Trial Outcomes & Findings for Melphalan, Prednisone, and Lenalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma (NCT NCT00477750)

NCT ID: NCT00477750

Last Updated: 2019-10-11

Results Overview

Response that was confirmed on 2 consecutive evaluations.\> * Complete Response (CR): Complete disappearance of M-protein from serum and urine on immunofixations, normalization of Free Light Chain (FLC) ratio and \<=5% plasma cells in bone marrow\> * Very Good Partial Response (VGPR): \>=90% reduction in serum M-spike, Urine M-spike \<100mg per 24 hours\> * Partial Response (PR): \>=50% reduction in serum M-spike, Urine M-spike \>=90% reduction or \< 200mg per 24 hours, or \>=50% decrease in difference between involved and uninvolved FLC levels or 50% decrease in bone marrow plasma cells

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

33 participants

Primary outcome timeframe

Every cycle during treatment

Results posted on

2019-10-11

Participant Flow

This was as Phase I/II trial. There were 7 patients recruited to the Phase I portion; no patients qualified for the Phase II portion. Twenty-six (26) patients were recruited for the Phase II portion. Results presented here are on the 26 Phase II patients.

Participant milestones

Participant milestones
Measure
Treatment (Lenalidomide, Melphalan, Prednisone)
Intervention: Drug: lenalidomide 10 mg orally days 1-21 every 28 days until progression Intervention: Drug: melphalan 5mg/m\^2 orally days 1-4 every 28 days until progression Intervention: Drug: prednisone 60mg/m\^2, orally days 1-4 every 28 days until progression
Overall Study
STARTED
26
Overall Study
COMPLETED
15
Overall Study
NOT COMPLETED
11

Reasons for withdrawal

Reasons for withdrawal
Measure
Treatment (Lenalidomide, Melphalan, Prednisone)
Intervention: Drug: lenalidomide 10 mg orally days 1-21 every 28 days until progression Intervention: Drug: melphalan 5mg/m\^2 orally days 1-4 every 28 days until progression Intervention: Drug: prednisone 60mg/m\^2, orally days 1-4 every 28 days until progression
Overall Study
Currently Receiving Treatment
11

Baseline Characteristics

Melphalan, Prednisone, and Lenalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Treatment (Lenalidomide, Melphalan, Prednisone)
n=26 Participants
Intervention: Drug: lenalidomide 10 mg orally days 1-21 every 28 days until progression Intervention: Drug: melphalan 5mg/m\^2 orally days 1-4 every 28 days until progression Intervention: Drug: prednisone 60mg/m\^2, orally days 1-4 every 28 days until progression
Age, Continuous
73.5 years
n=5 Participants
Sex: Female, Male
Female
9 Participants
n=5 Participants
Sex: Female, Male
Male
17 Participants
n=5 Participants
Region of Enrollment
United States
26 participants
n=5 Participants
Durie-Salmon Stage at Diagnosis
I - Low Cell Mass
1 participants
n=5 Participants
Durie-Salmon Stage at Diagnosis
II - Intermediate Cell Mass
11 participants
n=5 Participants
Durie-Salmon Stage at Diagnosis
III - High Cell Mass
11 participants
n=5 Participants
Durie-Salmon Stage at Diagnosis
unknown
3 participants
n=5 Participants
Parameter of Hematologic Response - Serum M-spike >= 1g/dL
Yes
20 participants
n=5 Participants
Parameter of Hematologic Response - Serum M-spike >= 1g/dL
No
6 participants
n=5 Participants
Parameter of Hematologic Response - Serum Immunoglobulin Free Light Chain >= 10mg/dL
Yes
12 participants
n=5 Participants
Parameter of Hematologic Response - Serum Immunoglobulin Free Light Chain >= 10mg/dL
No
14 participants
n=5 Participants
Parameter of Hematologic Response - Urine M-spike >= 200 mg/24 hours
Yes
6 participants
n=5 Participants
Parameter of Hematologic Response - Urine M-spike >= 200 mg/24 hours
No
20 participants
n=5 Participants
Parameter of Hematologic Response - None (non-secretory myeloma, bone marrow only)
Yes
0 participants
n=5 Participants
Parameter of Hematologic Response - None (non-secretory myeloma, bone marrow only)
No
26 participants
n=5 Participants

PRIMARY outcome

Timeframe: Every cycle during treatment

Response that was confirmed on 2 consecutive evaluations.\> * Complete Response (CR): Complete disappearance of M-protein from serum and urine on immunofixations, normalization of Free Light Chain (FLC) ratio and \<=5% plasma cells in bone marrow\> * Very Good Partial Response (VGPR): \>=90% reduction in serum M-spike, Urine M-spike \<100mg per 24 hours\> * Partial Response (PR): \>=50% reduction in serum M-spike, Urine M-spike \>=90% reduction or \< 200mg per 24 hours, or \>=50% decrease in difference between involved and uninvolved FLC levels or 50% decrease in bone marrow plasma cells

Outcome measures

Outcome measures
Measure
Treatment (Lenalidomide, Melphalan, Prednisone)
n=26 Participants
Intervention: Drug: lenalidomide 10 mg orally days 1-21 every 28 days until progression\> \> Intervention: Drug: melphalan 5mg/m\^2 orally days 1-4 every 28 days until progression\> \> Intervention: Drug: prednisone 60mg/m\^2, orally days 1-4 every 28 days until progression
Patients With Overall Confirmed Response
CR
3 participants
Patients With Overall Confirmed Response
VGPR
5 participants
Patients With Overall Confirmed Response
PR
10 participants

SECONDARY outcome

Timeframe: registration to progressive disease (up to 3 years)

Population: Phase 2 patients

Progression free survival (PFS) is defined as the time from the date of randomization to the date of disease progression or death resulting from any cause, whichever comes first. Progression was defined as any one or more of the following: An increase of 25% from lowest confirmed response in: * Serum M-component (absolute increase \>= 0.5g/dl) * Urine M-component (absolute increase \>= 200mg/24hour * Difference between involved and uninvolved Free Light Chain levels (absolute increase \>= 10mg/dl * Bone marrow plasma cell percentage (absolute increase of \>=10%)

Outcome measures

Outcome measures
Measure
Treatment (Lenalidomide, Melphalan, Prednisone)
n=26 Participants
Intervention: Drug: lenalidomide 10 mg orally days 1-21 every 28 days until progression\> \> Intervention: Drug: melphalan 5mg/m\^2 orally days 1-4 every 28 days until progression\> \> Intervention: Drug: prednisone 60mg/m\^2, orally days 1-4 every 28 days until progression
Progression-free Survival
21.4 months
Interval 15.3 to 23.5

SECONDARY outcome

Timeframe: registration to death (up to 3 years)

Population: Phase 2 patients.

OS was defined as the time from registration to death due to any cause. Patients who were alive were censored at date of last follow-up. The overall survival at 3 years (a percentage) is reported below.

Outcome measures

Outcome measures
Measure
Treatment (Lenalidomide, Melphalan, Prednisone)
n=26 Participants
Intervention: Drug: lenalidomide 10 mg orally days 1-21 every 28 days until progression\> \> Intervention: Drug: melphalan 5mg/m\^2 orally days 1-4 every 28 days until progression\> \> Intervention: Drug: prednisone 60mg/m\^2, orally days 1-4 every 28 days until progression
Overall Survival (OS) at 3 Years
58 percentage of patients
Interval 42.0 to 80.0

SECONDARY outcome

Timeframe: from first response to progression or death (up to 3 years)

Population: Phase 2 Patients who had a response of PR or better are included in this analysis.

Duration of response was calculated from documentation of first response to date of progression in the subset of patients who responded. Patients without progression were censored at the date of last tumor evaluation.

Outcome measures

Outcome measures
Measure
Treatment (Lenalidomide, Melphalan, Prednisone)
n=18 Participants
Intervention: Drug: lenalidomide 10 mg orally days 1-21 every 28 days until progression\> \> Intervention: Drug: melphalan 5mg/m\^2 orally days 1-4 every 28 days until progression\> \> Intervention: Drug: prednisone 60mg/m\^2, orally days 1-4 every 28 days until progression
Duration of Response (DOR)
16.3 months
Interval 14.3 to 26.4

SECONDARY outcome

Timeframe: Every cycle during treatment up to 3 years

Population: Patients who experienced an adverse event that is at least possibly related are included in this analysis.

The overall toxicity rates (percentages) for grade 3 or higher adverse events considered at least possibly related to treatment are reported below.

Outcome measures

Outcome measures
Measure
Treatment (Lenalidomide, Melphalan, Prednisone)
n=3 Participants
Intervention: Drug: lenalidomide 10 mg orally days 1-21 every 28 days until progression\> \> Intervention: Drug: melphalan 5mg/m\^2 orally days 1-4 every 28 days until progression\> \> Intervention: Drug: prednisone 60mg/m\^2, orally days 1-4 every 28 days until progression
Percentage of Participants With Toxicity, Assessed Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0 (v3)
Grade 3
33 percentage of patients
Percentage of Participants With Toxicity, Assessed Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0 (v3)
Grade 4
67 percentage of patients
Percentage of Participants With Toxicity, Assessed Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0 (v3)
Grade 5
0 percentage of patients

Adverse Events

Treatment (Lenalidomide, Melphalan, Prednisone)

Serious events: 7 serious events
Other events: 26 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Treatment (Lenalidomide, Melphalan, Prednisone)
n=26 participants at risk
Intervention: Drug: lenalidomide 10 mg orally days 1-21 every 28 days until progression Intervention: Drug: melphalan 5mg/m\^2 orally days 1-4 every 28 days until progression Intervention: Drug: prednisone 60mg/m\^2, orally days 1-4 every 28 days until progression
Gastrointestinal disorders
Diarrhea
3.8%
1/26 • Number of events 1
Infections and infestations
Sepsis
3.8%
1/26 • Number of events 1
Infections and infestations
Skin infection
3.8%
1/26 • Number of events 1
Investigations
Creatinine increased
3.8%
1/26 • Number of events 1
Investigations
Leukocyte count decreased
3.8%
1/26 • Number of events 1
Investigations
Neutrophil count decreased
7.7%
2/26 • Number of events 2
Investigations
Platelet count decreased
7.7%
2/26 • Number of events 2
Metabolism and nutrition disorders
Dehydration
3.8%
1/26 • Number of events 1
Skin and subcutaneous tissue disorders
Rash desquamating
3.8%
1/26 • Number of events 1

Other adverse events

Other adverse events
Measure
Treatment (Lenalidomide, Melphalan, Prednisone)
n=26 participants at risk
Intervention: Drug: lenalidomide 10 mg orally days 1-21 every 28 days until progression Intervention: Drug: melphalan 5mg/m\^2 orally days 1-4 every 28 days until progression Intervention: Drug: prednisone 60mg/m\^2, orally days 1-4 every 28 days until progression
Blood and lymphatic system disorders
Hemoglobin decreased
92.3%
24/26 • Number of events 103
Blood and lymphatic system disorders
Lymphatic disorder
3.8%
1/26 • Number of events 1
Cardiac disorders
Myocardial ischemia
3.8%
1/26 • Number of events 1
Gastrointestinal disorders
Abdominal pain
3.8%
1/26 • Number of events 1
Gastrointestinal disorders
Constipation
3.8%
1/26 • Number of events 1
Gastrointestinal disorders
Dyspepsia
15.4%
4/26 • Number of events 5
Gastrointestinal disorders
Ear, nose and throat examination abnormal
7.7%
2/26 • Number of events 2
Gastrointestinal disorders
Nausea
30.8%
8/26 • Number of events 12
General disorders
Edema limbs
42.3%
11/26 • Number of events 27
General disorders
Fatigue
80.8%
21/26 • Number of events 63
General disorders
Fever
3.8%
1/26 • Number of events 1
General disorders
Pain
7.7%
2/26 • Number of events 5
Immune system disorders
Hypersensitivity
3.8%
1/26 • Number of events 1
Infections and infestations
Colitis, infectious (e.g., Clostridium difficile)
3.8%
1/26 • Number of events 1
Infections and infestations
Pneumonia
3.8%
1/26 • Number of events 1
Infections and infestations
Skin infection
7.7%
2/26 • Number of events 5
Infections and infestations
Upper respiratory infection
3.8%
1/26 • Number of events 1
Infections and infestations
Urinary tract infection
3.8%
1/26 • Number of events 1
Investigations
Alkaline phosphatase increased
3.8%
1/26 • Number of events 1
Investigations
Aspartate aminotransferase increased
3.8%
1/26 • Number of events 1
Investigations
Blood bilirubin increased
3.8%
1/26 • Number of events 1
Investigations
Leukocyte count decreased
23.1%
6/26 • Number of events 13
Investigations
Lymphocyte count decreased
3.8%
1/26 • Number of events 1
Investigations
Neutrophil count decreased
69.2%
18/26 • Number of events 58
Investigations
Platelet count decreased
69.2%
18/26 • Number of events 70
Metabolism and nutrition disorders
Blood glucose increased
57.7%
15/26 • Number of events 32
Metabolism and nutrition disorders
Serum albumin decreased
3.8%
1/26 • Number of events 1
Metabolism and nutrition disorders
Serum calcium decreased
3.8%
1/26 • Number of events 1
Metabolism and nutrition disorders
Serum glucose decreased
3.8%
1/26 • Number of events 1
Metabolism and nutrition disorders
Serum potassium decreased
3.8%
1/26 • Number of events 1
Musculoskeletal and connective tissue disorders
Muscle weakness
34.6%
9/26 • Number of events 17
Musculoskeletal and connective tissue disorders
Pain in extremity
3.8%
1/26 • Number of events 1
Nervous system disorders
Headache
3.8%
1/26 • Number of events 1
Nervous system disorders
Peripheral sensory neuropathy
19.2%
5/26 • Number of events 8
Nervous system disorders
Syncope
7.7%
2/26 • Number of events 2
Psychiatric disorders
Confusion
3.8%
1/26 • Number of events 1
Skin and subcutaneous tissue disorders
Rash desquamating
23.1%
6/26 • Number of events 14
Vascular disorders
Hypotension
3.8%
1/26 • Number of events 1

Additional Information

Dr. Vivek Roy

Mayo Clinic

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place