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. 2019 Apr 17;11:3295–3313. doi: 10.2147/CMAR.S200059

Table 2.

A summary of clinical trials studies of metformin effects on cancers

Study NCT Number Status Participants Period Intervention Cancer Type Outcome
A Phase III randomized trial of metformin vs placebo in early stage breast cancer NCT01101438
(L https://clinicaltrials.gov/ct2/show/NCT01101438?term=NCT01101438&rank=1 ink)		 Active

  • 3,582 (original estimated enrollment)

  • 18 years to 74 years (adult, older adult)

  • All sex

 April 9, 2010 to February 2022 (Estimated Primary Completion Date)

  • Drug: metformin


(twice a day for 5 years after diagnosis, given orally)

  • Other: placebo (given orally)

 Breast cancer Metformin improved patients’ PFS
A randomized Phase II study of chemotherapy ± metformin in metastatic pancreatic cancer NCT01167738
(Link)
 https://clinicaltrials.gov/ct2/show/NCT01167738?term=NCT01167738&rank=1 		Terminated

  • 82 (original estimated enrollment)

  • 18 years to 75 years (adult, older adult)

  • All sex

 July 21, 2010 to December 2014

  • Drug: capecitabine, cisplatinum, epirubicine, gemcitabine and metformin

 Pancreatic cancer Metformin improved patients’ PFS with combination of chemotherapy compared to combined chemotherapy alone
A Phase II, randomized, placebo controlled study to evaluate the efficacy of the combination of gemcitabine, erlotinib and metformin in patients with locally advanced and metastatic pancreatic cancer NCT01210911
(Link)
 https://clinicaltrials.gov/ct2/show/NCT01210911?term=NCT01210911&rank=1 		Completed

  • 120

  • 18 years to 80 years (adult, older adult)

  • All sex

 September 28, 2010 to February 2014

  • Drug: gemcitabine, erlotinib, metformin and placebo

 Pancreatic cancer Metformin improved patients’ PFS
Prospective study of metformin in castration-resistant prostate cancer NCT01215032
(Link)
 https://clinicaltrials.gov/ct2/show/NCT01215032?term=NCT01215032&rank=1 		Terminated

  • 106 (original estimated enrollment)

  • Child, adult, older adult

  • Male

 October 4, 2010 to January 2015

  • Drug: metformin


(taken orally twice daily each 28-day cycle, for 12 cycles)		 Prostate cancer Metformin improved patients’ PSA
A Phase II single arm study to examine the effects of metformin on cancer metabolism in patients with early stage breast cancer receiving neoadjuvant chemotherapy NCT01266486
(Link)
 https://clinicaltrials.gov/ct2/show/NCT01266486?term=NCT01266486&rank=1 		Completed

  • 41

  • 18 years and older (adult, older adult)

  • All sex

 December 23, 2010 to May 2014

  • Drug: metformin


(extended release metformin 1,500 mg once daily for 14–21 days)		 Breast cancer Metformin activates AMPK and reduces the levels of 4E-BPs and S6Ks
A randomized Phase II, double blind trial of standard chemotherapy with metformin (vs placebo) in women with metastatic breast cancer receiving first, second, third or fourth line chemotherapy with anthracycline, taxane, platinum, capecitabine or vinorelbine based regimens NCT01310231
(Link)
 https://clinicaltrials.gov/ct2/show/NCT01310231?term=NCT01310231&rank=1 		Completed

  • 40

  • 18 years to 75 years (adult, older adult)

  • Female

 February 18, 2011 to July 14, 2016

  • Drug: metformin (metformin 850 mg bid in addition to standard chemotherapy)

  • Other: placebo (placebo bid in addition to standard chemotherapy)

 Metastatic breast cancer Metformin improved patients’ overall response rate (ORR)
Phase II study evaluating the efficacy of the combination of metformin to neoadjuvant radiochemotherapy in the treatment of locally advanced rectal cancer NCT02437656
(Link)
 https://clinicaltrials.gov/ct2/show/NCT02437656?term=NCT02437656&rank=1 		Completed

  • 60

  • 18 years and older (adult, older adult)

  • All Sex

 May 5, 2015 to March 2017

  • Drug: metformin (850 mg 2 times/day and 850 mg 3 times/day)

 Rectal cancer Metformin improved patients’ sphincter preservation rate (SPR)
Clinical and biologic effects of metformin in early stage breast cancer NCT00897884
(Link)
 https://clinicaltrials.gov/ct2/show/NCT00897884?term=NCT00897884&rank=1 		Completed

  • 40

  • 18 years to 70 years (adult, older adult)

  • Female

 May 8, 2009 to July 2011

  • Drug: metformin (500 mg tablet, taken 3 times a day for 2–3 weeks)

 Breast cancer Metformin can reduces cell proliferation rates in tumor tissue
A Phase I trial of lapatinib in combination with 1) sirolimus or 2) metformin in advanced cancer NCT01087983
(Link)
 https://clinicaltrials.gov/ct2/show/NCT01087983?term=NCT01087983&rank=1 		Completed

  • 118

  • Child, adult, older adult

  • All sex

 March 15, 2010 to September 2015

  • Drug: lapatinib (starting oral dose of 500 mg daily for 21)

  • Drug: sirolimus (starting oral dose 1 mg daily)

  • Drug: metformin (starting oral dose 1,000 mg daily)

 Advanced cancers Lapatinib when used in
combination with metformin will have beneficial effects in advanced cancer
Pre-surgical trial of metformin in patients with operable breast cancer NCT00984490
(Link)
 https://clinicaltrials.gov/ct2/show/NCT00984490?term=NCT00984490&rank=1 		Terminated

  • 30 (original estimated enrollment)

  • 18 years and older (adult, older adult)

  • Not provided

 September 24, 2009 to July 2010

  • Drug: metformin (metformin: 850 mg orally twice daily for 7–21 days, discontinued 24–36 h prior to surgery)

  • Other: laboratory biomarker analysis (blood and tissue collection to determine the in situ biologic effect of metformin)

 Breast cancer Metformin changes proliferation
(Ki67) and apoptosis before and after treatment in women with operable
stage I or II breast cancer
APhase II evaluation of metformin, targeting cancer stem cells for the prevention of relapse in patients with stage IIC/III/IV ovarian, fallopian tube, and primary peritoneal cancer NCT01579812
(Link)
 https://clinicaltrials.gov/ct2/show/NCT01579812?term=NCT01579812&rank=1 		Completed

  • 90

  • 19 years to 79 years (adult, older adult)

  • Female

 March 30, 2012 to June 2017

  • Drug: metformin

 Ovarian, fallopian tube, and primary peritoneal cancer Metformin improved patients’ RFS
APhase II study of metformin plus modified FOLFOX 6 in patients with metastatic pancreatic cancer NCT01666730
(Link)
 https://clinicaltrials.gov/ct2/show/NCT01666730?term=NCT01666730&rank=1 		Active, not recruiting

  • 50

  • 18 years and older (adult, older adult)

  • All sex

 August 14, 2012 to March 2019 (estimated study completion date)

  • Drug: metformin (given orally) oxaliplatin, leucovorin calcium, and fluorouracil (given IV)

  • Other: laboratory biomarker analysis correlative studies

 1. Acinar cell adenocarcinoma of the pancreas
2. Duct cell adenocarcinoma of the pancreas
3. Recurrent pancreatic cancer
4. Stage IV pancreatic cancer		 Metformin improved patients’ PFS and OS
An exploratory study of metformin with or without rapamycin as maintenance therapy after induction chemotherapy in subjects with metastatic pancreatic adenocarcinoma NCT02048384
(Link)
 https://clinicaltrials.gov/ct2/show/NCT02048384?term=NCT02048384&rank=1 		Active, not recruiting

  • 22

  • 18 years and older (adult, older adult)

  • All Sex

 January 23, 2014 to December 2020

  • Drug: metformin alone

  • Drug: rapamycin + metformin

 Metastatic pancreatic adenocarcinoma Metformin inhibits mTOR activity peripheral blood mononuclear cells (PBMC) of subjects treated with metformin with or without rapamycin
Phase II study of metformin in a pre-prostatectomy prostate cancer cohort NCT01433913
(Link)
 https://clinicaltrials.gov/ct2/show/NCT01433913?term=NCT01433913&rank=1 		Completed

  • 20

  • 18 years and older (adult, older adult)

  • Male

 September 9, 2011 to April 2014

  • Drug: metformin and placebo (given orally)

  • Other: laboratory biomarker analysis correlative studies

 1. Adenocarcinoma of the prostate
2. Recurrent prostate cancer
3. Stage I prostate cancer
4. Stage IIA prostate cancer
5. Stage IIB prostate cancer		 Metformin has effects on apoptosis, cell cycle regulation, mTOR regulation, angiogenesis and AMPK activation that lead prevents cancer cells growth
Effect of the addition of metformin hydrochloride on the prognosis of patients with B-cell precursor (Ph+ negative) acute lymphoblastic leukemia with high expression of ABCB1 gene NCT03118128
(Link)
 https://clinicaltrials.gov/ct2/show/NCT03118128?term=NCT03118128&rank=1 		Completed

  • 102

  • 18 years to 70 years (adult, older adult)

  • All sex

 January 1, 2015 to December 1, 2016

  • Drug: metformin


(metformin 850 mg PO three times a day plus prednisone in one 7-day cycle as pre-treatment and during the 28 days induction remission treatment, consolidation therapy and maintenance)		 Acute lymphoblastic leukemia Beneficial effects of metformin on relapse rate and patients’ RFS
APphase I study of temsirolimus in combination with metformin in advanced solid tumors NCT00659568
(Link)
 https://clinicaltrials.gov/ct2/show/NCT00659568?term=NCT00659568&rank=1 		Completed

  • 28

  • 18 years and older (adult, older adult)

  • All sex

 April 15, 2008 to February 2010

  • Drug: metformin hydrochloride

  • Drug: temsirolimus

 1. Breast cancer
2. Endometrial cancer
3. Kidney cancer
4. Lung cancer
5. Lymphoma
6. Unspecified adult solid tumor, protocol specific		 Metformin improved patients’ survival and has antitumor activity, including tumor response rate and time to progression
Phase II study of metformin and 5-fluorouracil in patients with advanced colorectal cancer previously treated with oxaliplatin and irinotecan based chemotherapy NCT01941953
(Link)
 https://clinicaltrials.gov/ct2/show/NCT01941953?term=NCT01941953&rank=1 		Completed

  • 50

  • 16 years and older (child, adult, older adult)

  • All sex

 September 4, 2013 to November 2014

  • Drug: metformin and fluorouracil (metformin 850 mg PO BID plus 5FU 425 mg/m2+ leucovorin 50 mg IV weekly)

 Metastatic colorectal cancer Metformin improved patients’ OS and PFS
A pilot study of metformin as a chemoprevention agent in non-small cell lung cancer NCT01717482
(Link)
 https://clinicaltrials.gov/ct2/show/NCT01717482?term=NCT01717482&rank=1 		Active, not recruiting

  • 24

  • 18 years and older (adult, older adult)

  • All sex

 October 25, 2012 to June 2019

  • Drug: metformin

  • Other: placebo comparator (standard of care observation)

 1. Lung neoplasms
2. Carcinoma, non-small-cell lung		 Metformin decreased level of Ki67