Trial Outcomes & Findings for Metformin for Ectopic Fat Deposition and Metabolic Markers in Polycystic Ovary Syndrome (PCOS) (NCT NCT02500147)

Results Overview

To compare percentage liver fat by magnetic resonance spectroscopy in the metformin group and placebo group to baseline and between the groups in order to determine if metformin is efficacious for reducing liver fat compared to placebo in adolescents and young women with Polycystic Ovary Syndrome (PCOS)

Recruitment status

TERMINATED

Study phase

PHASE4

Target enrollment

3 participants

Primary outcome timeframe

6 months

Results posted on

2025-07-02

Participant Flow

Participant milestones

Participant milestones
Measure	Metformin Adolescents and young adults with PCOS and liver fat greater than or equal to 4.8% will be randomized to metformin or placebo. Metformin ER (extended release) will be administered as two pills of 500 mg each. For the first week subjects will take one pill orally on a daily basis and thereafter will take two pills orally on a daily basis. The intervention will last six months.	Placebo Adolescents and young adults with PCOS and liver fat greater than or equal to 4.8% will be randomized to metformin or placebo. Subjects randomized to placebo will be instructed to take one pill daily by mouth for one week and then to take one pill daily by mouth for the remainder of six months.
Overall Study STARTED	1	2
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	1	2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Metformin n=1 Participants Adolescents and young adults with PCOS and liver fat greater than or equal to 4.8% will be randomized to metformin or placebo. Metformin ER (extended release) will be administered as two pills of 500 mg each. For the first week subjects will take one pill orally on a daily basis and thereafter will take two pills orally on a daily basis. The intervention will last six months.	Placebo n=2 Participants Adolescents and young adults with PCOS and liver fat greater than or equal to 4.8% will be randomized to metformin or placebo. Subjects randomized to placebo will be instructed to take one pill daily by mouth for one week and then to take one pill daily by mouth for the remainder of six months.	Total n=3 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=1 Participants	0 Participants n=2 Participants	0 Participants n=3 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=1 Participants	2 Participants n=2 Participants	2 Participants n=3 Participants
Age, Categorical >=65 years	1 Participants n=1 Participants	0 Participants n=2 Participants	1 Participants n=3 Participants
Sex: Female, Male Female	1 Participants n=1 Participants	2 Participants n=2 Participants	3 Participants n=3 Participants
Sex: Female, Male Male	0 Participants n=1 Participants	0 Participants n=2 Participants	0 Participants n=3 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment United States	1 participants n=1 Participants	2 participants n=2 Participants	3 participants n=3 Participants

PRIMARY outcome

Timeframe: 6 months

To compare percentage liver fat by magnetic resonance spectroscopy in the metformin group and placebo group to baseline and between the groups in order to determine if metformin is efficacious for reducing liver fat compared to placebo in adolescents and young women with Polycystic Ovary Syndrome (PCOS)

Outcome measures

Outcome measures
Measure	Metformin n=1 Participants Adolescents and young adults with PCOS and liver fat greater than or equal to 4.8% will be randomized to metformin or placebo. Metformin ER (extended release) will be administered as two pills of 500 mg each. For the first week subjects will take one pill orally on a daily basis and thereafter will take two pills orally on a daily basis. The intervention will last six months.	Placebo n=2 Participants Adolescents and young adults with PCOS and liver fat greater than or equal to 4.8% will be randomized to metformin or placebo. Subjects randomized to placebo will be instructed to take one pill daily by mouth for one week and then to take one pill daily by mouth for the remainder of six months.
Percentage Liver Fat in Participants	7.4 percent Standard Deviation NA Standard deviation cannot be calculated from one data point	3.1 percent Standard Deviation 2.6

SECONDARY outcome

Timeframe: 6 months

Population: Data was not collected due to early termination of study

The association of percent liver fat with insulin resistance as measured by HOMA-IR will be measured by correlation/regression. Change in HOMA-IR with change in percent liver fat following metformin will be assessed using multiple regression analysis.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 months

Population: Study was terminated before any participant could have M30 measured.

The association of percent liver fat with M30 will be measured by correlation/regression. Change in M30 with change in percent liver fat following metformin will be assessed using multiple regression analysis.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 months

Population: Study was terminated before any participant could have pancreatic polypeptides measured.

The association of percent liver fat with pancreatic polypeptide will be measured by correlation/regression. Change in pancreatic polypeptide with change in percent liver fat following metformin will be assessed using multiple regression analysis.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 months

Population: Study was terminated before any participant could have adipose tissue measured.

The association of percent liver fat with total body adipose tissue will be measured by correlation/regression. Change in total body adipose tissue with change in percent liver fat following metformin will be assessed using multiple regression analysis.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 months

Population: Study was terminated before any participant could have adipose tissue measured.

The association of percent liver fat with visceral adipose tissue will be measured by correlation/regression. Change in visceral adipose tissue with change in percent liver fat following metformin will be assessed using multiple regression analysis.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 months

Population: Study was terminated before any participant could have triglycerides measured.

The association of percent liver fat with triglycerides will be measured by correlation/regression. Change in triglycerides with change in percent liver fat following metformin will be assessed using multiple regression analysis.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 6 months

Population: Study was terminated before any participant could have data collected for the purposes of determining the presence of PNPLA3 allele.

The proportion of PCOS subjects with the high risk I148M PNPLA3 allele in the PCOS groups with elevated and normal liver fat will be compared using a chi-squared or Fisher's Exact test.

Outcome measures

Outcome data not reported

Adverse Events

Metformin

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Aviva B Sopher, MD, MS

Columbia University

Phone: 212-305-9362

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place