NCT02963493 · OUTCOME

Overall Response Rate (ORR)

A Study of Melphalan Flufenamide (Melflufen) Plus Dexamethasone in Patients With Relapsed or Refractory Multiple Myeloma · Source last updated 2022-11-22

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

Measure: COUNT_OF_PARTICIPANTS
Unit: Participants
Interval / dispersion: Not reported
Time frame: Patients were followed until documented progression, unacceptable toxicity, patient/physician decision to withdraw or date of death, whichever came first. Longest time to response in study recorded as 15.3 months. Longest time on treatment 35 months.

What was measured

The overall response rate (ORR) will be estimated as the percentage of patients who achieve sCR, CR, VGPR, or PR as their best response as assessed by the investigator. Response assessed by IMWG (International myeloma working group) criteria sCR-stringent complete response: CR plus Normal FLC (free light chain) ratio and absence of clonal cells in BM CR-complete response: Negative immunofixation in serum/urine; Disappearance of soft tissue plasmacytomas; \<5% plasma cells in BM; If only FLC disease, normal FLC ratio (0.26-1.65) VGPR-very good partial response: Serum/urine M-protein detectable by immunofixation but not electrophoresis or ≥90% reduction in serum M-protein and urine M-protein \<100 mg/24 h; If only FLC disease, \>90% decrease in the difference between involved and uninvolved FLC levels PR-partial response: 50% reduction of serum M-protein and soft tissue plasmacytomas, ≥90% reduction in urinary M-protein or to \<200 mg/24 h; other special cases if M-protein unmeasurable

Groups in this outcome

Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set

Full analysis set Melphalan flufenamide (melflufen) 40 mg Day 1 and dexamethasone 40 mg (reduced dose for patients 75 years or older) on Days 1, 8, 15 and 22 of each 28-day cycle. Melphalan flufenamide (Melflufen) Dexamethasone: IV dexamethasone may be substituted for oral dexamethasone in the US. Oral only in Europe.

Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Extramedullary Disease

Patients with extramedullary disease at baseline defined as myeloma disease either originating in, but extending beyond, the cortical bone or as a separate soft tissue mass

Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease

Triple class refractory population defined as refractory to or intolerant of at least one immunomodulatory drug, at least one proteasome inhibitor, and at least one anti-CD38 monoclonal antibody

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

Analysis denominator · Participants

Group	Source count
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set	157
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Extramedullary Disease	55
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease	119

Reported measurements

Source class 1

Values in Participants · Interval/dispersion: Not reported
Group	Value	Spread	Lower	Upper	Comment
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set	53	Not reported	Not reported	Not reported	Not reported
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Extramedullary Disease	14	Not reported	Not reported	Not reported	Not reported
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease	35	Not reported	Not reported	Not reported	Not reported

Complete source fields

classes

categories
measurements
group Id
OG000
value
53
group Id
OG001
value
14
group Id
OG002
value
35

denoms

counts
group Id
OG000
value
157
group Id
OG001
value
55
group Id
OG002
value
119
units
Participants

description

groups

description
Full analysis set Melphalan flufenamide (melflufen) 40 mg Day 1 and dexamethasone 40 mg (reduced dose for patients 75 years or older) on Days 1, 8, 15 and 22 of each 28-day cycle. Melphalan flufenamide (Melflufen) Dexamethasone: IV dexamethasone may be substituted for oral dexamethasone in the US. Oral only in Europe.
id
OG000
title
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set
description
Patients with extramedullary disease at baseline defined as myeloma disease either originating in, but extending beyond, the cortical bone or as a separate soft tissue mass
id
OG001
title
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Extramedullary Disease
description
Triple class refractory population defined as refractory to or intolerant of at least one immunomodulatory drug, at least one proteasome inhibitor, and at least one anti-CD38 monoclonal antibody
id
OG002
title
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease

param Type

COUNT_OF_PARTICIPANTS

reporting Status

POSTED

time Frame

Patients were followed until documented progression, unacceptable toxicity, patient/physician decision to withdraw or date of death, whichever came first. Longest time to response in study recorded as 15.3 months. Longest time on treatment 35 months.

title

Overall Response Rate (ORR)

type

PRIMARY

unit Of Measure

Participants

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

Preserved source evidence · Not medical advice