Cushing's SyndromePhase 2NCT02468193
Study of Efficacy and Safety of Osilodrostat in Cushing's Syndrome
A Phase 2 study testing Osilodrostat in people with Cushing's Syndrome and Ectopic Corticotropin Syndrome, run by Novartis Pharmaceuticals at 4 sites in Japan.
Trial summary
The study aim was to investigate the efficacy and safety of Osilodrostat in patients with Cushing's syndrome due to causes other than Cushing's disease in Japan. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Cushing's Syndrome, Ectopic Corticotropin Syndrome, Adrenal Adenoma
- What is being tested?
- Osilodrostat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 4 sitesJapan
- Study dates
- 24 Sep 2015to 29 Oct 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 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with confirmed Cushing's syndrome [i.e. ectopic corticotropin syndrome, adrenal adenoma, adrenal carcinoma, ACTH-Independent Macronodular Adrenal Hyperplasia (AIMAH), or Primary Pigmented Nodular Adrenal Dysplasia (PPNAD)]
- For patients on medical treatment for hypercortisolism due to Cushing's syndrome, the washout periods had to be completed prior to baseline efficacy assessments
Who cannot join
- Patients with Cushing's disease
- History of hypersensitivity to osilodrostat or to drugs of similar chemical classes
- History of malignancy of any organ system, treated or untreated, within the past 5 years
- Patients receiving treatment for within 4 weeks or ≤5 x half-life of the agent (whichever is longer) before first dose of osilodrostat
- Patients with risk factors for QTc prolongation or Torsade de Pointes
Trial locations and contacts
4 sites in 1 country.
Japan4 sites
Novartis Investigative Site
Chiba, 260 8677
Novartis Investigative Site
Fukushima, Fukushima, 960 1295
Novartis Investigative Site
Sendai, Miyagi, 980 8574
Novartis Investigative Site
Yokohama, Kanagawa, 245-8575
About this study
Official title: A Phase II, Open-label, Dose Titration, Multi-center Study to Assess the Safety/Tolerability and Efficacy of Osilodrostat in Patients With All Types of Endogenous Cushing's Syndrome Except Cushing's Disease
The study aim was to investigate the efficacy and safety of Osilodrostat in patients with Cushing's syndrome due to causes other than Cushing's disease in Japan.
Read the detailed description
This was a Phase II, single arm, open-label, dose titration, multi-center study which consisted of two distinct Study Periods plus an optional extension period in non-CD patients with CS. The 3 Study Periods (two distinct Study Periods plus an optional extension period) were as follows:
Study Period I [Week 0 (Day 1) to Week-12]: Study Period I was the dose titration period to achieve a stable therapeutic dose and to assess the efficacy and safety of osilodrostat.
The dosing regimen of osilodrostat in this study was titrated according to the following escalation sequence: osilodrostat 2 mg bid, 5 mg bid, 10 mg bid, 20 mg bid, and 30 mg bid. Dose adjustments were based on the serum cortisol values measured by the local lab at each site. Osilodrostat titration was done weekly for the initial 4-weeks, up to a maximum dose of 10 mg bid.
The mean of three 24-hour UFC (mUFC) values were measured to evaluate the efficacy in this period.
Study Period II (After Week-12 to Week-48): Study Period II was the period to assess the sustainability of efficacy and long term safety.
During Study Period II, only patients who tolerated and agreed to continue osilodrostat treatment continued on the study. The patient was administered with the stable therapeutic dose which was achieved in the Study Period I.
Optional extension period (After Week-48): Patients who continued to receive clinical benefit, as assessed by the study Investigator and who wished to enter the extension period were reconsented at Week-48. Patients who entered the extension period continued to be treated with the study drug without interruption to be assessed for efficacy and safety. Patients who continued to benefit from study treatment as assessed by the study investigator and who completed Week-72 were offered to participate in a separate long-term safety follow-up study. The optional extension period ended after all patients had completed Week-72 or had discontinued early.
Post-treatment Follow-up: All patients had 30 days safety follow-up after the last dose of study treatment.
- Conditions
- Cushing's Syndrome, Ectopic Corticotropin Syndrome, Adrenal Adenoma, Adrenal Carcinoma, AIMAH, PPNAD
- Being tested
- Osilodrostat
- Keywords
- Cushing's syndrome, osilodrostat, LCI699, ectopic corticotropin syndrome, adrenal adenoma, adrenal carcinoma, ACTH-Independent Macronodular Adrenal Hyperplasia, AIMAH, Primary Pigmented Nodular Adrenal Dysplasia, PPNAD
- Medical subject headings
- Cushing Syndrome, ACTH Syndrome, Ectopic, Adrenal Cortex Neoplasms, Acth-Independent Macronodular Adrenal Hyperplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OsilodrostatDrug
Also called LCI699
Osirodrostat 1mg, 5mg \& 10mg in the form of film-coated tablets was used for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| Osilodrostat | Experimental | Patients in this arm took the study drug, osilodrostat. Receives: Drug: Osilodrostat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12
Percent change from baseline in the mUFC at the individual patient level
Measured: Baseline, 12 weeks
Secondary outcomes (36)
Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337)
Percent change from baseline in the mUFC at the individual patient level
Measured: Baseline, Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)
Absolute change from baseline in the mUFC
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)
Percent change from baseline in the mUFC
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response
Complete response rate = percentage of participants who had mUFC≤ ULN; Partial response rate = Percentage of participants who had mUFC>ULN and at least 50% reduction from baseline in mUFC. Overall response rate = Percentage of participants who had mUFC ≤ ULN or at least 50% reduction from baseline.
Measured: 12, 24 and 48 weeks
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level
Absolute change from baseline in morning serum cortisol at the individual patient level
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level
Percentage change from baseline in morning serum cortisol at the individual patient level
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level
Absolute change from baseline in several steroid hormones at individual levels: ACTH, Serum 11-deoxycorticosterone, Aldosterone, Estradiol
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level
Percent change from baseline in several steroid hormones at individual levels: ACTH, Serum 11-deoxycorticosterone, Aldosterone, Estradiol
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels
Absolute change from baseline in several steroid hormones at individual levels: Serum 11-deoxycortisol, Testosterone
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels
Percent change from baseline in several steroid hormones at individual levels: Serum 11-deoxycortisol, Testosterone
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level
Absolute change in cardiovascular-related metabolic parameter fasting glucose, associated with Cushing's syndrome (CS)
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level
Percentasge change in cardiovascular-related metabolic parameter fasting glucose, associated with Cushing's syndrome (CS)
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level
Absolute change in cardiovascular-related metabolic parameter HbA1c associated with Cushing's syndrome (CS)
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level
Percentage change in cardiovascular-related metabolic parameter HbA1c associated with Cushing's syndrome (CS)
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level
Absolute change in cardiovascular-related metabolic parameters: cholesterol, HDL cholesterol, LDL cholesterol \& triglycerides, associated with Cushing's syndrome (CS)
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level
Percent change in cardiovascular-related metabolic parameters: cholesterol, HDL cholesterol, LDL cholesterol \& triglycerides, associated with Cushing's syndrome (CS)
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level
Absolute change in cardiovascular-related metabolic parameter: BMI, associated with Cushing's syndrome (CS)
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level
Percent change in cardiovascular-related metabolic parameter: BMI, associated with Cushing's syndrome (CS)
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level
Absolute change in cardiovascular-related metabolic parameter: Waist circumference, associated with Cushing's syndrome (CS)
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level
Percent change in cardiovascular-related metabolic parameter: Waist circumference, associated with Cushing's syndrome (CS)
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level
Absolute change in cardiovascular-related metabolic parameter: sitting systolic BP \& sitting diastolic BP, associated with Cushing's syndrome (CS)
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level
Percentage change in cardiovascular-related metabolic parameter: sitting systolic BP \& sitting diastolic BP, associated with Cushing's syndrome
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level
The Cushing's Disease Health-Related Quality of Life Questionnaire (Cushing QoL) (version 1.0) was developed to evaluate quality of life in patients with Cushing's syndrome (Webb et al 2008). The Cushing QoL is comprised of 12 items that capture patient responses on seven concepts: daily activities, healing and pain, mood and self-confidence, social concerns, physical appearance, memory and concern about the future. Each questionnaire of the Cushing QOL has a scale of 1-5 where '1' corresponding to 'Always' or 'Very much' and '5' to 'Never' or 'Not at all'. The lower the score, the greater the impact on HRQoL. The score is the sum of all the item response and can range from 12 (worst) to 60 points (best).
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Beck Depression Inventory II (BDI-ll) Depression Score at Individual Level
The Beck Depression Inventory II (BDI-II) is a patient reported instrument that consists of 21 items designed to assess the intensity of depression in clinical \& normal patients in the preceding two weeks. Each item is a list of four statements arranged in increasing severity about a particular symptom of depression. Each of 21 items corresponds to a symptom of depression and the sum of total score will be calculated where each item has a four-point scale ranging from 0 to 3, leading to a total score from zero to 63.
Measured: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Plasma Concentrations of Osilodrostat (LCI699) at Week 0
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 0
Plasma Concentrations of Osilodrostat (LCI699) at Week 1
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 1, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 2
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 2
Plasma Concentrations of Osilodrostat (LCI699) at Week 3
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 3, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 4
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 4, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 6
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 6, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 8
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 8, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 10
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 10, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 12
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 12
Plasma Concentrations of Osilodrostat (LCI699) at Week 16
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 16, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 20
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 20, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 24
Osilodrostat plasma concentration data at each time-point were summarized by incident dose. About a half of pre-dose concentration data were excluded from analysis due to deviation from the pre-defined acceptable time window.
Measured: Week 24, 2 hours post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2015 (estimated)
- Study start24 Sep 2015
- Primary completion7 Jun 2018
- Study completion29 Oct 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 Cushing's 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02468193
- Sponsor's protocol ID
- CLCI699C1201
- Status last verified
- Apr 2020
- First submitted
- 8 Jun 2015
- Last updated
- 6 May 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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 expert 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 is currently available according to the process described on www.clinicalstudydatarequest.com
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open-label, Dose Titration, Multi-center Study to Assess the Safety/Tolerability and Efficacy of Osilodrostat in Patients With All Types of Endogenous Cushing's Syndrome Except Cushing's Disease
- ClinicalTrials.gov ID
- NCT02468193
- Acronym / sponsor ID
- CLCI699C1201
- Conditions
- Cushing's Syndrome, Ectopic Corticotropin Syndrome, Adrenal Adenoma, Adrenal Carcinoma, AIMAH, PPNAD
- Keywords
- Cushing's syndrome, osilodrostat, LCI699, ectopic corticotropin syndrome, adrenal adenoma, adrenal carcinoma, ACTH-Independent Macronodular Adrenal Hyperplasia, AIMAH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 24 Sep 2015 (actual)
- Primary completion
- 7 Jun 2018 (actual)
- Study completion
- 29 Oct 2018 (actual)
- First posted
- 10 Jun 2015 (estimated)
- Results
- Posted 11 Mar 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study aim was to investigate the efficacy and safety of Osilodrostat in patients with Cushing's syndrome due to causes other than Cushing's disease in Japan.
Detailed description
This was a Phase II, single arm, open-label, dose titration, multi-center study which consisted of two distinct Study Periods plus an optional extension period in non-CD patients with CS. The 3 Study Periods (two distinct Study Periods plus an optional extension period) were as follows:
Study Period I [Week 0 (Day 1) to Week-12]: Study Period I was the dose titration period to achieve a stable therapeutic dose and to assess the efficacy and safety of osilodrostat.
The dosing regimen of osilodrostat in this study was titrated according to the following escalation sequence: osilodrostat 2 mg bid, 5 mg bid, 10 mg bid, 20 mg bid, and 30 mg bid. Dose adjustments were based on the serum cortisol values measured by the local lab at each site. Osilodrostat titration was done weekly for the initial 4-weeks, up to a maximum dose of 10 mg bid.
The mean of three 24-hour UFC (mUFC) values were measured to evaluate the efficacy in this period.
Study Period II (After Week-12 to Week-48): Study Period II was the period to assess the sustainability of efficacy and long term safety.
During Study Period II, only patients who tolerated and agreed to continue osilodrostat treatment continued on the study. The patient was administered with the stable therapeutic dose which was achieved in the Study Period I.
Optional extension period (After Week-48): Patients who continued to receive clinical benefit, as assessed by the study Investigator and who wished to enter the extension period were reconsented at Week-48. Patients who entered the extension period continued to be treated with the study drug without interruption to be assessed for efficacy and safety. Patients who continued to benefit from study treatment as assessed by the study investigator and who completed Week-72 were offered to participate in a separate long-term safety follow-up study. The optional extension period ended after all patients had completed Week-72 or had discontinued early.
Post-treatment Follow-up: All patients had 30 days safety follow-up after the last dose of study treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Osilodrostat | Experimental | Drug: OsilodrostatPatients in this arm took the study drug, osilodrostat. |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patients with confirmed Cushing's syndrome [i.e. ectopic corticotropin syndrome, adrenal adenoma, adrenal carcinoma, ACTH-Independent Macronodular Adrenal Hyperplasia (AIMAH), or Primary Pigmented Nodular Adrenal Dysplasia (PPNAD)]
- For patients on medical treatment for hypercortisolism due to Cushing's syndrome, the washout periods had to be completed prior to baseline efficacy assessments
Key exclusion criteria (5 listed)
- Patients with Cushing's disease
- History of hypersensitivity to osilodrostat or to drugs of similar chemical classes
- History of malignancy of any organ system, treated or untreated, within the past 5 years
- Patients receiving treatment for within 4 weeks or ≤5 x half-life of the agent (whichever is longer) before first dose of osilodrostat
- Patients with risk factors for QTc prolongation or Torsade de Pointes
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 |
|---|---|---|
| OsilodrostatAlso: LCI699Osirodrostat 1mg, 5mg \& 10mg in the form of film-coated tablets was used for oral administration. | Drug | Osilodrostat |
Primary outcome
Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12
Time frame: Baseline, 12 weeks
Percent change from baseline in the mUFC at the individual patient level
36 secondary outcomes
Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337)
Time frame: Baseline, Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response
Time frame: 12, 24 and 48 weeks
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Beck Depression Inventory II (BDI-ll) Depression Score at Individual Level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Plasma Concentrations of Osilodrostat (LCI699) at Week 0
Time frame: Week 0
Plasma Concentrations of Osilodrostat (LCI699) at Week 1
Time frame: Week 1, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 2
Time frame: Week 2
Plasma Concentrations of Osilodrostat (LCI699) at Week 3
Time frame: Week 3, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 4
Time frame: Week 4, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 6
Time frame: Week 6, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 8
Time frame: Week 8, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 10
Time frame: Week 10, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 12
Time frame: Week 12
Plasma Concentrations of Osilodrostat (LCI699) at Week 16
Time frame: Week 16, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 20
Time frame: Week 20, 2 hours post-dose
Plasma Concentrations of Osilodrostat (LCI699) at Week 24
Time frame: Week 24, 2 hours post-dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. 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 expert 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 is currently available according to the process described on www.clinicalstudydatarequest.com
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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
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Osilodrostat in people with Cushing's Syndrome and Ectopic Corticotropin Syndrome, run by Novartis Pharmaceuticals at 4 sites in Japan.
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 2 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 6 May 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Japan. 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. 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 11 Mar 2020). 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?
Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12 (time frame: Baseline, 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 6 May 2020.