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NCT01897714 · OUTCOME

Overall Response Rate (ORR)

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
NUMBER
Unit
percentage of patients
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

ORR was defined as percentage of patients with an overall response (OR), defined as first occurrence of confirmed disease response including PR or better (i.e, PR, VGPR, CR or sCR). Starting on completion of Cycle 2, response was assessed according to IMWG criteria based on Investigator's assessment for all patients at every cycle during treatment period. VGPR was defined as serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥90% reduction in serum plus urine M-protein level \<100 mg/24hr and \>90% decrease in difference between involved and uninvolved free light chain (FLC) levels (only in FLC diseased patients). CR was defined as negative immunofixation on serum and urine, loss of any soft tissue plasmacytomas, \<5% plasma cells in bone marrow and normal FLC ratio of 0.26 to 1.65 (only in FLC diseased patients). sCR was defined as CR plus normal FLC ratio and absence of clonal cells in bone marrow by immunohistochemistry or 2 to 4 color flow cytometry.

Analysis population: For Phase I arms: The Safety Analysis Set included all patients who received at least 1 dose, or part thereof, of study drug. For 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.

Groups in this outcome

Phase I: Melflufen 15 mg + Dexamethasone

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.

Phase I: Melflufen 25 mg + Dexamethasone

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.

Phase I: 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 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.

Phase I: Melflufen 55 mg + Dexamethasone

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.

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: Melflufen 15 mg + Dexamethasone4
Phase I: Melflufen 25 mg + Dexamethasone7
Phase I: Melflufen 40 mg + Dexamethasone6
Phase I: Melflufen 55 mg + Dexamethasone6
Phase I + II: Melflufen 40 mg + Dexamethasone45
Phase II: Melflufen 40 mg (Single Agent)13

Reported measurements

Source class 1
Values in percentage of patients · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
Phase I: Melflufen 15 mg + Dexamethasone0.0Not reported0.060.2Not reported
Phase I: Melflufen 25 mg + Dexamethasone0.0Not reported0.041.0Not reported
Phase I: Melflufen 40 mg + Dexamethasone66.7Not reported22.395.7Not reported
Phase I: Melflufen 55 mg + Dexamethasone16.7Not reported0.464.1Not reported
Phase I + II: Melflufen 40 mg + Dexamethasone31.1Not reported18.246.6Not reported
Phase II: Melflufen 40 mg (Single Agent)7.7Not reported0.236.0Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        0.0
        upper Limit
        60.2
        value
        0.0
      2. group Id
        OG001
        lower Limit
        0.0
        upper Limit
        41.0
        value
        0.0
      3. group Id
        OG002
        lower Limit
        22.3
        upper Limit
        95.7
        value
        66.7
      4. group Id
        OG003
        lower Limit
        0.4
        upper Limit
        64.1
        value
        16.7
      5. group Id
        OG004
        lower Limit
        18.2
        upper Limit
        46.6
        value
        31.1
      6. group Id
        OG005
        lower Limit
        0.2
        upper Limit
        36.0
        value
        7.7
denoms
  1. counts
    1. group Id
      OG000
      value
      4
    2. group Id
      OG001
      value
      7
    3. group Id
      OG002
      value
      6
    4. group Id
      OG003
      value
      6
    5. group Id
      OG004
      value
      45
    6. group Id
      OG005
      value
      13
    units
    Participants
description
ORR was defined as percentage of patients with an overall response (OR), defined as first occurrence of confirmed disease response including PR or better (i.e, PR, VGPR, CR or sCR). Starting on completion of Cycle 2, response was assessed according to IMWG criteria based on Investigator's assessment for all patients at every cycle during treatment period. VGPR was defined as serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥90% reduction in serum plus urine M-protein level \<100 mg/24hr and \>90% decrease in difference between involved and uninvolved free light chain (FLC) levels (only in FLC diseased patients). CR was defined as negative immunofixation on serum and urine, loss of any soft tissue plasmacytomas, \<5% plasma cells in bone marrow and normal FLC ratio of 0.26 to 1.65 (only in FLC diseased patients). sCR was defined as CR plus normal FLC ratio and absence of clonal cells in bone marrow by immunohistochemistry or 2 to 4 color flow cytometry.
dispersion Type
95% Confidence Interval
groups
  1. 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
  2. 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
  3. 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
  4. 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
  5. 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
  6. 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)
param Type
NUMBER
population Description
For Phase I arms: The Safety Analysis Set included all patients who received at least 1 dose, or part thereof, of study drug. For 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.
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
Overall Response Rate (ORR)
type
PRIMARY
unit Of Measure
percentage of patients

Download exact source JSON → · Snapshot ctgov-results-25a395029948b04d52b1253f

Preserved source evidence · Not medical advice