Trial Outcomes & Findings for Investigation of Metformin for the Prevention of Progression of Precursor Multiple Myeloma (NCT NCT04850846)
NCT ID: NCT04850846
Last Updated: 2026-02-09
Results Overview
Assessed by using the serum-protein electrophoresis
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
60 participants
Primary outcome timeframe
Baseline to 6-months
Results posted on
2026-02-09
Participant Flow
Participant milestones
| Measure |
Metformin
Randomly assigned participants receive a stepped dose escalation until target daily dose of 1500mg Metformin XR is reached (3 x 500mg pills/day). The intervention duration will last an additional 6 months.
Metformin Extension:
Participants will have the option of unblinding at the end of their 6months treatment and those who were randomly assigned to the metformin experimental arm can continue taking metformin if they opt in. The extended intervention duration will last an additional 6 months. (1500mg Metformin XR or highest tolerated dose )
Metformin XR: Orally by mouth
|
Placebo
Randomly assigned participants receive a stepped dose escalation until target daily dose of 3 pills/day is reached. The intervention duration will last 6 months.
Placebo: Orally by mouth
|
|---|---|---|
|
Overall Study
STARTED
|
30
|
30
|
|
Overall Study
COMPLETED
|
22
|
23
|
|
Overall Study
NOT COMPLETED
|
8
|
7
|
Reasons for withdrawal
| Measure |
Metformin
Randomly assigned participants receive a stepped dose escalation until target daily dose of 1500mg Metformin XR is reached (3 x 500mg pills/day). The intervention duration will last an additional 6 months.
Metformin Extension:
Participants will have the option of unblinding at the end of their 6months treatment and those who were randomly assigned to the metformin experimental arm can continue taking metformin if they opt in. The extended intervention duration will last an additional 6 months. (1500mg Metformin XR or highest tolerated dose )
Metformin XR: Orally by mouth
|
Placebo
Randomly assigned participants receive a stepped dose escalation until target daily dose of 3 pills/day is reached. The intervention duration will last 6 months.
Placebo: Orally by mouth
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
2
|
|
Overall Study
Adverse Event
|
3
|
1
|
|
Overall Study
Subject stopped taking medication
|
2
|
4
|
|
Overall Study
Other
|
2
|
0
|
Baseline Characteristics
Investigation of Metformin for the Prevention of Progression of Precursor Multiple Myeloma
Baseline characteristics by cohort
| Measure |
Placebo
n=30 Participants
Randomly assigned participants receive a stepped dose escalation until target daily dose of 3 pills/day is reached. The intervention duration will last 6 months.
Placebo: Orally by mouth
|
Metformin
n=30 Participants
Randomly assigned participants receive a stepped dose escalation until target daily dose of 1500mg Metformin XR is reached (3 x 500mg pills/day). The intervention duration will last an additional 6 months.
Metformin Extension:
Participants will have the option of unblinding at the end of their 6months treatment and those who were randomly assigned to the metformin experimental arm can continue taking metformin if they opt in. The extended intervention duration will last an additional 6 months. (1500mg Metformin XR or highest tolerated dose )
Metformin XR: Orally by mouth
|
Total
n=60 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
65 years
n=1581 Participants
|
65 years
n=41 Participants
|
65 years
n=4626 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=1581 Participants
|
16 Participants
n=41 Participants
|
30 Participants
n=4626 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=1581 Participants
|
14 Participants
n=41 Participants
|
30 Participants
n=4626 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=1581 Participants
|
0 Participants
n=41 Participants
|
1 Participants
n=4626 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
29 Participants
n=1581 Participants
|
29 Participants
n=41 Participants
|
58 Participants
n=4626 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=1581 Participants
|
1 Participants
n=41 Participants
|
1 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=1581 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=1581 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=1581 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=1581 Participants
|
2 Participants
n=41 Participants
|
4 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
White
|
25 Participants
n=1581 Participants
|
26 Participants
n=41 Participants
|
51 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=1581 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=4626 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=1581 Participants
|
2 Participants
n=41 Participants
|
5 Participants
n=4626 Participants
|
|
Region of Enrollment
United States
|
30 participants
n=1581 Participants
|
30 participants
n=41 Participants
|
60 participants
n=4626 Participants
|
|
Diagnosis
MGUS
|
12 Participants
n=1581 Participants
|
11 Participants
n=41 Participants
|
23 Participants
n=4626 Participants
|
|
Diagnosis
SMM
|
18 Participants
n=1581 Participants
|
19 Participants
n=41 Participants
|
37 Participants
n=4626 Participants
|
PRIMARY outcome
Timeframe: Baseline to 6-monthsPopulation: 8 participants in the metformin arm and 6 participants in the control arm did not have analyzable m-protein data.
Assessed by using the serum-protein electrophoresis
Outcome measures
| Measure |
Metformin
n=22 Participants
Randomly assigned participants receive a stepped dose escalation until target daily dose of 1500mg Metformin XR is reached (3 x 500mg pills/day). The intervention duration will last an additional 6 months.
Metformin Extension:
Participants will have the option of unblinding at the end of their 6months treatment and those who were randomly assigned to the metformin experimental arm can continue taking metformin if they opt in. The extended intervention duration will last an additional 6 months. (1500mg Metformin XR or highest tolerated dose )
Metformin XR: Orally by mouth
|
Placebo
n=24 Participants
Randomly assigned participants receive a stepped dose escalation until target daily dose of 3 pills/day is reached. The intervention duration will last 6 months.
Placebo: Orally by mouth
|
|---|---|---|
|
(M-)Protein Concentrations Change
|
-3.2 percent change
Interval -33.0 to 20.4
|
7.7 percent change
Interval -15.4 to 29.7
|
SECONDARY outcome
Timeframe: Baseline to 6-monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to 6-monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to 6-monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to 6-monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to 6-monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to 6-monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline to 6-monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline- 12 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline- 12 monthsOutcome measures
Outcome data not reported
Adverse Events
Metformin
Serious events: 1 serious events
Other events: 26 other events
Deaths: 0 deaths
Placebo
Serious events: 1 serious events
Other events: 18 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Metformin
n=30 participants at risk
Randomly assigned participants receive a stepped dose escalation until target daily dose of 1500mg Metformin XR is reached (3 x 500mg pills/day). The intervention duration will last an additional 6 months.
Metformin Extension:
Participants will have the option of unblinding at the end of their 6months treatment and those who were randomly assigned to the metformin experimental arm can continue taking metformin if they opt in. The extended intervention duration will last an additional 6 months. (1500mg Metformin XR or highest tolerated dose ) Metformin XR: Orally by mouth
|
Placebo
n=30 participants at risk
Randomly assigned participants receive a stepped dose escalation until target daily dose of 3 pills/day is reached. The intervention duration will last 6 months.
Placebo: Orally by mouth
|
|---|---|---|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
0.00%
0/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
3.3%
1/30 • Number of events 1 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Gastrointestinal disorders
Bloating
|
3.3%
1/30 • Number of events 1 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
0.00%
0/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Infections and infestations
Sepsis
|
0.00%
0/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
3.3%
1/30 • Number of events 1 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
Other adverse events
| Measure |
Metformin
n=30 participants at risk
Randomly assigned participants receive a stepped dose escalation until target daily dose of 1500mg Metformin XR is reached (3 x 500mg pills/day). The intervention duration will last an additional 6 months.
Metformin Extension:
Participants will have the option of unblinding at the end of their 6months treatment and those who were randomly assigned to the metformin experimental arm can continue taking metformin if they opt in. The extended intervention duration will last an additional 6 months. (1500mg Metformin XR or highest tolerated dose ) Metformin XR: Orally by mouth
|
Placebo
n=30 participants at risk
Randomly assigned participants receive a stepped dose escalation until target daily dose of 3 pills/day is reached. The intervention duration will last 6 months.
Placebo: Orally by mouth
|
|---|---|---|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.7%
2/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
0.00%
0/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Gastrointestinal disorders
Vomiting
|
6.7%
2/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
0.00%
0/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Gastrointestinal disorders
Nausea
|
46.7%
14/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
30.0%
9/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Gastrointestinal disorders
Diarrhea
|
46.7%
14/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
16.7%
5/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Gastrointestinal disorders
Constipation
|
26.7%
8/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
10.0%
3/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Nervous system disorders
Headache
|
20.0%
6/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
16.7%
5/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Gastrointestinal disorders
Bloating
|
26.7%
8/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
6.7%
2/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Gastrointestinal disorders
Flatulence
|
13.3%
4/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
13.3%
4/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Metabolism and nutrition disorders
Anorexia
|
20.0%
6/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
3.3%
1/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Gastrointestinal disorders
Dyspepsia
|
10.0%
3/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
10.0%
3/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Gastrointestinal disorders
Abdominal pain
|
6.7%
2/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
10.0%
3/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
General disorders
Fatigue
|
10.0%
3/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
3.3%
1/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Investigations
Creatinine increased
|
6.7%
2/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
3.3%
1/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Nervous system disorders
Dizziness
|
6.7%
2/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
3.3%
1/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Nervous system disorders
Dysgeusia
|
6.7%
2/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
3.3%
1/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Investigations
Weight loss
|
10.0%
3/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
0.00%
0/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
|
Gastrointestinal disorders
Belching
|
0.00%
0/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
6.7%
2/30 • Adverse events were collected from the time of informed consent, through 30 days after the last dose of study drug, up to 7 months for the initial randomized placebo-controlled phase and up to 13 months for participants who continued onto the extension phase
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place