Trial · NCT02656017

Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease

NCT02656017, Phase 2, Completed, Kyongtae Ty Bae, M.D., Ph.D.; results first posted 2022-08-09

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease. Trials, record NCT02656017. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02656017

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease
NCT number: NCT02656017
Status: Completed
Phases: Phase 2
Enrollment: 97 participants, actual
Lead sponsor: Kyongtae Ty Bae, M.D., Ph.D.
Conditions: Polycystic Kidney, Autosomal Dominant
Interventions: Metformin, Placebo
First posted: 2016-01-14
Last updated: 2022-08-09
Results first posted: 2022-08-09

Source ClinicalTrials.gov

Registered outcomes14

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change in the Gastrointestinal Symptoms Rating Scale (GSRS) to 24 Months	Baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter 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.
Primary	Drug Tolerability	Baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months	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.
Primary	Rate of Serious Adverse Events (SAE)	26 months	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.
Secondary	Quality of Life Physical Component	Baseline, 1 month, 3 months and every 3 months thereafter to 24 months	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.
Secondary	Quality of Life Mental Component	Baseline, 1 month, 3 months and every 3 months thereafter to 24 months	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.
Secondary	Back Pain Frequency Over the Past 3 Months Since Last Visit	Baseline, 1 month, 3 months and every 3 months thereafter to 24 months	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.
Secondary	Estimated Glomerular Filtration Rate (eGFR)	Baseline, 2 weeks, and 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months	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.
Secondary	Total Kidney Volume From Magnetic Resonance Imaging	Baseline, 6 months, 12 months, 18 months, 24 months	Annual percent change of height adjusted and natural log transformed total kidney volume \[ln(htTKV)\] was estimated with a linear mixed model.
Secondary	Total Kidney Cyst Volume From Magnetic Resonance Imaging	Baseline, 6 months, 12 months, 18 months, 24 months	Annual percent change of height adjusted and natural log transformed total kidney cyst volume \[ln(htTKCV)\] was estimated with a linear mixed model.
Secondary	Liver Volume From Magnetic Resonance Imaging	Baseline, 6 months, 12 months, 18 months, 24 months	Annual percent change of height adjusted and natural log transformed liver volume \[ln(htLV)\] was estimated with a linear mixed model.

1 to 10 of 14Source ClinicalTrials.gov

See all 14 registered outcomes

Outcomes14

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in the Gastrointestinal Symptoms Rating Scale (GSRS) to 24 Months	Baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months		score on a scale	MEAN
PRIMARY	Drug Tolerability	Baseline, 2 weeks, 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months	Intention to treat analysis	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Rate of Serious Adverse Events (SAE)	26 months	Participants who received and took metformin or placebo	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Quality of Life Physical Component	Baseline, 1 month, 3 months and every 3 months thereafter to 24 months	Intention to treat analysis	score on a scale	MEAN
SECONDARY	Quality of Life Mental Component	Baseline, 1 month, 3 months and every 3 months thereafter to 24 months	Intention to treat analysis	score on a scale	MEAN
SECONDARY	Back Pain Frequency Over the Past 3 Months Since Last Visit	Baseline, 1 month, 3 months and every 3 months thereafter to 24 months	Intention to treat analysis	odds ratio	NUMBER
SECONDARY	Estimated Glomerular Filtration Rate (eGFR)	Baseline, 2 weeks, and 6 weeks, 1 month, 3 months and every 3 months thereafter to 24 months	Intention to treat analysis	ml/min/1.73m^2	MEAN
SECONDARY	Total Kidney Volume From Magnetic Resonance Imaging	Baseline, 6 months, 12 months, 18 months, 24 months	Intention to treat analysis	annual percent change	MEAN
SECONDARY	Total Kidney Cyst Volume From Magnetic Resonance Imaging	Baseline, 6 months, 12 months, 18 months, 24 months	Intention to treat analysis	annual percent change	MEAN
SECONDARY	Liver Volume From Magnetic Resonance Imaging	Baseline, 6 months, 12 months, 18 months, 24 months	Intention to treat analysis	annual percent change	MEAN

1 to 10 of 14Source ClinicalTrials.gov

See all 14 outcomes

Results by group28

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Change in the Gastrointestinal Symptoms Rating Scale (GSRS) to 24 Months	Metformin	-0.04	95% Confidence Interval -0.18 to 0.10	score on a scale
Change in the Gastrointestinal Symptoms Rating Scale (GSRS) to 24 Months	Placebo	-0.11	95% Confidence Interval -0.24 to 0.03	score on a scale
Drug Tolerability	Metformin	21		Participants
Drug Tolerability	Placebo	14		Participants
Rate of Serious Adverse Events (SAE)	Metformin	4		Participants
Rate of Serious Adverse Events (SAE)	Placebo	4		Participants
Quality of Life Physical Component	Metformin	-0.11	95% Confidence Interval -1.69 to 1.46	score on a scale
Quality of Life Physical Component	Placebo	-0.51	95% Confidence Interval -2.05 to 1.03	score on a scale
Quality of Life Mental Component	Metformin	1.01	95% Confidence Interval -1.23 to 3.25	score on a scale
Quality of Life Mental Component	Placebo	-0.11	95% Confidence Interval -2.29 to 2.08	score on a scale

1 to 10 of 28Source ClinicalTrials.gov

See all 28 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Metformin	49
Overall Study	STARTED	Placebo	48
Overall Study	COMPLETED	Metformin	40
Overall Study	COMPLETED	Placebo	42
Overall Study	NOT COMPLETED	Metformin	9
Overall Study	NOT COMPLETED	Placebo	6

Source ClinicalTrials.gov

Adverse events78

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Diarrhea	Gastrointestinal disorders	Metformin	21	49	42
other	Diarrhea	Gastrointestinal disorders	Placebo	13	48	18
other	Nausea	Gastrointestinal disorders	Metformin	17	49	29
other	Nausea	Gastrointestinal disorders	Placebo	10	48	15
other	Abdominal Pain	Gastrointestinal disorders	Metformin	8	49	11
other	Abdominal Pain	Gastrointestinal disorders	Placebo	9	48	9
other	Flatulence	Gastrointestinal disorders	Metformin	10	49	12
other	Flatulence	Gastrointestinal disorders	Placebo	7	48	7
other	Constipation	Gastrointestinal disorders	Metformin	6	49	6
other	Constipation	Gastrointestinal disorders	Placebo	7	48	7

1 to 10 of 78Source ClinicalTrials.gov

See all 78 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Metformin	0	49
Total, all-cause mortality	Placebo	0	48
Total, other adverse events	Metformin	21	49
Total, other adverse events	Placebo	13	48
Total, serious adverse events	Metformin	4	49
Total, serious adverse events	Placebo	4	48

Source ClinicalTrials.gov

Papers3

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
33768205	DERIVED	PubMed
38837240	DERIVED	PubMed
39356039	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons4

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Metformin vs Placebo: Drug Tolerability	Trial comparisons
Metformin vs Placebo: deaths from any cause	Trial comparisons
Metformin vs Placebo: Rate of Serious Adverse Events (SAE)	Trial comparisons
Metformin vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT02656017

GET https://conductscience.org/api/trials/v1/NCT02656017/results?page=2

API reference For AI assistants