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NCT01897714 · OUTCOME

Time to Disease Response in Patients Who Achieved OR and CBR

Safety and Efficacy of Melflufen and Dexamethasone in Relapsed and/or Relapsed-Refractory Multiple Myeloma Patients · Source last updated 2020-10-23

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
MEDIAN
Unit
months
Interval / dispersion
95% Confidence Interval
Time frame
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.

What was measured

Time to first OR was defined as the time from the date of the first dose of study drug (overall reference start date) to the date of the first occurrence of PR or better (first of 2 consecutive assessments-confirmed response). Time to first CBR was defined as the time from the date of the first dose of study drug (overall reference start date) to the date of the first occurrence of MR or better (first of 2 consecutive assessments-confirmed response). Time to disease response was estimated using Kaplan-Meier statistics.

Analysis population: 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. Only patients who had achieved OR and CBR were evaluated, for each respective time to response parameter.

Groups in this outcome

Phase I + II: Melflufen 40 mg + Dexamethasone

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.

Phase II: Melflufen 40 mg (Single Agent)

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.

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
Phase I + II: Melflufen 40 mg + Dexamethasone22
Phase II: Melflufen 40 mg (Single Agent)3

Reported measurements

OR

Analysis denominator · Participants

GroupSource count
Phase I + II: Melflufen 40 mg + Dexamethasone14
Phase II: Melflufen 40 mg (Single Agent)1
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Phase I + II: Melflufen 40 mg + Dexamethasone2.8Not reported1.64.7Not reported
Phase II: Melflufen 40 mg (Single Agent)6.7Not reportedNANAThe confidence interval could not be determined as only 1 patient was analysed.
CBR

Analysis denominator · Participants

GroupSource count
Phase I + II: Melflufen 40 mg + Dexamethasone22
Phase II: Melflufen 40 mg (Single Agent)3
Values in months · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Phase I + II: Melflufen 40 mg + Dexamethasone2.4Not reported1.43.0Not reported
Phase II: Melflufen 40 mg (Single Agent)2.8Not reported2.86.1Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        1.6
        upper Limit
        4.7
        value
        2.8
      2. comment
        The confidence interval could not be determined as only 1 patient was analysed.
        group Id
        OG001
        lower Limit
        NA
        upper Limit
        NA
        value
        6.7
    denoms
    1. counts
      1. group Id
        OG000
        value
        14
      2. group Id
        OG001
        value
        1
      units
      Participants
    title
    OR
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        1.4
        upper Limit
        3.0
        value
        2.4
      2. group Id
        OG001
        lower Limit
        2.8
        upper Limit
        6.1
        value
        2.8
    denoms
    1. counts
      1. group Id
        OG000
        value
        22
      2. group Id
        OG001
        value
        3
      units
      Participants
    title
    CBR
denoms
  1. counts
    1. group Id
      OG000
      value
      22
    2. group Id
      OG001
      value
      3
    units
    Participants
description
Time to first OR was defined as the time from the date of the first dose of study drug (overall reference start date) to the date of the first occurrence of PR or better (first of 2 consecutive assessments-confirmed response). Time to first CBR was defined as the time from the date of the first dose of study drug (overall reference start date) to the date of the first occurrence of MR or better (first of 2 consecutive assessments-confirmed response). Time to disease response was estimated using Kaplan-Meier statistics.
dispersion Type
95% Confidence Interval
groups
  1. 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
    OG000
    title
    Phase I + II: Melflufen 40 mg + Dexamethasone
  2. 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
    OG001
    title
    Phase II: Melflufen 40 mg (Single Agent)
param Type
MEDIAN
population Description
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. Only patients who had achieved OR and CBR were evaluated, for each respective time to response parameter.
reporting Status
POSTED
time Frame
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
Time to Disease Response in Patients Who Achieved OR and CBR
type
SECONDARY
unit Of Measure
months

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