Trial Outcomes & Findings for Feasibility Study of Metformin Therapy in ADPKD (NCT NCT02903511)
NCT ID: NCT02903511
Last Updated: 2021-09-05
Results Overview
Percentage of participants who at the end of 12 months are still prescribed the full randomized dose of metformin or placebo, and the percentage of participants who are prescribed at least 50% of the randomized dose
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
56 participants
Primary outcome timeframe
12 months
Results posted on
2021-09-05
Participant Flow
5 participants screen failed or dropped out prior to randomization
Participant milestones
| Measure |
Metformin
Participants will receive metformin 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Metformin: Monitoring of safety and tolerability
|
Placebo
Participants will receive placebo 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Placebo: Monitoring of safety and tolerability
|
|---|---|---|
|
Overall Study
STARTED
|
26
|
25
|
|
Overall Study
Received Intervention
|
25
|
25
|
|
Overall Study
COMPLETED
|
22
|
23
|
|
Overall Study
NOT COMPLETED
|
4
|
2
|
Reasons for withdrawal
| Measure |
Metformin
Participants will receive metformin 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Metformin: Monitoring of safety and tolerability
|
Placebo
Participants will receive placebo 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Placebo: Monitoring of safety and tolerability
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
|
Overall Study
Lost to Follow-up
|
3
|
2
|
Baseline Characteristics
Feasibility Study of Metformin Therapy in ADPKD
Baseline characteristics by cohort
| Measure |
Metformin
n=26 Participants
Participants will receive metformin 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Metformin: Monitoring of safety and tolerability
|
Placebo
n=25 Participants
Participants will receive placebo 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Placebo: Monitoring of safety and tolerability
|
Total
n=51 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
48 years
STANDARD_DEVIATION 8 • n=93 Participants
|
48 years
STANDARD_DEVIATION 7 • n=4 Participants
|
48 years
STANDARD_DEVIATION 8 • n=27 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=93 Participants
|
17 Participants
n=4 Participants
|
32 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
19 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
26 Participants
n=93 Participants
|
24 Participants
n=4 Participants
|
50 Participants
n=27 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
White
|
26 Participants
n=93 Participants
|
25 Participants
n=4 Participants
|
51 Participants
n=27 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Region of Enrollment
United States
|
26 participants
n=93 Participants
|
25 participants
n=4 Participants
|
51 participants
n=27 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPercentage of participants who at the end of 12 months are still prescribed the full randomized dose of metformin or placebo, and the percentage of participants who are prescribed at least 50% of the randomized dose
Outcome measures
| Measure |
Metformin
n=22 Participants
Participants will receive metformin 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Metformin: Monitoring of safety and tolerability
|
Placebo
n=23 Participants
Participants will receive placebo 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Placebo: Monitoring of safety and tolerability
|
|---|---|---|
|
Safety and Tolerability of Metformin
Full Dose
|
50 percentage of participants
|
100 percentage of participants
|
|
Safety and Tolerability of Metformin
50% Dose
|
82 percentage of participants
|
100 percentage of participants
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: 2 participants (1 from each group) were not analyzed due to insufficient image quality for accurate analysis.
Total kidney volume will be measured by MRI (magnetic resonance imaging) at baseline and at 12 months. Percentage change from baseline in height-adjusted total kidney volume is reported.
Outcome measures
| Measure |
Metformin
n=21 Participants
Participants will receive metformin 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Metformin: Monitoring of safety and tolerability
|
Placebo
n=22 Participants
Participants will receive placebo 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Placebo: Monitoring of safety and tolerability
|
|---|---|---|
|
Change in Total Kidney Volume
|
3.45 percent change
Standard Error 1.3
|
3.15 percent change
Standard Error 1.61
|
SECONDARY outcome
Timeframe: 12 monthsEstimated glomerular filtration rate (eGFR) will be calculated from serum creatinine measurements at baseline and after 3, 6, 9 and 12 months. Change from baseline at 12 months is reported.
Outcome measures
| Measure |
Metformin
n=22 Participants
Participants will receive metformin 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Metformin: Monitoring of safety and tolerability
|
Placebo
n=23 Participants
Participants will receive placebo 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Placebo: Monitoring of safety and tolerability
|
|---|---|---|
|
Change in Kidney Function
|
-0.41 mL/min/1.73 m^2
Standard Error 1.81
|
-3.35 mL/min/1.73 m^2
Standard Error 1.70
|
SECONDARY outcome
Timeframe: 12 monthsSerious adverse events occurring from the time of signing informed consent until the end of the study will be monitored in both treatment arms
Outcome measures
| Measure |
Metformin
n=26 Participants
Participants will receive metformin 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Metformin: Monitoring of safety and tolerability
|
Placebo
n=25 Participants
Participants will receive placebo 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Placebo: Monitoring of safety and tolerability
|
|---|---|---|
|
Rate of Serious Adverse Events (SAE)
|
2 Participants
|
0 Participants
|
Adverse Events
Metformin
Serious events: 2 serious events
Other events: 26 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 25 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Metformin
n=26 participants at risk
Participants will receive metformin 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Metformin: Monitoring of safety and tolerability
|
Placebo
n=25 participants at risk
Participants will receive placebo 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Placebo: Monitoring of safety and tolerability
|
|---|---|---|
|
Infections and infestations
Infection
|
7.7%
2/26 • Number of events 2 • 12 Months
|
0.00%
0/25 • 12 Months
|
Other adverse events
| Measure |
Metformin
n=26 participants at risk
Participants will receive metformin 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Metformin: Monitoring of safety and tolerability
|
Placebo
n=25 participants at risk
Participants will receive placebo 500 mg tablets, starting with 1 tab twice a day. The dose will be increased by 500 mg every 2 weeks up to 1000 mg by mouth twice a day, as tolerated, for 12 months.
Placebo: Monitoring of safety and tolerability
|
|---|---|---|
|
Gastrointestinal disorders
diarrhea
|
61.5%
16/26 • Number of events 16 • 12 Months
|
28.0%
7/25 • Number of events 7 • 12 Months
|
|
Gastrointestinal disorders
Nausea
|
42.3%
11/26 • Number of events 11 • 12 Months
|
16.0%
4/25 • Number of events 4 • 12 Months
|
|
Gastrointestinal disorders
Vomiting
|
26.9%
7/26 • Number of events 7 • 12 Months
|
12.0%
3/25 • Number of events 3 • 12 Months
|
|
Gastrointestinal disorders
Acid Reflux
|
7.7%
2/26 • Number of events 2 • 12 Months
|
16.0%
4/25 • Number of events 4 • 12 Months
|
|
General disorders
Light-headedness
|
23.1%
6/26 • Number of events 6 • 12 Months
|
16.0%
4/25 • Number of events 4 • 12 Months
|
|
General disorders
Weakness
|
19.2%
5/26 • Number of events 5 • 12 Months
|
0.00%
0/25 • 12 Months
|
|
Infections and infestations
Viral Illness
|
19.2%
5/26 • Number of events 5 • 12 Months
|
28.0%
7/25 • Number of events 7 • 12 Months
|
|
Infections and infestations
sinus infection
|
19.2%
5/26 • Number of events 5 • 12 Months
|
8.0%
2/25 • Number of events 2 • 12 Months
|
|
Gastrointestinal disorders
Flatulence
|
15.4%
4/26 • Number of events 4 • 12 Months
|
0.00%
0/25 • 12 Months
|
|
Gastrointestinal disorders
Abdominal cramping/pain
|
7.7%
2/26 • Number of events 2 • 12 Months
|
12.0%
3/25 • Number of events 3 • 12 Months
|
Additional Information
Godela Brosnahan, MD
University of Colorado Denver | Anschutz
Phone: 3037241111
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place