Trial Outcomes & Findings for Melphalan, Prednisone, and Thalidomide or Lenalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma (NCT NCT00602641)

NCT ID: NCT00602641

Last Updated: 2025-11-21

Results Overview

PFS is defined as the time from randomization to the earlier of progression or death of any cause.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE3

Target enrollment

306 participants

Primary outcome timeframe

Assessed every 3 months for 2 years, then every 6 months for 3 years, then annually for 10 years from the date of randomization.

Results posted on

2025-11-21

Participant Flow

The study was activated on February 29, 2008 and closed to accrual on November 30, 2011 with final accrual of 306 patients.

Participant milestones

Participant milestones
Measure	Arm I (MPT-T) Patients receive melphalan, prednisone and thalidomide induction plus thalidomide maintenance (MPT-T). INDUCTION THERAPY: Patients receive melphalan 9 mg/m\^2 PO and prednisone 100 mg PO daily on days 1-4, and thalidomide 100 mg PO daily on days 1-28. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive thalidomide 100 mg PO daily and continue in the absence of disease progression.	Arm II (mPR-R) Patients receive lower-dose melphalan, prednisone and lenalidomide (Revlimid®) induction plus lenalidomide maintenance (mPR-R). INDUCTION THERAPY: Patients receive melphalan 5 mg/m\^2 PO and prednisone 100 mg PO daily on days 1-4, and lenalidomide 10 mg PO on days 1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive lenalidomide 10 mg PO daily on days 1-21. Courses repeat every 28 days in the absence of disease progression.
Overall Study STARTED	154	152
Overall Study Started Protocol Therapy	148	150
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	154	152

Reasons for withdrawal

Reasons for withdrawal
Measure	Arm I (MPT-T) Patients receive melphalan, prednisone and thalidomide induction plus thalidomide maintenance (MPT-T). INDUCTION THERAPY: Patients receive melphalan 9 mg/m\^2 PO and prednisone 100 mg PO daily on days 1-4, and thalidomide 100 mg PO daily on days 1-28. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive thalidomide 100 mg PO daily and continue in the absence of disease progression.	Arm II (mPR-R) Patients receive lower-dose melphalan, prednisone and lenalidomide (Revlimid®) induction plus lenalidomide maintenance (mPR-R). INDUCTION THERAPY: Patients receive melphalan 5 mg/m\^2 PO and prednisone 100 mg PO daily on days 1-4, and lenalidomide 10 mg PO on days 1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive lenalidomide 10 mg PO daily on days 1-21. Courses repeat every 28 days in the absence of disease progression.
Overall Study Never started protocol therapy	6	2
Overall Study Still on treatment at time of analysis	8	15
Overall Study Adverse Event	62	53
Overall Study Disease progression	38	55
Overall Study Death	6	5
Overall Study Withdrawal by Subject	15	12
Overall Study Alternative therapy	5	2
Overall Study Other Complicating Disease	5	2
Overall Study Physician Decision	5	4
Overall Study Administrative Closure	1	1
Overall Study Other	3	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm I (MPT-T) n=154 Participants Patients receive melphalan, prednisone and thalidomide induction plus thalidomide maintenance (MPT-T). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and thalidomide PO daily on days 1-28. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive thalidomide PO daily and continue in the absence of disease progression. melphalan: Given PO prednisone: Given PO thalidomide: Given PO	Arm II (mPR-R) n=152 Participants Patients receive lower-dose melphalan, prednisone and lenalidomide (Revlimid®) induction plus lenalidomide maintenance (mPR-R). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and lenalidomide PO on days 1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive lenalidomide PO daily on days 1-21. Courses repeat every 28 days in the absence of disease progression. melphalan: Given PO prednisone: Given PO lenalidomide: Given PO	Total n=306 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=68 Participants	0 Participants n=76 Participants	0 Participants n=48 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=68 Participants	6 Participants n=76 Participants	15 Participants n=48 Participants
Age, Categorical >=65 years	145 Participants n=68 Participants	146 Participants n=76 Participants	291 Participants n=48 Participants
Sex: Female, Male Female	68 Participants n=68 Participants	71 Participants n=76 Participants	139 Participants n=48 Participants
Sex: Female, Male Male	86 Participants n=68 Participants	81 Participants n=76 Participants	167 Participants n=48 Participants
International Staging System (ISS) Stage I	45 participants n=68 Participants	36 participants n=76 Participants	81 participants n=48 Participants
International Staging System (ISS) Stage II	58 participants n=68 Participants	70 participants n=76 Participants	128 participants n=48 Participants
International Staging System (ISS) Stage III	49 participants n=68 Participants	46 participants n=76 Participants	95 participants n=48 Participants
International Staging System (ISS) Unknown/Missing	2 participants n=68 Participants	0 participants n=76 Participants	2 participants n=48 Participants

PRIMARY outcome

Timeframe: Assessed every 3 months for 2 years, then every 6 months for 3 years, then annually for 10 years from the date of randomization.

Population: Intention-to-treat (ITT) population

PFS is defined as the time from randomization to the earlier of progression or death of any cause.

Outcome measures

Outcome measures
Measure	Arm I (MPT-T) n=154 Participants Patients receive melphalan, prednisone and thalidomide induction plus thalidomide maintenance (MPT-T). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and thalidomide PO daily on days 1-28. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive thalidomide PO daily and continue in the absence of disease progression.	Arm II (mPR-R) n=152 Participants Patients receive lower-dose melphalan, prednisone and lenalidomide (Revlimid®) induction plus lenalidomide maintenance (mPR-R). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and lenalidomide PO on days 1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive lenalidomide PO daily on days 1-21. Courses repeat every 28 days in the absence of disease progression.
Progression-Free Survival (PFS)	21.0 months Interval 18.2 to 27.5	18.7 months Interval 16.0 to 22.1

SECONDARY outcome

Timeframe: Assessed every 3 months for 2 years, then every 6 months for 3 years, then annually for 10 years from the date of randomization.

Population: Intention-to-treatment (ITT) population

Overall survival was defined as time from randomization to death from any cause.

Outcome measures

Outcome measures
Measure	Arm I (MPT-T) n=154 Participants Patients receive melphalan, prednisone and thalidomide induction plus thalidomide maintenance (MPT-T). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and thalidomide PO daily on days 1-28. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive thalidomide PO daily and continue in the absence of disease progression.	Arm II (mPR-R) n=152 Participants Patients receive lower-dose melphalan, prednisone and lenalidomide (Revlimid®) induction plus lenalidomide maintenance (mPR-R). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and lenalidomide PO on days 1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive lenalidomide PO daily on days 1-21. Courses repeat every 28 days in the absence of disease progression.
Overall Survival	52.6 months Interval 40.4 to Upper limit of the confidence interval has not been reached.	47.7 months Interval 41.0 to 56.4

SECONDARY outcome

Timeframe: Assessed every cycle (1 cycle=28 days) for the first 12 cycles, and then every 2 cycles while on treatment. Post treatment assessed every 3 months < 2 years from study entry, every 6 months if 2-5 years, every 12 months if 6-10 years from study entry.

Population: Intention-to-treat (ITT) population

Response evaluation was based on the International Myeloma Working Group (IMWG) response criteria. VGPR rate was defined as patients achieving at least VGPR which include patients who achieving complete response (CR) and VGPR. CR refers to patients who have complete disappearance of an M-protein and no evidence of myeloma in the bone marrow. VGPR refers to patients who meet the following criteria: Serum and urine M-component detectable by immunofixation but not on electrophoresis; Or 90% or greater reduction in serum M-component plus urine M-component \<100 mg per 24 hours; If the serum and urine M protein are unmeasurable and the immunoglobulin free light chain parameter is being used to measure response, a ≥ 90% decrease in the difference between involved and uninvolved free light chain (FLC) levels is required in place of the M protein criteria.

Outcome measures

Outcome measures
Measure	Arm I (MPT-T) n=154 Participants Patients receive melphalan, prednisone and thalidomide induction plus thalidomide maintenance (MPT-T). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and thalidomide PO daily on days 1-28. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive thalidomide PO daily and continue in the absence of disease progression.	Arm II (mPR-R) n=152 Participants Patients receive lower-dose melphalan, prednisone and lenalidomide (Revlimid®) induction plus lenalidomide maintenance (mPR-R). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and lenalidomide PO on days 1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive lenalidomide PO daily on days 1-21. Courses repeat every 28 days in the absence of disease progression.
Very Good Partial Response (VGPR) Rate	0.247 proportion of participants Interval 0.179 to 0.315	0.316 proportion of participants Interval 0.242 to 0.39

SECONDARY outcome

Timeframe: Administered at registration, the beginning of cycle 7 d1, the end of cycle 12 d28, then at the end of cycle 18, 24, and 38 d28. For patients who discontinue treatment early, assessed at time of discontinuation and at the next quarterly follow-up visit.

Population: Patients who completed both baseline and cycle 12 QOL assessments.

A combined scale was used to assess the quality of life (QOL) comprising of the well established and validated functional well-being (FWB) and physical well-being (PWB) components of FACT-G version 4 (14 questions), which will address the physical and functional well-being of multiple myeloma patients plus the FACT-neurotoxicity (NTX, 11 questions), which will evaluate symptoms of neurotoxicity. This pooled scale is referred to as the FACT Ntx TOI. The FACT-Ntx TOI has 25 items and the score ranges from 0 (worst possible outcome) to 100 (best possible outcome).

Outcome measures

Outcome measures
Measure	Arm I (MPT-T) n=66 Participants Patients receive melphalan, prednisone and thalidomide induction plus thalidomide maintenance (MPT-T). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and thalidomide PO daily on days 1-28. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive thalidomide PO daily and continue in the absence of disease progression.	Arm II (mPR-R) n=68 Participants Patients receive lower-dose melphalan, prednisone and lenalidomide (Revlimid®) induction plus lenalidomide maintenance (mPR-R). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and lenalidomide PO on days 1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive lenalidomide PO daily on days 1-21. Courses repeat every 28 days in the absence of disease progression.
Change in Functional Assessment of Cancer Therapy-Neurotoxicity Trial Outcome Index (FACT-Ntx TOI) Score From Baseline to Cycle 12	-2.8 units on a scale Standard Deviation 15.0	3.3 units on a scale Standard Deviation 13.7

Adverse Events

MPT-T

Serious events: 110 serious events

Other events: 29 other events

Deaths: 0 deaths

mPR-R

Serious events: 88 serious events

Other events: 34 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	MPT-T n=148 participants at risk Patients receive melphalan, prednisone and thalidomide induction plus thalidomide maintenance (MPT-T). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and thalidomide PO daily on days 1-28. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive thalidomide PO daily and continue in the absence of disease progression.	mPR-R n=151 participants at risk；n=150 participants at risk Patients receive lower-dose melphalan, prednisone and lenalidomide (Revlimid®) induction plus lenalidomide maintenance (mPR-R). INDUCTION THERAPY: Patients receive melphalan PO and prednisone PO daily on days 1-4, and lenalidomide PO on days 1-21. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity. MAINTENANCE THERAPY: Patients receive lenalidomide PO daily on days 1-21. Courses repeat every 28 days in the absence of disease progression.
Blood and lymphatic system disorders Anemia	7.4% 11/148 • Assessed every 28 days while on treatment and for 30 days after the end of treatment	14.6% 22/151 • Assessed every 28 days while on treatment and for 30 days after the end of treatment
Investigations White blood cell decreased	8.1% 12/148 • Assessed every 28 days while on treatment and for 30 days after the end of treatment	11.3% 17/151 • Assessed every 28 days while on treatment and for 30 days after the end of treatment
Investigations Lymphocyte count decreased	6.8% 10/148 • Assessed every 28 days while on treatment and for 30 days after the end of treatment	6.0% 9/151 • Assessed every 28 days while on treatment and for 30 days after the end of treatment
Investigations Neutrophil count decreased	5.4% 8/148 • Assessed every 28 days while on treatment and for 30 days after the end of treatment	7.9% 12/151 • Assessed every 28 days while on treatment and for 30 days after the end of treatment
Investigations Platelet count decreased	6.8% 10/148 • Assessed every 28 days while on treatment and for 30 days after the end of treatment	11.3% 17/151 • Assessed every 28 days while on treatment and for 30 days after the end of treatment
General disorders Fatigue	9.5% 14/148 • Assessed every 28 days while on treatment and for 30 days after the end of treatment	9.9% 15/151 • Assessed every 28 days while on treatment and for 30 days after the end of treatment
Gastrointestinal disorders Constipation	6.8% 10/148 • Assessed every 28 days while on treatment and for 30 days after the end of treatment	6.0% 9/151 • Assessed every 28 days while on treatment and for 30 days after the end of treatment
Nervous system disorders Peripheral sensory neuropathy	5.4% 8/148 • Assessed every 28 days while on treatment and for 30 days after the end of treatment	1.3% 2/151 • Assessed every 28 days while on treatment and for 30 days after the end of treatment

Additional Information

Study Statistician

ECOG Statistical Office

Phone: 617-632-3012

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60