Trial Outcomes & Findings for Treatment of Overweight Induced by Antipsychotic Medication in Young People With Autism Spectrum Disorders (ASD) (NCT NCT01825798)

NCT ID: NCT01825798

Last Updated: 2025-07-16

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

60 participants

Primary outcome timeframe

Baseline, 16 Weeks

Results posted on

2025-07-16

Participant Flow

Participant milestones

Participant milestones
Measure
Placebo Hydrochloride Oral Solution
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Overall Study
STARTED
32
28
Overall Study
COMPLETED
30
23
Overall Study
NOT COMPLETED
2
5

Reasons for withdrawal

Reasons for withdrawal
Measure
Placebo Hydrochloride Oral Solution
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Overall Study
Adverse Event
1
5
Overall Study
Lost to Follow-up
1
0

Baseline Characteristics

Treatment of Overweight Induced by Antipsychotic Medication in Young People With Autism Spectrum Disorders (ASD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Total
n=60 Participants
Total of all reporting groups
Age, Categorical
<=18 years
32 Participants
n=5 Participants
28 Participants
n=7 Participants
60 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Continuous
12.7 years
STANDARD_DEVIATION 2.64 • n=5 Participants
12.9 years
STANDARD_DEVIATION 2.85 • n=7 Participants
12.8 years
STANDARD_DEVIATION 2.7 • n=5 Participants
Sex: Female, Male
Female
8 Participants
n=5 Participants
7 Participants
n=7 Participants
15 Participants
n=5 Participants
Sex: Female, Male
Male
24 Participants
n=5 Participants
21 Participants
n=7 Participants
45 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
2 Participants
n=5 Participants
0 Participants
n=7 Participants
2 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
29 Participants
n=5 Participants
28 Participants
n=7 Participants
57 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
2 Participants
n=5 Participants
2 Participants
n=7 Participants
4 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=5 Participants
3 Participants
n=7 Participants
4 Participants
n=5 Participants
Race (NIH/OMB)
White
28 Participants
n=5 Participants
22 Participants
n=7 Participants
50 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
1 Participants
n=5 Participants
1 Participants
n=7 Participants
2 Participants
n=5 Participants
Region of Enrollment
Canada
8 participants
n=5 Participants
8 participants
n=7 Participants
16 participants
n=5 Participants
Region of Enrollment
United States
24 participants
n=5 Participants
20 participants
n=7 Participants
44 participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Change in Body Mass Index Z-score
0.02 16-wk change in BMI z-score
Interval -0.03 to 0.06
-0.08 16-wk change in BMI z-score
Interval -0.13 to -0.04

SECONDARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Changes in Additional Body Composition Parameters (Absolute Change in Weight)
2.80 16-wk change in weight (kg)
Interval 1.9 to 3.7
0.07 16-wk change in weight (kg)
Interval -0.88 to 1.02

SECONDARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Changes in Additional Body Composition Parameters (Relative Change in Weight)
0.04 16-wk change in weight (z-score)
Interval -0.01 to 0.08
-0.10 16-wk change in weight (z-score)
Interval -0.15 to -0.05

SECONDARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Changes in Additional Body Composition Parameters (Absolute BMI)
0.52 16-wk change in BMI (kg/m2)
Interval 0.18 to 0.87
-0.43 16-wk change in BMI (kg/m2)
Interval -0.8 to -0.06

SECONDARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Changes in Additional Body Composition Parameters (Abdominal Circumference)
1.45 centimetres
Interval 0.39 to 2.51
-0.21 centimetres
Interval -1.32 to 0.91

SECONDARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Changes in Additional Body Composition Parameters (Hip Circumference)
1.06 centimetres
Interval -0.06 to 2.17
-0.63 centimetres
Interval -1.81 to 0.54

SECONDARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Changes in Fasting Metabolic Parameters (Total Cholesterol)
-3.29 16-week change total cholesterol (mg/dL)
Interval -10.33 to 3.75
-1.05 16-week change total cholesterol (mg/dL)
Interval -8.76 to 6.67

SECONDARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Changes in Fasting Metabolic Parameters (LDL)
-0.41 16-week change in LDL (mg/dL)
Interval -6.11 to 5.29
-4.41 16-week change in LDL (mg/dL)
Interval -10.47 to 1.64

SECONDARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Changes in Fasting Metabolic Parameters (HDL)
-0.98 16-week change in HDL (mg/dL)
Interval -5.26 to 3.29
3.27 16-week change in HDL (mg/dL)
Interval -1.41 to 7.94

SECONDARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Changes in Fasting Metabolic Parameters (Triglycerides)
6.18 16-week change in triglycerides (mg/dL)
Interval -19.62 to 31.98
5.74 16-week change in triglycerides (mg/dL)
Interval -22.34 to 33.81

SECONDARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Changes in Fasting Metabolic Parameters (Glucose)
-2.41 16-week change in gluclose (mg/dL)
Interval -5.76 to 0.94
-3.06 16-week change in gluclose (mg/dL)
Interval -6.68 to 0.56

SECONDARY outcome

Timeframe: Baseline, 16 Weeks

Outcome measures

Outcome measures
Measure
Placebo Hydrochloride Oral Solution
n=32 Participants
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 Participants
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Changes in Fasting Metabolic Parameters (Insulin)
2.95 16-week change insulin fasting (µIU/mL)
Interval -8.44 to 14.35
1.97 16-week change insulin fasting (µIU/mL)
Interval -10.44 to 14.38

Adverse Events

Placebo Hydrochloride Oral Solution

Serious events: 1 serious events
Other events: 32 other events
Deaths: 0 deaths

Metformin

Serious events: 0 serious events
Other events: 28 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Placebo Hydrochloride Oral Solution
n=32 participants at risk
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 participants at risk
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Psychiatric disorders
Aggression
3.1%
1/32 • Number of events 1
0.00%
0/28

Other adverse events

Other adverse events
Measure
Placebo Hydrochloride Oral Solution
n=32 participants at risk
Placebo: The placebo hydrochloride oral solution will be prepared to match the appearance, smell and taste of the metformin as closely as possible. For children from 6-9 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, placebo will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if placebo is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if placebo is well-tolerated, the dose will be increased to 850 mg twice daily.
Metformin
n=28 participants at risk
Metformin: Metformin will be dispensed in a liquid suspension of 100 mg/mL. For children 6-9 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. For children from 10-17 years of age, metformin will be started at 250 mg at their evening meal for 1 week, followed by the addition of a 250 mg dose at breakfast for 1 week. At the Week 2 visit, if metformin is well-tolerated, the dose will be increased to 500 mg twice daily. At the Week 4 visit, if metformin is well-tolerated, the dose will be increased to 850 mg twice daily.
Ear and labyrinth disorders
Overall
9.4%
3/32 • Number of events 4
0.00%
0/28
Gastrointestinal disorders
Abdominal Discomfort
9.4%
3/32 • Number of events 4
7.1%
2/28 • Number of events 3
Gastrointestinal disorders
Abdominal Pain
9.4%
3/32 • Number of events 3
10.7%
3/28 • Number of events 3
Gastrointestinal disorders
Abdominal Pain, upper
12.5%
4/32 • Number of events 4
10.7%
3/28 • Number of events 3
Gastrointestinal disorders
Abnormal Feces
0.00%
0/32
10.7%
3/28 • Number of events 3
Gastrointestinal disorders
Chapped lips
6.2%
2/32 • Number of events 2
3.6%
1/28 • Number of events 1
Gastrointestinal disorders
Constipation
9.4%
3/32 • Number of events 4
7.1%
2/28 • Number of events 2
Gastrointestinal disorders
Diarrhea
40.6%
13/32 • Number of events 19
60.7%
17/28 • Number of events 26
Gastrointestinal disorders
Flatulence
9.4%
3/32 • Number of events 4
10.7%
3/28 • Number of events 3
Gastrointestinal disorders
Nausea
9.4%
3/32 • Number of events 3
7.1%
2/28 • Number of events 3
Gastrointestinal disorders
Vomiting
12.5%
4/32 • Number of events 8
28.6%
8/28 • Number of events 9
Gastrointestinal disorders
Overall
59.4%
19/32 • Number of events 55
85.7%
24/28 • Number of events 64
General disorders
Fatigue
18.8%
6/32 • Number of events 6
21.4%
6/28 • Number of events 8
General disorders
Irritability
21.9%
7/32 • Number of events 7
25.0%
7/28 • Number of events 9
General disorders
Product taste abnormal
0.00%
0/32
10.7%
3/28 • Number of events 3
General disorders
Pyrexia
3.1%
1/32 • Number of events 2
10.7%
3/28 • Number of events 4
General disorders
Overall
43.8%
14/32 • Number of events 18
46.4%
13/28 • Number of events 27
Infections and infestations
Ear Infection
3.1%
1/32 • Number of events 1
7.1%
2/28 • Number of events 4
Infections and infestations
Gastroenteritis
3.1%
1/32 • Number of events 1
10.7%
3/28 • Number of events 4
Infections and infestations
Otitis media
3.1%
1/32 • Number of events 1
7.1%
2/28 • Number of events 2
Infections and infestations
Sinusitis
6.2%
2/32 • Number of events 2
3.6%
1/28 • Number of events 1
Infections and infestations
Upper respiratory tract infection
34.4%
11/32 • Number of events 11
21.4%
6/28 • Number of events 6
Infections and infestations
Overall
59.4%
19/32 • Number of events 25
50.0%
14/28 • Number of events 24
Injury, poisoning and procedural complications
Overall
15.6%
5/32 • Number of events 5
14.3%
4/28 • Number of events 4
Investigations
Blood insulin level increased
6.2%
2/32 • Number of events 2
7.1%
2/28 • Number of events 2
Investigations
Blood triglyceride level increased
6.2%
2/32 • Number of events 2
3.6%
1/28 • Number of events 1
Investigations
Overall
15.6%
5/32 • Number of events 7
14.3%
4/28 • Number of events 4
Metabolism and nutrition disorders
Decreased Appetite
12.5%
4/32 • Number of events 4
28.6%
8/28 • Number of events 8
Metabolism and nutrition disorders
Increased Appetite
12.5%
4/32 • Number of events 4
0.00%
0/28
Metabolism and nutrition disorders
Overall
25.0%
8/32 • Number of events 8
28.6%
8/28 • Number of events 8
Musculoskeletal and connective tissue disorders
Back pain
6.2%
2/32 • Number of events 2
0.00%
0/28
Musculoskeletal and connective tissue disorders
Pain in Extremity
3.1%
1/32 • Number of events 1
7.1%
2/28 • Number of events 2
Musculoskeletal and connective tissue disorders
Overall
21.9%
7/32 • Number of events 9
14.3%
4/28 • Number of events 5
Nervous system disorders
Dizziness
3.1%
1/32 • Number of events 1
7.1%
2/28 • Number of events 2
Nervous system disorders
Headache
18.8%
6/32 • Number of events 9
17.9%
5/28 • Number of events 8
Nervous system disorders
Somnolence
9.4%
3/32 • Number of events 3
7.1%
2/28 • Number of events 2
Nervous system disorders
Overall
31.2%
10/32 • Number of events 16
42.9%
12/28 • Number of events 20
Psychiatric disorders
Affect Lability
12.5%
4/32 • Number of events 4
0.00%
0/28
Psychiatric disorders
Aggression
6.2%
2/32 • Number of events 3
17.9%
5/28 • Number of events 6
Psychiatric disorders
Anger
3.1%
1/32 • Number of events 1
14.3%
4/28 • Number of events 4
Psychiatric disorders
Anxiety
9.4%
3/32 • Number of events 3
3.6%
1/28 • Number of events 1
Psychiatric disorders
Depressed Mood
6.2%
2/32 • Number of events 2
0.00%
0/28
Psychiatric disorders
Initial Insomnia
6.2%
2/32 • Number of events 3
10.7%
3/28 • Number of events 3
Psychiatric disorders
Middle Insomnia
3.1%
1/32 • Number of events 1
7.1%
2/28 • Number of events 2
Psychiatric disorders
Overall
40.6%
13/32 • Number of events 27
50.0%
14/28 • Number of events 27
Reproductive system and breast disorders
Overall
6.2%
2/32 • Number of events 2
3.6%
1/28 • Number of events 1
Respiratory, thoracic and mediastinal disorders
Cough
9.4%
3/32 • Number of events 3
7.1%
2/28 • Number of events 2
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
6.2%
2/32 • Number of events 2
10.7%
3/28 • Number of events 4
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
12.5%
4/32 • Number of events 4
7.1%
2/28 • Number of events 3
Respiratory, thoracic and mediastinal disorders
Sneezing
9.4%
3/32 • Number of events 4
7.1%
2/28 • Number of events 2
Respiratory, thoracic and mediastinal disorders
Overall
37.5%
12/32 • Number of events 19
28.6%
8/28 • Number of events 16
Skin and subcutaneous tissue disorders
Rash
6.2%
2/32 • Number of events 2
14.3%
4/28 • Number of events 4
Skin and subcutaneous tissue disorders
Overall
25.0%
8/32 • Number of events 8
21.4%
6/28 • Number of events 7

Additional Information

Dr. Evdokia Anagnostou

Holland Bloorview Kids Rehabilition Hospital

Phone: 416-753-6005

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place