Polycystic Ovary SyndromePhase 2PCOSNCT03152591

Study of Pharmacodynamics of LIK066 in Overweight and Obese Women With Polycystic Ovary Syndrome

A Phase 2 study testing LIK066 in people with Polycystic Ovary Syndrome, run by Novartis Pharmaceuticals at 5 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of the study was to evaluate whether LIK066 can be developed for the treatment of polycystic ovary syndrome (PCOS) in overweight and obese women. Read more about this study

Who is it for?: Ages 18 to 40 years, women only
Conditions the study is for: Polycystic Ovary Syndrome
What is being tested?: LIK066
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 29 participants
Where: 5 sitesin 2 countries
Study dates: 24 Jul 2017to 25 Jun 2018
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 40 years
Women only
No healthy volunteers
Adult

Who may be able to join

PCOS (diagnosed as clinical or biochemical hyperandrogenism, amenorrhea or oligomenorrhea and exclusion of other causes of hyperandrogenism.
Overweight/obese female subjects with BMI of 28 - 45 kg/m^2, inclusive, and stable weight +/- 3 kg over previous 3 months
Subjects must use non-hormonal methods of contraception during the study.

Who cannot join

Subjects with exogenous causes of hirsutism
Menstruation in the 30 days prior to screening or treatment
Pregnant or nursing (lactating) women
Use of prohibited medications
Preexisting medical condition which may significantly alter the absorption, metabolism, or excretion of the study drug, or which may jeopardize the subject in case of participation in the study

Trial locations and contacts

5 sites in 2 countries.

Country

City, postcode or hospital

5 sites in 2 countries

Germany3 sites

Novartis Investigative Site
Berlin, 10117
Novartis Investigative Site
Essen, 45147
Novartis Investigative Site
Freiburg im Breisgau, 79106

Other trials in Germany

United States2 sites

Novartis Investigative Site
Philadelphia, Pennsylvania, 19104
Novartis Investigative Site
Springfield, Missouri, 65802

Other trials in United States

About this study

Official title: A Randomized, Subject- and Investigator-blinded, Placebo-controlled Pharmacodynamic Study of Oral LIK066 in Overweight and Obese Women With Polycystic Ovary Syndrome

The purpose of the study was to evaluate whether LIK066 can be developed for the treatment of polycystic ovary syndrome (PCOS) in overweight and obese women.

Conditions: Polycystic Ovary Syndrome
Being tested: LIK066, Placebo
Keywords: Polycystic ovary syndrome, PCOS, hyperandrogenism, insulin resistance, overweight, obesity
Medical subject headings: Polycystic Ovary Syndrome, Hyperandrogenism, Insulin Resistance, Overweight, Obesity

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

LIK066Drug
LIK066 tablets for oral administration
PlaceboDrug
Placebo tablets matching LIK066 tablets, for oral administration

Study groups in NCT03152591
Study group	Role	What they receive
LIK066	Experimental	LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test Receives: Drug: LIK066
Placebo	Placebo comparator	Placebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change in Average Morning Fasting Free Testosterone Blood Concentrations From Baseline
Measured: Baseline, Day 15

Secondary outcomes (8)

Change From Baseline in Luteinizing Hormone (LH) at Day 15
Measured: Baseline, Day 15
Change From Baseline in Follicle Stimulating Hormone (FSH) at Day 15
Measured: Baseline, Day 15
Change From Baseline in Sex Hormone Binding Globulin (SHBG) at Day 15
Measured: Baseline, Day 15
Change From Baseline in Androstenedione at Day 15
Measured: Baseline, Day 15
Change From Baseline in Dehydroepiandrostenedione (DHEA) at Day 15
Measured: Baseline, Day 15
Change From Baseline in Dehydroepiandrostenedione Sulfate (DHEAS) at Day 15
Measured: Baseline, Day 15
Change From Baseline in Total Testosterone, at Day 15
Measured: Baseline, Day 15
Change From Baseline in Free Androgen Index (FAI), at Day 15
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.
Measured: Baseline, Day 15

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

24 Jul 201725 Jun 2018

First posted15 May 2017
Study start24 Jul 2017
Primary completion25 Jun 2018
Study completion25 Jun 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Polycystic Ovary SyndromeStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Basic science
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 29 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03152591
Sponsor's protocol ID: CLIK066X2205
Other IDs: 2017-001373-16, 2017
Acronym: PCOS
Status last verified: Sep 2020
First submitted: 11 May 2017
Last updated: 5 Jan 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Undecided: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Recruiting
Efficacy of Ovarian Drilling Via vNOTES Laparoscopy in Patients With PCOS
PCOS (Polycystic Ovary Syndrome)
RecruitingPhase 1
Efficacy of Injectable Vitamin D Supplementation in Females With Polycystic Ovary Syndrome
Anovulatory Infertility
RecruitingObservational study
The Value of LC-MS/MS Detection of Androgen Profiles in the Diagnosis of PCOS: A Prospective, Multicenter Real-world Study
Polycystic Ovary Syndrome (PCOS)
Recruiting
The Effects of the Mediterranean Diet and Synbiotics in Polycystic Ovary Syndrome
PCOS (Polycystic Ovary Syndrome)
Recruiting
Brown Adipose Tissue Modulation in Polycystic Ovary Syndrome Using Infrared Thermography
Polycystic Ovary Syndrome (PCOS)

All Polycystic Ovary Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 5 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Subject- and Investigator-blinded, Placebo-controlled Pharmacodynamic Study of Oral LIK066 in Overweight and Obese Women With Polycystic Ovary Syndrome
ClinicalTrials.gov ID: NCT03152591
Acronym / sponsor ID: PCOS / CLIK066X2205
Conditions: Polycystic Ovary Syndrome
Keywords: Polycystic ovary syndrome, PCOS, hyperandrogenism, insulin resistance, overweight, obesity
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 29 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 24 Jul 2017 (actual)
Primary completion: 25 Jun 2018 (actual)
Study completion: 25 Jun 2018 (actual)
First posted: 15 May 2017 (actual)
Results: Posted 9 May 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The purpose of the study was to evaluate whether LIK066 can be developed for the treatment of polycystic ovary syndrome (PCOS) in overweight and obese women.

Study design

Primary purpose: Basic science
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
LIK066	Experimental	Drug: LIK066LIK066 tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test
Placebo	Placebo comparator	Drug: PlaceboPlacebo tablets received three times daily; before breakfast, lunch and dinner for 14 days and once on day 15 morning before meal test

Who may qualify

Ages: 18 to 40 years
Sex: Female only
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (3 listed)

PCOS (diagnosed as clinical or biochemical hyperandrogenism, amenorrhea or oligomenorrhea and exclusion of other causes of hyperandrogenism.
Overweight/obese female subjects with BMI of 28 - 45 kg/m^2, inclusive, and stable weight +/- 3 kg over previous 3 months
Subjects must use non-hormonal methods of contraception during the study.

Key exclusion criteria (5 listed)

Subjects with exogenous causes of hirsutism
Menstruation in the 30 days prior to screening or treatment
Pregnant or nursing (lactating) women
Use of prohibited medications
Preexisting medical condition which may significantly alter the absorption, metabolism, or excretion of the study drug, or which may jeopardize the subject in case of participation in the study

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
LIK066LIK066 tablets for oral administration	Drug	LIK066
PlaceboPlacebo tablets matching LIK066 tablets, for oral administration	Drug	Placebo

Primary outcome

Change in Average Morning Fasting Free Testosterone Blood Concentrations From Baseline
Time frame: Baseline, Day 15

8 secondary outcomes

Change From Baseline in Luteinizing Hormone (LH) at Day 15
Time frame: Baseline, Day 15
Change From Baseline in Follicle Stimulating Hormone (FSH) at Day 15
Time frame: Baseline, Day 15
Change From Baseline in Sex Hormone Binding Globulin (SHBG) at Day 15
Time frame: Baseline, Day 15
Change From Baseline in Androstenedione at Day 15
Time frame: Baseline, Day 15
Change From Baseline in Dehydroepiandrostenedione (DHEA) at Day 15
Time frame: Baseline, Day 15
Change From Baseline in Dehydroepiandrostenedione Sulfate (DHEAS) at Day 15
Time frame: Baseline, Day 15
Change From Baseline in Total Testosterone, at Day 15
Time frame: Baseline, Day 15
Change From Baseline in Free Androgen Index (FAI), at Day 15
Time frame: Baseline, Day 15

Results and safety

Results posted to the registry on 9 May 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
LIK066	15	15
Placebo	14	14

Primary outcome: Change in Average Morning Fasting Free Testosterone Blood Concentrations From Baseline

Time frame: Baseline, Day 15. Geometric mean (90% Confidence Interval). Unit: nmol/L

Measure	LIK066 n = 15	Placebo n = 14
Value	0.91 (0.77 to 1.07)	1.03 (0.88 to 1.21)

LIK066 vs Placebo: Ratio LIK066/Placebo: 0.88 (90% CI 0.70 to 1.11), p = 0.353, t-test, 2 sided

Population analysed: Pharmacodynamic (PD) analysis set

8 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse Events were collected for the maximum duration of participants' treatment exposure plus any follow up period, approximately 2 months.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
LIK066	0 of 15 (0%)	15 of 15 (100%)	0 of 15
Placebo	0 of 14 (0%)	10 of 14 (71.4%)	0 of 14

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
A Plain Language Trial Summary is available on novartisclinicatrials.comLink provided by the sponsor.
Individual participant dataSharing planned: Undecided. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Related trials for Polycystic Ovary Syndrome

Recruiting
Efficacy of Ovarian Drilling Via vNOTES Laparoscopy in Patients With PCOS
PCOS (Polycystic Ovary Syndrome)
RecruitingPhase 1
Efficacy of Injectable Vitamin D Supplementation in Females With Polycystic Ovary Syndrome
Anovulatory Infertility
RecruitingObservational study
The Value of LC-MS/MS Detection of Androgen Profiles in the Diagnosis of PCOS: A Prospective, Multicenter Real-world Study
Polycystic Ovary Syndrome (PCOS)
Recruiting
The Effects of the Mediterranean Diet and Synbiotics in Polycystic Ovary Syndrome
PCOS (Polycystic Ovary Syndrome)
Recruiting
Brown Adipose Tissue Modulation in Polycystic Ovary Syndrome Using Infrared Thermography
Polycystic Ovary Syndrome (PCOS)

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Novartis Pharmaceuticals study director

Novartis Pharmaceuticals

5 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing LIK066 in people with Polycystic Ovary Syndrome, run by Novartis Pharmaceuticals at 5 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in Germany, United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 May 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in Average Morning Fasting Free Testosterone Blood Concentrations From Baseline (time frame: Baseline, Day 15). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03152591.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2021.