still exceeds 200 mg/24 hours. In addition to above; if present at baseline, 25 to 49% reduction in the size of soft tissue plasmacytomas is also required. No increase in size or number of lytic bone lesions (development of compression fractures does not exclude response). PR was defined as 50% reduction of serum M-protein and reduction in 24 hour urinary M-protein by ≥90% or to \\<200 mg/24 hour.","dispersionType":"95% Confidence Interval","groups":[{"description":"Patients were treated with 15 mg melflufen as IV infusion on Day 1 and 40 mg dexamethasone oral tablet or IV infusion on Days 1, 8 and 15 of each 21-day treatment cycle. Patients could continue receiving treatment for up to 8 cycles or until they experienced unacceptable toxicity, disease progression, withdrew consent, and/or the study drug was discontinued at the discretion of the Investigator.","id":"OG000","title":"Phase I: Melflufen 15 mg + Dexamethasone"},{"description":"Patients were treated with 25 mg melflufen as IV infusion on Day 1 and 40 mg dexamethasone oral tablet or IV infusion on Days 1, 8 and 15 of each 21-day treatment cycle. Patients could continue receiving treatment for up to 8 cycles or until they experienced unacceptable toxicity, disease progression, withdrew consent, and/or the study drug was discontinued at the discretion of the Investigator.","id":"OG001","title":"Phase I: Melflufen 25 mg + Dexamethasone"},{"description":"Patients were treated with 40 mg melflufen as IV infusion on Day 1 and 40 mg dexamethasone oral tablet or IV infusion on Days 1, 8 and 15 of each 21-day treatment cycle. Patients could continue receiving treatment for up to 8 cycles or until they experienced unacceptable toxicity, disease progression, withdrew consent, and/or the study drug was discontinued at the discretion of the Investigator.","id":"OG002","title":"Phase I: Melflufen 40 mg + Dexamethasone"},{"description":"Patients were treated with 55 mg melflufen as IV infusion on Day 1 and 40 mg dexamethasone oral tablet or IV infusion on Days 1, 8 and 15 of each 21-day treatment cycle. Patients could continue receiving treatment for up to 8 cycles or until they experienced unacceptable toxicity, disease progression, withdrew consent, and/or the study drug was discontinued at the discretion of the Investigator.","id":"OG003","title":"Phase I: Melflufen 55 mg + Dexamethasone"},{"description":"Patients were treated with 40 mg melflufen as IV infusion on Day 1 and 40 mg dexamethasone oral tablet or IV infusion on Days 1, 8 and 15 of each 21-day or 28-day treatment cycle. Patients could continue receiving treatment for up to 8 cycles or until they experienced unacceptable toxicity, disease progression, withdrew consent, and/or the study drug was discontinued at the discretion of the Investigator. In Protocol amendment 4, the cycle of melflufen was increased from 21 to 28 days. For any patients on the 28-day treatment schedule, an additional dose of 40 mg dexamethasone was administered on Day 22 of each cycle.","id":"OG004","title":"Phase I + II: Melflufen 40 mg + Dexamethasone"},{"description":"Patients were treated with 40 mg melflufen as IV infusion on Day 1 of each 28-day treatment cycle. Dexamethasone was not administered as an antitumor compound. However, all patients were treated with 8 mg dexamethasone as an antiemetic on Days 1 and 2, and an optional 4 mg dexamethasone as an antiemetic on Days 3 and 4 of the same 28-day cycle, unless the Investigator decided to switch a patient to a combination regimen. Patients could continue receiving treatment for up to 8 cycles or until they experienced unacceptable toxicity, disease progression, withdrew consent, and/or the study drug was discontinued at the discretion of the Investigator.","id":"OG005","title":"Phase II: Melflufen 40 mg (Single Agent)"}],"paramType":"NUMBER","populationDescription":"For Phase I arms: The Safety Analysis Set included all patients who received at least 1 dose, or part thereof, of study drug.\n\nFor Phase II arms: The mITT Analysis Set included all patients considered to be valid for the Safety Analysis Set and who received at least 1 dose of study drug at the MTD as the initial dose.","reportingStatus":"POSTED","timeFrame":"Baseline and every cycle from Cycle 2 onwards until confirmed disease progression. Assessed until EOT date of 29 October 2019; up to a maximum of approximately 76 months.","title":"Clinical Benefit Response Rate (CBRR)","type":"PRIMARY","unitOfMeasure":"percentage of patients"}