Phase 2 N=97 Randomized Quadruple-blind Treatment

Metformin as a Novel Therapy for Autosomal Dominant Polycystic Kidney Disease

Polycystic Kidney, Autosomal Dominant

Bottom Line

View on ClinicalTrials.gov: NCT02656017 ↗

Enrolled (actual)

Serious AEs

8.3%

Results posted

Aug 2022

Primary outcome: Primary: Change in the Gastrointestinal Symptoms Rating Scale (GSRS) to 24 Months — -0.04; -0.11 score on a scale — p=0.50

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Metformin (Drug); Placebo (Other)
Age: Adult · 18+ yrs
Sex: All
Sponsor: Kyongtae Ty Bae, M.D., Ph.D.
Primary completion: Dec 2020

Outcome Measures

Outcome	Result	p-value
PRIMARY Change in the Gastrointestinal Symptoms Rating Scale (GSRS) to 24 Months	-0.04; -0.11	0.50
PRIMARY Drug Tolerability	21; 14	0.12
PRIMARY Rate of Serious Adverse Events (SAE)	4; 4	0.98
SECONDARY Quality of Life Physical Component	-0.11; -0.51	0.72
SECONDARY Quality of Life Mental Component	1.01; -0.11	0.49
SECONDARY Back Pain Frequency Over the Past 3 Months Since Last Visit	0.95; 0.92	0.44
SECONDARY Estimated Glomerular Filtration Rate (eGFR)	-3.41; -6.14	0.20
SECONDARY Total Kidney Volume From Magnetic Resonance Imaging	3.87; 2.16	0.38
SECONDARY Total Kidney Cyst Volume From Magnetic Resonance Imaging	9.37; 5.36	0.31
SECONDARY Liver Volume From Magnetic Resonance Imaging	1.60; 1.21	0.72
SECONDARY Liver Cyst Volume From Magnetic Resonance Imaging	12.72; 10.94	0.80
SECONDARY Frequency Abdominal Fullness Interfered With Ability to Perform Usual Physical Activity Over the Past 3 Months Since Last Visit.	1.00; 0.99	0.83
SECONDARY Interference of Pain With Sleep Over the Past 3 Months Since Last Visit	1.00; 0.97	0.72
SECONDARY Interference of Pain With Strenuous Physical Activity Over the Past 3 Months Since Last Visit	0.99; 0.95	0.28

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.

Eligibility Criteria

Inclusion Criteria

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

Exclusion Criteria

Subject is not on active military duty; Subject is not currently participating in another clinical trial; Subject's current GFR 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)

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02656017). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.