ClinicalTrials.gg
CompletedNCT02656017TAMEUpdated Aug 9, 2022Results posted

Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease

A Phase 2 interventional study of Metformin and Placebo in Polycystic Kidney, Autosomal Dominant, sponsored by Kyongtae Ty Bae, M.D., Ph.D.. Completed at 2 sites in United States. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2022-08-09.

Sponsored by Kyongtae Ty Bae, M.D., Ph.D. · Phase 2, Interventional, and Treatment

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Phase
Phase 2
Study type
Interventional
Enrollment
97
Allocation
Randomized
Ages
18 Years to 60 Years
Sex
All
01

Where this study stands

Counted across the records on this site · 2026-11-13

Among 85 studies in Polycystic Kidney, Autosomal Dominant with a site in United States — 14 open to participants now, 25 under way, 1 of them phase 3. Polycystic Kidney, Autosomal Dominant · United States.

Enrollment
21st of 63
97 · median 48
Started
2016
with 2 others
Phase 2
18 of 85
3 open now
Registered
34th of 85
by date

Among the 85 studies in Polycystic Kidney, Autosomal Dominant with a site in United States, this one is the 21st largest by enrollment of the 63 interventional ones that give a count, is one of 3 that started in 2016, is one of 18 in phase 2, and was the 34th registered.

Polycystic Kidney, Autosomal Dominant

185 studies on the registry are indexed under Polycystic Kidney, Autosomal Dominant; 42 are open to participants now.

Browse Polycystic Kidney, Autosomal Dominant studies →

Open to participants now

14 of the 85 studies in Polycystic Kidney, Autosomal Dominant with a site in United States are open now. Recruiting first, the newest first:

9 more — sign in →

All 14 open studies →

A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →

Who these studies take

Of the 30 Polycystic Kidney, Autosomal Dominant studies recruiting now, 8 (27%) set no upper age limit and 19 (63%) stop at 75 or younger; 7 take children under 18; 9 take healthy volunteers.

This study: 18 Years to 60 Years · all sexes · no healthy volunteers.

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Watch this landscapeDownload CSVNarrow it in search →With an account: a weekly mail of what changed among 85 studies in Polycystic Kidney, Autosomal Dominant with a site in United States, or all of them as a file.
02

Study summary

This study will test to see if metformin is safe and if it is tolerated compared to placebo in adult Autosomal Dominant Polycystic Kidney Disease (ADPKD) patients with beginning stages of chronic kidney disease. We will also measure its effect on progression of kidney disease as reflected in the kidney size and the kidney function, along with its effect on kidney pain and quality of life.

Read the detailed description

There is growing evidence that metformin, a drug widely used for the treatment of type 2 diabetes and polycystic ovary syndrome, may serve as a novel therapy for individuals in the early stages of Autosomal Dominant Polycystic Kidney Disease ADPKD by activating the metabolic sensor AMP-activated protein kinase (AMPK). AMPK is activated under conditions of metabolic and other cellular stresses. Through its actions on downstream mediators, AMPK activation during low energy states decreases cellular energy consumption while stimulating energy generating pathways. It has been shown that AMPK phosphorylates and inhibits cystic fibrosis transmembrane conductance regulator (CFTR), thus suppressing epithelial fluid and electrolyte secretion. Similarly, AMPK phosphorylates the tuberin protein, leading to indirect inhibition of the mTOR pathway. Thus, AMPK inhibits both CFTR and mTOR, suggesting that targeted activation of this kinase by metformin may provide a therapeutic benefit in ADPKD. It has been shown that metformin treatment of kidney epithelial cells leads to stimulation of AMPK and subsequent inhibition of both mTOR and CFTR activity. It has also been shown that metformin slows cystogenesis in animal models of PKD, supporting the potential of this drug in ADPKD treatment.

03

Conditions studied

  • Polycystic Kidney, Autosomal Dominant

Keywords

  • kidney disease
  • polycystic kidney disease
  • autosomal dominant kidney disease
  • kidney cysts
  • PKD
04

Who can participate

Ages eligible
18 Years to 60 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

Subject has Autosomal Dominant Polycystic Kidney Disease; Subject is fluent in English

Exclusion criteria

Exclusion Criteria:

Subject is not on active military duty; Subject is not currently participating in another clinical trial; Subject's current is not \<50 cc/min/1.73m2; Subject does not have diabetes; Subject does not have a systemic disease other than hypertension and PKD; Subject does not have a solitary kidney; Subject does not have an allergy or intolerance to metformin; Subject is not pregnant or lactating or intending to become pregnant within the next three years; Subject does not have an unstable or unclipped cerebral aneurysm; Subject does not have active coronary artery disease; Subject does not have an MRI incompatible device/implant; Subject does not have severe claustrophobia; Subject has not had any solid organ transplant; Subject does not have a Vitamin B12 deficiency; Subject does not currently take any medications that interact with metformin, such as nifedipine, furosemide, cationic drugs (amiloride, ranitidine, triamterene digoxin, procainamide, quinidine, vancomycin, trimethoprim); Subject does not currently take nor has taken (within 2 weeks) the drug tolvaptan (Jynarque or Samsca)

Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
97 participants (actual)

Study arms

  • Experimental
    Metformin

    Participants will be started on 500 mg of metformin once daily, with the following scheduled dose titrations: * Increase to 500mg twice daily at week 2 * Increase to 1000mg qAM, 500mg qPM at week 4 * Increase to 1000mg twice daily at week 6 for the duration of their participation (26 months). * Increased titrations based on tolerability

    Drug: Metformin

  • Placebo comparator
    Placebo

    Participants will be started on 500 mg of placebo once daily, with the following scheduled dose titrations: * Increase to 500mg twice daily at week 2 * Increase to 1000mg qAM, 500mg qPM at week 4 * Increase to 1000mg twice daily at week 6 for the duration of their participation (26 months). * Increased titrations based on tolerability

    Other: Placebo

Interventions

  • Monitoring of tolerability and symptoms.

    Also known as: Glucophage, Metformin hydrochloride

  • OtherPlacebo

    Monitoring of tolerability and symptoms.

06

What researchers measure

Primary outcomes

  1. Change in the Gastrointestinal Symptoms Rating Scale (GSRS) to 24 Months

    GSRS is a widely used, validated 15-item questionnaire used to assess GI symptom burden (minimum, maximum: 1, 7, where higher mean score is worse outcome). Mean change to 24 months, estimated with a repeated measures analysis (baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months) using a linear mixed model.

    Time frame: Baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months

  2. Drug Tolerability

    Tolerability was based on the first visit a participant responded no to the following question "Can you tolerate this dose of study drug the rest of your life?", which was asked at baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months.

    Time frame: Baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months

  3. Rate of Serious Adverse Events (SAE)

    Serious adverse events (SAE) occurring from the time a participant signs the informed consent (at the screening visit) until the end of the study, meeting 1 or more of the criteria of: 1) Resulting in death, 2) Non-elective hospitalization, 3) Life threatening (if patient continued on study drug would result in death), 4) Harming or disabling persistently or permanently , 5) Exceeding the nature, severity or frequency of risk described in the protocol or 6) Resulting in congenital anomaly.

    Time frame: 26 months

Secondary outcomes

  1. Quality of Life Physical Component

    Short Form-36 Quality of Life Physical Component Summary (SF-36 PCS) ranges from 0 (worst possible outcome) to 100 (best possible outcome). Mean change to 24 months, estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a linear mixed model.

    Time frame: Baseline, 1 month, 3 months and every 3 months thereafter to 24 months

  2. Quality of Life Mental Component

    Short Form-36 Quality of Life Mental Component Summary (SF-36 MCS) ranges from 0 (worst possible outcome) to 100 (best possible outcome). Mean change to 24 months, estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a linear mixed model.

    Time frame: Baseline, 1 month, 3 months and every 3 months thereafter to 24 months

  3. Back Pain Frequency Over the Past 3 Months Since Last Visit

    Odds ratio (OR) per month of back pain Often, Usually, or Always (vs. Never, Rarely, Sometimes) estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a generalized linear mixed model.

    Time frame: Baseline, 1 month, 3 months and every 3 months thereafter to 24 months

  4. Estimated Glomerular Filtration Rate (eGFR)

    Mean change to 24 months, estimated with a repeated measures analysis (baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months) using a linear mixed model.

    Time frame: Baseline, 2 weeks, and 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months

  5. Total Kidney Volume From Magnetic Resonance Imaging

    Annual percent change of height adjusted and natural log transformed total kidney volume \[ln(htTKV)\] was estimated with a linear mixed model.

    Time frame: Baseline, 6 months, 12 months, 18 months, 24 months

  6. Total Kidney Cyst Volume From Magnetic Resonance Imaging

    Annual percent change of height adjusted and natural log transformed total kidney cyst volume \[ln(htTKCV)\] was estimated with a linear mixed model.

    Time frame: Baseline, 6 months, 12 months, 18 months, 24 months

  7. Liver Volume From Magnetic Resonance Imaging

    Annual percent change of height adjusted and natural log transformed liver volume \[ln(htLV)\] was estimated with a linear mixed model.

    Time frame: Baseline, 6 months, 12 months, 18 months, 24 months

  8. Liver Cyst Volume From Magnetic Resonance Imaging

    Annual percent change of height adjusted and natural log transformed liver cyst volume \[ln(htLCV)\] was estimated with a linear mixed model.

    Time frame: Baseline, 6 months, 12 months, 18 months, 24 months

  9. Frequency Abdominal Fullness Interfered With Ability to Perform Usual Physical Activity Over the Past 3 Months Since Last Visit.

    Odds ratio (OR) per month of abdominal fullness interfered Often, Usually, or Always (vs. Never, Rarely, Sometimes) estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a generalized linear mixed model.

    Time frame: Baseline, 1 month, 3 months and every 3 months thereafter to 24 months

  10. Interference of Pain With Sleep Over the Past 3 Months Since Last Visit

    Odds ratio (OR) per month of pain interfered with sleep Quite a bit or Extremely (vs. Not at all, A little bit, Moderately) estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a generalized linear mixed model.

    Time frame: Baseline, 1 month, 3 months and every 3 months thereafter to 24 months

  11. Interference of Pain With Strenuous Physical Activity Over the Past 3 Months Since Last Visit

    Odds ratio (OR) per month of pain interfered with strenuous physical activity Quite a bit or Extremely (vs. Not at all, A little bit, Moderately) estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a generalized linear mixed model.

    Time frame: Baseline, 1 month, 3 months and every 3 months thereafter to 24 months

07

Results

Posted Aug 9, 2022

Participant flow

Participant flow — Overall Study
MilestoneMetforminPlacebo
Started4948
Completed4042
Not completed96
Withdrew: Study burden33
Withdrew: Adverse event21
Withdrew: Unable/unwilling to take study medication21
Withdrew: Began tolvaptan11
Withdrew: Withdrawal by subject10

Outcome measures

PrimaryChange in the Gastrointestinal Symptoms Rating Scale (GSRS) to 24 Months

GSRS is a widely used, validated 15-item questionnaire used to assess GI symptom burden (minimum, maximum: 1, 7, where higher mean score is worse outcome). Mean change to 24 months, estimated with a repeated measures analysis (baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months) using a linear mixed model.

Time frame:
Baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months
Reported as:
Mean · score on a scale
Change in the Gastrointestinal Symptoms Rating Scale (GSRS) to 24 Months
score on a scaleMetforminPlacebo
Change in the Gastrointestinal Symptoms Rating Scale (GSRS) to 24 Months-0.04 (-0.18 to 0.10)-0.11 (-0.24 to 0.03)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.50 · Mean difference (net): 0.07 · 95% CI -0.13 to 0.26
PrimaryDrug Tolerability

Tolerability was based on the first visit a participant responded no to the following question "Can you tolerate this dose of study drug the rest of your life?", which was asked at baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months.

Time frame:
Baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months
Reported as:
Count of participants · Participants
Drug Tolerability
ParticipantsMetforminPlacebo
Drug Tolerability2114
Statistical analysis
  • Metformin vs Placebo · Gray's test · p = 0.12
PrimaryRate of Serious Adverse Events (SAE)

Serious adverse events (SAE) occurring from the time a participant signs the informed consent (at the screening visit) until the end of the study, meeting 1 or more of the criteria of: 1) Resulting in death, 2) Non-elective hospitalization, 3) Life threatening (if patient continued on study drug would result in death), 4) Harming or disabling persistently or permanently , 5) Exceeding the nature, severity or frequency of risk described in the protocol or 6) Resulting in congenital anomaly.

Time frame:
26 months
Reported as:
Count of participants · Participants
Rate of Serious Adverse Events (SAE)
ParticipantsMetforminPlacebo
Rate of Serious Adverse Events (SAE)44
Statistical analysis
  • Metformin vs Placebo · Regression, Logistic · p = 0.98
SecondaryQuality of Life Physical Component

Short Form-36 Quality of Life Physical Component Summary (SF-36 PCS) ranges from 0 (worst possible outcome) to 100 (best possible outcome). Mean change to 24 months, estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a linear mixed model.

Time frame:
Baseline, 1 month, 3 months and every 3 months thereafter to 24 months
Reported as:
Mean · score on a scale
Quality of Life Physical Component
score on a scaleMetforminPlacebo
Quality of Life Physical Component-0.11 (-1.69 to 1.46)-0.51 (-2.05 to 1.03)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.72 · Mean difference (final values): 0.40 · 95% CI -1.81 to 2.60
SecondaryQuality of Life Mental Component

Short Form-36 Quality of Life Mental Component Summary (SF-36 MCS) ranges from 0 (worst possible outcome) to 100 (best possible outcome). Mean change to 24 months, estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a linear mixed model.

Time frame:
Baseline, 1 month, 3 months and every 3 months thereafter to 24 months
Reported as:
Mean · score on a scale
Quality of Life Mental Component
score on a scaleMetforminPlacebo
Quality of Life Mental Component1.01 (-1.23 to 3.25)-0.11 (-2.29 to 2.08)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.49 · Mean difference (final values): 1.11 · 95% CI -2.02 to 4.25
SecondaryBack Pain Frequency Over the Past 3 Months Since Last Visit

Odds ratio (OR) per month of back pain Often, Usually, or Always (vs. Never, Rarely, Sometimes) estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a generalized linear mixed model.

Time frame:
Baseline, 1 month, 3 months and every 3 months thereafter to 24 months
Reported as:
Number · odds ratio
Back Pain Frequency Over the Past 3 Months Since Last Visit
odds ratioMetforminPlacebo
Back Pain Frequency Over the Past 3 Months Since Last Visit0.95 (0.90 to 1.00)0.92 (0.88 to 0.98)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.44
SecondaryEstimated Glomerular Filtration Rate (eGFR)

Mean change to 24 months, estimated with a repeated measures analysis (baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months) using a linear mixed model.

Time frame:
Baseline, 2 weeks, and 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months
Reported as:
Mean · ml/min/1.73m^2
Estimated Glomerular Filtration Rate (eGFR)
ml/min/1.73m^2MetforminPlacebo
Estimated Glomerular Filtration Rate (eGFR)-3.41 (-6.36 to -0.46)-6.14 (-9.04 to -3.24)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.20 · Mean difference (final values): 2.73 · 95% CI -1.40 to 6.87
SecondaryTotal Kidney Volume From Magnetic Resonance Imaging

Annual percent change of height adjusted and natural log transformed total kidney volume \[ln(htTKV)\] was estimated with a linear mixed model.

Time frame:
Baseline, 6 months, 12 months, 18 months, 24 months
Reported as:
Mean · annual percent change
Total Kidney Volume From Magnetic Resonance Imaging
annual percent changeMetforminPlacebo
Total Kidney Volume From Magnetic Resonance Imaging3.87 (1.09 to 6.74)2.16 (-0.52 to 4.91)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.38 · Mean difference (final values): 1.68 · 95% CI -2.11 to 5.62
SecondaryTotal Kidney Cyst Volume From Magnetic Resonance Imaging

Annual percent change of height adjusted and natural log transformed total kidney cyst volume \[ln(htTKCV)\] was estimated with a linear mixed model.

Time frame:
Baseline, 6 months, 12 months, 18 months, 24 months
Reported as:
Mean · annual percent change
Total Kidney Cyst Volume From Magnetic Resonance Imaging
annual percent changeMetforminPlacebo
Total Kidney Cyst Volume From Magnetic Resonance Imaging9.37 (3.82 to 15.22)5.36 (0.13 to 10.86)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.31 · Mean difference (final values): 3.81 · 95% CI -3.48 to 11.65
SecondaryLiver Volume From Magnetic Resonance Imaging

Annual percent change of height adjusted and natural log transformed liver volume \[ln(htLV)\] was estimated with a linear mixed model.

Time frame:
Baseline, 6 months, 12 months, 18 months, 24 months
Reported as:
Mean · annual percent change
Liver Volume From Magnetic Resonance Imaging
annual percent changeMetforminPlacebo
Liver Volume From Magnetic Resonance Imaging1.60 (0.02 to 3.21)1.21 (-0.32 to 2.77)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.72 · Mean difference (final values): 0.39 · 95% CI -1.78 to 2.61
SecondaryLiver Cyst Volume From Magnetic Resonance Imaging

Annual percent change of height adjusted and natural log transformed liver cyst volume \[ln(htLCV)\] was estimated with a linear mixed model.

Time frame:
Baseline, 6 months, 12 months, 18 months, 24 months
Reported as:
Mean · annual percent change
Liver Cyst Volume From Magnetic Resonance Imaging
annual percent changeMetforminPlacebo
Liver Cyst Volume From Magnetic Resonance Imaging12.72 (3.47 to 22.79)10.94 (1.73 to 20.99)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.80 · Mean difference (final values): 1.60 · 95% CI -10.05 to 14.76
SecondaryFrequency Abdominal Fullness Interfered With Ability to Perform Usual Physical Activity Over the Past 3 Months Since Last Visit.

Odds ratio (OR) per month of abdominal fullness interfered Often, Usually, or Always (vs. Never, Rarely, Sometimes) estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a generalized linear mixed model.

Time frame:
Baseline, 1 month, 3 months and every 3 months thereafter to 24 months
Reported as:
Number · odds ratio
Frequency Abdominal Fullness Interfered With Ability to Perform Usual Physical Activity Over the Past 3 Months Since Last Visit.
odds ratioMetforminPlacebo
Frequency Abdominal Fullness Interfered With Ability to Perform Usual Physical Activity Over the Past 3 Months Since Last Visit.1.00 (0.89 to 1.13)0.99 (0.87 to 1.12)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.83
SecondaryInterference of Pain With Sleep Over the Past 3 Months Since Last Visit

Odds ratio (OR) per month of pain interfered with sleep Quite a bit or Extremely (vs. Not at all, A little bit, Moderately) estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a generalized linear mixed model.

Time frame:
Baseline, 1 month, 3 months and every 3 months thereafter to 24 months
Reported as:
Number · odds ratio
Interference of Pain With Sleep Over the Past 3 Months Since Last Visit
odds ratioMetforminPlacebo
Interference of Pain With Sleep Over the Past 3 Months Since Last Visit1.00 (0.87 to 1.14)0.97 (0.88 to 1.07)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.72
SecondaryInterference of Pain With Strenuous Physical Activity Over the Past 3 Months Since Last Visit

Odds ratio (OR) per month of pain interfered with strenuous physical activity Quite a bit or Extremely (vs. Not at all, A little bit, Moderately) estimated with a repeated measures analysis (baseline, 1 month, 3 months and every 3 months thereafter to 24 months) using a generalized linear mixed model.

Time frame:
Baseline, 1 month, 3 months and every 3 months thereafter to 24 months
Reported as:
Number · odds ratio
Interference of Pain With Strenuous Physical Activity Over the Past 3 Months Since Last Visit
odds ratioMetforminPlacebo
Interference of Pain With Strenuous Physical Activity Over the Past 3 Months Since Last Visit0.99 (0.93 to 1.06)0.95 (0.90 to 1.01)
Statistical analysis
  • Metformin vs Placebo · Mixed Models Analysis · p = 0.28

Adverse events

Collected over Adverse event were data collected at 2, 4, and 6 weeks during the titration period and every 3 months thereafter through 26 months.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Metformin0/49 (0%)4/49 (8.2%)21/49 (42.9%)
Placebo0/48 (0%)4/48 (8.3%)13/48 (27.1%)
Most frequent serious events
Showing 10 of 12
Most frequent serious events
EventMetforminPlacebo
Other - lightheadednessNervous system disorders0/491/48
Appendicitis perforatedInfections and infestations0/491/48
Other - urinary tract infection and renal cyst hemorrhage (mild)Infections and infestations0/491/48
Other - renal cystRenal and urinary disorders0/491/48
Other - renal cyst ruptureRenal and urinary disorders0/491/48
Other - diarrhea (c. difficile infection)Infections and infestations1/490/48
Other - bone fractureMusculoskeletal and connective tissue disorders1/490/48
SeizureNervous system disorders1/490/48
Gastric hemorrhageGastrointestinal disorders1/490/48
Suicide attemptPsychiatric disorders1/490/48
Most frequent other events
Showing 10 of 27
Most frequent other events
EventMetforminPlacebo
DiarrheaGastrointestinal disorders21/4913/48
NauseaGastrointestinal disorders17/4910/48
FlatulenceGastrointestinal disorders10/497/48
Abdominal PainGastrointestinal disorders8/499/48
CoughRespiratory, thoracic and mediastinal disorders5/499/48
HeadacheNervous system disorders8/496/48
ConstipationGastrointestinal disorders6/497/48
Sore ThroatRespiratory, thoracic and mediastinal disorders5/497/48
Abdominal DistensionGastrointestinal disorders7/494/48
VomitingGastrointestinal disorders5/496/48

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)MetforminPlaceboTotal
<=18 years000
Between 18 and 65 years494897
>=65 years000
Age, Continuous
Age, Continuous(years)MetforminPlaceboTotal
Mean41.8 ± 10.442.1 ± 10.141.9 ± 10.2
Sex: Female, Male
Sex: Female, Male(Participants)MetforminPlaceboTotal
Female383270
Male111627
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)MetforminPlaceboTotal
Hispanic or Latino268
Not Hispanic or Latino474289
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)MetforminPlaceboTotal
American Indian or Alaska Native101
Asian213
Native Hawaiian or Other Pacific Islander000
Black or African American011
White464692
More than one race000
Unknown or Not Reported000
Region of Enrollment
Region of Enrollment(participants)MetforminPlaceboTotal
United States494897
PKD genotype
PKD genotype(Participants)MetforminPlaceboTotal
PKD1372865
PKD271017
Other145
No mutation detected246
N/A224
GFR
GFR((ml/min/1.73 m²))MetforminPlaceboTotal
Mean86.1 ± 20.685.9 ± 19.086.0 ± 19.7

14 further baseline measures are reported on the registry.

08

Study locations

2 sites
  • University of Maryland Medical Center
    Baltimore, Maryland 21201, United States
  • Tufts Medical Center
    Boston, Massachusetts 02111, United States
09

References and documents

Publications

  • Seliger SL, Watnick T, Althouse AD, Perrone RD, Abebe KZ, Hallows KR, Miskulin DC, Bae KT. Baseline Characteristics and Patient-Reported Outcomes of ADPKD Patients in the Multicenter TAME-PKD Clinical Trial. Kidney360. 2020 Dec 31;1(12):1363-1372. doi: 10.34067/KID.0004002020. PubMed 33768205 ↗

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Jun 2016 to primary completion in Dec 2020, 53 months.

Results were first posted in Aug 2022, 1 year 8 months after primary completion.

Of 17 phase 2 interventional studies under Polycystic Kidney, Autosomal Dominant, half reached primary completion within 39 months of starting (the median), counting those still running; a quarter within 18 and three quarters within 53. This study's 53 months is above that.

How these figures are worked out, and which studies are left out of them.

Registered
Dec 23, 2015
First posted
Jan 14, 2016
StartActual
Jun 27, 2016
Primary completionActual
Dec 7, 2020
CompletionActual
Dec 7, 2020
Results first posted
Aug 9, 2022
Last update
Aug 9, 2022
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Aug 9, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02656017
Lead sponsor
Kyongtae Ty Bae, M.D., Ph.D.
Responsible party
Kyongtae Ty Bae, M.D., Ph.D. (Principal Investigator, University of Pittsburgh) — Sponsor-investigator
First posted
Jan 14, 2016
Start date
Jun 27, 2016
Primary completion
Dec 7, 2020
Completion
Dec 7, 2020
Results posted
Aug 9, 2022
Last update
Aug 9, 2022

Study contacts

Kyongtae Bae, MD, PhD
principal investigator · University of Pittsburgh

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

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Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drug tested here: Metformin

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