Trial Outcomes & Findings for Study of Pharmacodynamics of LIK066 in Overweight and Obese Women With Polycystic Ovary Syndrome (NCT NCT03152591)
NCT ID: NCT03152591
Last Updated: 2021-01-05
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
29 participants
Primary outcome timeframe
Baseline, Day 15
Results posted on
2021-01-05
Participant Flow
This study was conducted in 5 centers in 2 countries: Germany (3), and USA (2).
Participants were randomized in the ratio of 1:1 to receive either LIK066 50 mg tid or placebo for 14 days and morning dose on Day 15.
Participant milestones
| Measure |
LIK066
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
|---|---|---|
|
Overall Study
STARTED
|
15
|
14
|
|
Overall Study
Pharmacokinetic (PK) Analysis Set
|
14
|
0
|
|
Overall Study
Pharmacodynamic (PD) Analysis Set
|
15
|
14
|
|
Overall Study
COMPLETED
|
15
|
14
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Pharmacodynamics of LIK066 in Overweight and Obese Women With Polycystic Ovary Syndrome
Baseline characteristics by cohort
| Measure |
LIK066
n=15 Participants
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
n=14 Participants
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Total
n=29 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
26.1 Years
STANDARD_DEVIATION 4.76 • n=5 Participants
|
29.1 Years
STANDARD_DEVIATION 5.66 • n=7 Participants
|
27.6 Years
STANDARD_DEVIATION 5.34 • n=5 Participants
|
|
Sex/Gender, Customized
Female
|
15 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
15 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
|
Average fasting total testosterone
|
1.98 nmol/L
STANDARD_DEVIATION 0.841 • n=5 Participants
|
2.07 nmol/L
STANDARD_DEVIATION 0.608 • n=7 Participants
|
2.02 nmol/L
STANDARD_DEVIATION 0.724 • n=5 Participants
|
|
Average fasting free testosterone
|
0.037 nmol/L
STANDARD_DEVIATION 0.0163 • n=5 Participants
|
0.032 nmol/L
STANDARD_DEVIATION 0.0086 • n=7 Participants
|
0.034 nmol/L
STANDARD_DEVIATION 0.0132 • n=5 Participants
|
|
Sex hormone binding globulin (SHBG)
|
18.3 nmol/L
STANDARD_DEVIATION 7.72 • n=5 Participants
|
24.6 nmol/L
STANDARD_DEVIATION 10.51 • n=7 Participants
|
21.4 nmol/L
STANDARD_DEVIATION 9.58 • n=5 Participants
|
|
Free androgen Index
|
12.4 ratio
STANDARD_DEVIATION 6.65 • n=5 Participants
|
9.0 ratio
STANDARD_DEVIATION 2.77 • n=7 Participants
|
10.7 ratio
STANDARD_DEVIATION 5.27 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Day 15Population: Pharmacodynamic (PD) analysis set
Outcome measures
| Measure |
LIK066
n=15 Participants
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
n=14 Participants
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
|---|---|---|
|
Change in Average Morning Fasting Free Testosterone Blood Concentrations From Baseline
|
0.91 nmol/L
Interval 0.77 to 1.07
|
1.03 nmol/L
Interval 0.88 to 1.21
|
SECONDARY outcome
Timeframe: Baseline, Day 15Population: Pharmacodynamic (PD) analysis set
Outcome measures
| Measure |
LIK066
n=15 Participants
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
n=14 Participants
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
|---|---|---|
|
Change From Baseline in Luteinizing Hormone (LH) at Day 15
|
1.37 U/L
Interval 1.11 to 1.69
|
1.10 U/L
Interval 0.89 to 1.36
|
SECONDARY outcome
Timeframe: Baseline, Day 15Population: Pharmacodynamic (PD) analysis set
Outcome measures
| Measure |
LIK066
n=15 Participants
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
n=14 Participants
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
|---|---|---|
|
Change From Baseline in Follicle Stimulating Hormone (FSH) at Day 15
|
1.13 U/L
Interval 0.89 to 1.43
|
0.89 U/L
Interval 0.7 to 1.13
|
SECONDARY outcome
Timeframe: Baseline, Day 15Population: Pharmacodynamic (PD) analysis set
Outcome measures
| Measure |
LIK066
n=15 Participants
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
n=14 Participants
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
|---|---|---|
|
Change From Baseline in Sex Hormone Binding Globulin (SHBG) at Day 15
|
1.06 nmol/L
Interval 0.95 to 1.2
|
0.93 nmol/L
Interval 0.83 to 1.03
|
SECONDARY outcome
Timeframe: Baseline, Day 15Population: Pharmacodynamic (PD) analysis set
Outcome measures
| Measure |
LIK066
n=15 Participants
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
n=14 Participants
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
|---|---|---|
|
Change From Baseline in Androstenedione at Day 15
|
0.85 nmol/L
Interval 0.74 to 0.97
|
1.03 nmol/L
Interval 0.91 to 1.17
|
SECONDARY outcome
Timeframe: Baseline, Day 15Population: Pharmacodynamic (PD) analysis set
Outcome measures
| Measure |
LIK066
n=15 Participants
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
n=14 Participants
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
|---|---|---|
|
Change From Baseline in Dehydroepiandrostenedione (DHEA) at Day 15
|
0.75 nmol/L
Interval 0.58 to 0.98
|
1.09 nmol/L
Interval 0.85 to 1.39
|
SECONDARY outcome
Timeframe: Baseline, Day 15Population: Pharmacodynamic (PD) analysis set
Outcome measures
| Measure |
LIK066
n=15 Participants
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
n=14 Participants
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
|---|---|---|
|
Change From Baseline in Dehydroepiandrostenedione Sulfate (DHEAS) at Day 15
|
0.84 umol/L
Interval 0.75 to 0.94
|
1.10 umol/L
Interval 0.99 to 1.23
|
SECONDARY outcome
Timeframe: Baseline, Day 15Population: Pharmacodynamic (PD) analysis set
Outcome measures
| Measure |
LIK066
n=15 Participants
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
n=14 Participants
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
|---|---|---|
|
Change From Baseline in Total Testosterone, at Day 15
|
0.95 nmol/L
Interval 0.84 to 1.06
|
1.04 nmol/L
Interval 0.92 to 1.17
|
SECONDARY outcome
Timeframe: Baseline, Day 15Population: Pharmacodynamic (PD) analysis set
Free Androgen Index (FAI) is a ratio used to determine abnormal androgen status in humans. The ratio is the total testosterone level divided by the sex hormone binding globulin (SHBG) level, and then multiplying by 100. FAI has no units.
Outcome measures
| Measure |
LIK066
n=15 Participants
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
n=14 Participants
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
|---|---|---|
|
Change From Baseline in Free Androgen Index (FAI), at Day 15
|
0.85 Ratio
Interval 0.69 to 1.05
|
1.07 Ratio
Interval 0.88 to 1.31
|
Adverse Events
LIK066
Serious events: 0 serious events
Other events: 15 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
LIK066
n=15 participants at risk
LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
Placebo
n=14 participants at risk
Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
|
|---|---|---|
|
Gastrointestinal disorders
ABDOMINAL DISTENSION
|
13.3%
2/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
13.3%
2/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Gastrointestinal disorders
ABDOMINAL PAIN UPPER
|
6.7%
1/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Gastrointestinal disorders
DIARRHOEA
|
100.0%
15/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
21.4%
3/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Gastrointestinal disorders
DYSPEPSIA
|
6.7%
1/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Gastrointestinal disorders
FLATULENCE
|
40.0%
6/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Gastrointestinal disorders
GASTROINTESTINAL TRACT IRRITATION
|
0.00%
0/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
7.1%
1/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Gastrointestinal disorders
NAUSEA
|
33.3%
5/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
14.3%
2/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Gastrointestinal disorders
VOMITING
|
0.00%
0/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
7.1%
1/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
General disorders
THIRST
|
26.7%
4/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
7.1%
1/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Infections and infestations
NASOPHARYNGITIS
|
13.3%
2/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
7.1%
1/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Infections and infestations
RHINITIS
|
6.7%
1/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Infections and infestations
VAGINAL INFECTION
|
6.7%
1/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Infections and infestations
VULVOVAGINAL MYCOTIC INFECTION
|
6.7%
1/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Investigations
MENSTRUATION NORMAL
|
26.7%
4/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
14.3%
2/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Metabolism and nutrition disorders
DECREASED APPETITE
|
6.7%
1/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Musculoskeletal and connective tissue disorders
MUSCULOSKELETAL DISCOMFORT
|
0.00%
0/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
7.1%
1/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Nervous system disorders
HEADACHE
|
26.7%
4/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
7.1%
1/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Nervous system disorders
MIGRAINE
|
0.00%
0/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
7.1%
1/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Psychiatric disorders
INSOMNIA
|
6.7%
1/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Psychiatric disorders
MOOD ALTERED
|
6.7%
1/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Renal and urinary disorders
POLYURIA
|
0.00%
0/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
7.1%
1/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Reproductive system and breast disorders
HYPOMENORRHOEA
|
13.3%
2/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
7.1%
1/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Reproductive system and breast disorders
MENSTRUAL DISORDER
|
0.00%
0/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
7.1%
1/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Respiratory, thoracic and mediastinal disorders
DRY THROAT
|
0.00%
0/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
7.1%
1/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
|
Skin and subcutaneous tissue disorders
RASH
|
6.7%
1/15 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
0.00%
0/14 • Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER