Adrenal Adenoma, Adrenal Carcinoma, AIMAH, Cushing's Syndrome, Ectopic Corticotropin Syndrome, PPNAD
Conditions
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
Brief 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.
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.
Interventions
DRUGOsilodrostat
Osirodrostat 1mg, 5mg & 10mg in the form of film-coated tablets was used for oral administration.
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12 | Baseline, 12 weeks | Percent change from baseline in the mUFC at the individual patient level |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Absolute Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337) | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Absolute change from baseline in the mUFC |
| Percentage Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337) | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Percent change from baseline in the mUFC |
| Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response | 12, 24 and 48 weeks | 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. |
| Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Absolute change from baseline in morning serum cortisol at the individual patient level |
| Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Percentage change from baseline in morning serum cortisol at the individual patient level |
| Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Absolute change from baseline in several steroid hormones at individual levels: ACTH, Serum 11-deoxycorticosterone, Aldosterone, Estradiol |
| Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Percent change from baseline in several steroid hormones at individual levels: ACTH, Serum 11-deoxycorticosterone, Aldosterone, Estradiol |
| Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Absolute change from baseline in several steroid hormones at individual levels: Serum 11-deoxycortisol, Testosterone |
| Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Percent change from baseline in several steroid hormones at individual levels: Serum 11-deoxycortisol, Testosterone |
| Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Absolute change in cardiovascular-related metabolic parameter fasting glucose, associated with Cushing's syndrome (CS) |
| Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Percentasge change in cardiovascular-related metabolic parameter fasting glucose, associated with Cushing's syndrome (CS) |
| Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Absolute change in cardiovascular-related metabolic parameter HbA1c associated with Cushing's syndrome (CS) |
| Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Percentage change in cardiovascular-related metabolic parameter HbA1c associated with Cushing's syndrome (CS) |
| Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Absolute change in cardiovascular-related metabolic parameters: cholesterol, HDL cholesterol, LDL cholesterol & triglycerides, associated with Cushing's syndrome (CS) |
| Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Percent change in cardiovascular-related metabolic parameters: cholesterol, HDL cholesterol, LDL cholesterol & triglycerides, associated with Cushing's syndrome (CS) |
| Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Absolute change in cardiovascular-related metabolic parameter: BMI, associated with Cushing's syndrome (CS) |
| Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Percent change in cardiovascular-related metabolic parameter: BMI, associated with Cushing's syndrome (CS) |
| Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337) | Baseline, Week 24 (day 169) and Week 48 (day 337) | Percent change from baseline in the mUFC at the individual patient level |
| Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Percent change in cardiovascular-related metabolic parameter: Waist circumference, associated with Cushing's syndrome (CS) |
| Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Absolute change in cardiovascular-related metabolic parameter: sitting systolic BP & sitting diastolic BP, associated with Cushing's syndrome (CS) |
| Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Percentage change in cardiovascular-related metabolic parameter: sitting systolic BP & sitting diastolic BP, associated with Cushing's syndrome |
| Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | 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). |
| 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 | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 0 | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 1 | Week 1, 2 hours post-dose | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 3 | Week 3, 2 hours post-dose | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 4 | Week 4, 2 hours post-dose | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 6 | Week 6, 2 hours post-dose | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 8 | Week 8, 2 hours post-dose | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 10 | Week 10, 2 hours post-dose | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 12 | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 16 | Week 16, 2 hours post-dose | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 20 | Week 20, 2 hours post-dose | 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. |
| Plasma Concentrations of Osilodrostat (LCI699) at Week 24 | Week 24, 2 hours post-dose | 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. |
| Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337) | Absolute change in cardiovascular-related metabolic parameter: Waist circumference, associated with Cushing's syndrome (CS) |
Countries
Japan
Participant flow
Recruitment details
Planned subjects: 10 subjects Actual enrolled: 9 subjects
Pre-assignment details
Planned subjects: 10 subjects
Participants by arm
| Arm | Count |
|---|---|
| Osilodrostat Patients in this arm took the study drug, osilodrostat. | 9 |
| Total | 9 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 4 |
| Overall Study | Subject/ Guardian decision | 2 |
Baseline characteristics
| Characteristic | Osilodrostat |
|---|---|
| Age, Continuous | 51.0 years STANDARD_DEVIATION 18.17 |
| Race/Ethnicity, Customized Japanese | 9 participants |
| Sex: Female, Male Female | 7 Participants |
| Sex: Female, Male Male | 2 Participants |
| Type of disease ACTH-Independent Macronodular Adrena;l Hyperplasia | 1 Participants |
| Type of disease Adrenal Adenoma | 5 Participants |
| Type of disease Ectopic Corticotropin Syndrome | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 9 |
| other Total, other adverse events | 9 / 9 |
| serious Total, serious adverse events | 4 / 9 |
Outcome results
Primary
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
Time frame: Baseline, 12 weeks
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12 | AIMAH- Patient 1 (n=1) | -99.0 percentage change |
| Osilodrostat | Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12 | Adrenal adenoma - Patient 1 (n=1) | -97.8 percentage change |
| Osilodrostat | Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12 | Adrenal adenoma - Patient 2 (n=1) | -94.5 percentage change |
| Osilodrostat | Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12 | Adrenal adenoma - Patient 3 (n=1) | -91.5 percentage change |
| Osilodrostat | Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12 | Adrenal adenoma - Patient 4 (n=1) | -81.8 percentage change |
| Osilodrostat | Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12 | Adrenal adenoma - Patient 5 (n=1) | -52.6 percentage change |
| Osilodrostat | Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12 | Ectopic corticotropin syndrome - Patient 1 (n=1) | -99.0 percentage change |
Secondary
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
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 12 (n=1,1,1,1) | -67.7 pmol/L |
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 48 (n=1,1,1,1) | 13.3 pmol/L |
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1,1,1,1) | 0 pmol/L |
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n=1,1,1,1) | 0 pmol/L |
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1,1,1,1) | 0 pmol/L |
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 48 (n=1,1,1,1) | 0 pmol/L |
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1,1,1,1) | 1.1 pmol/L |
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 24 (n=1,1,1,1) | 0 pmol/L |
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 24 (n=1,1,1,1) | -47.3 pmol/L |
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1,1,1,1) | 0 pmol/L |
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 24(n=1,1,1,1) | 0 pmol/L |
| Osilodrostat | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1,1,1,1) | 0 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 24 (n=1,1,1,1) | 3601 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1,1,1,1) | 0 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1,1,1,1) | 2966 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1,1,1,1) | 0 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 12 (n=1,1,1,1) | -696 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1,1,1,1) | 787 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n=1,1,1,1) | 2179 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 24(n=1,1,1,1) | 0 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1,1,1,1) | 5145 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 48 (n=1,1,1,1) | 454 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 48 (n=1,1,1,1) | -91 pmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 24 (n=1,1,1,1) | -91 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1,1,1,1) | 3 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n=1,1,1,1) | 52 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 24 (n=1,1,1,1) | 236 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 48 (n=1,1,1,1) | 170 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1,1,1,1) | -27 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1,1,1,1) | -981 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 24(n=1,1,1,1) | 450 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1,1,1,1) | -147 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1,1,1,1) | 0 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 12 (n=1,1,1,1) | 145 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 24 (n=1,1,1,1) | 40 pmol/L |
| Aldosterone | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 48 (n=1,1,1,1) | 37 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 24(n=1,1,1,1) | -4 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1,1,1,1) | 4 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n=1,1,1,1) | 705 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 12 (n=1,1,1,1) | 51 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1,1,1,1) | -404 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 48 (n=1,1,1,1) | 400 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 48 (n=1,1,1,1) | 11 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 24 (n=1,1,1,1) | -11 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1,1,1,1) | 18 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1,1,1,1) | 77 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 24 (n=1,1,1,1) | 235 pmol/L |
| Estradiol | Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1,1,1,1) | -11 pmol/L |
Secondary
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)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1) | 0.6 kg/m^2 |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1) | -0.5 kg/m^2 |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | AIMAH-Pt 1 Wk 24 (n =1) | -2.3 kg/m^2 |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | AIMAH-Pt 1 Wk 48 (n =1) | -1.6 kg/m^2 |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1) | -1.2 kg/m^2 |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1) | -3.6 kg/m^2 |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1) | 0.7 kg/m^2 |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1) | 0.2 kg/m^2 |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1) | 0.1 kg/m^2 |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n =1) | 1.8 kg/m^2 |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1) | 3.0 kg/m^2 |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1) | 3.6 kg/m^2 |
Secondary
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)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | AIMAH-Patient (Pt) 1 Wk 48 (n =1) | -0.78 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1) | -0.78 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | AIMAH-Patient (Pt) 1 Wk 24 (n =1) | -0.84 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1) | 1.22 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1) | -1.89 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1) | 0.22 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1) | -0.28 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1) | -0.67 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1) | -0.05 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12 (n =1) | -0.33 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1) | -0.39 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1) | 0.06 mmol/L |
Secondary
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)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n=1) | -0.1 percentage of HbA1c |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | AIMAH-Pt 1 Wk 24 (n=1) | -0.1 percentage of HbA1c |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | AIMAH-Pt 1 Wk 48 (n=1) | -0.2 percentage of HbA1c |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1) | 0.3 percentage of HbA1c |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1) | -0.4 percentage of HbA1c |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n=1) | -0.7 percentage of HbA1c |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1) | -1.4 percentage of HbA1c |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1) | -0.9 percentage of HbA1c |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1) | -0.1 percentage of HbA1c |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12 (n=1) | 0.3 percentage of HbA1c |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1) | 0.5 percentage of HbA1c |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1) | 0.8 percentage of HbA1c |
Secondary
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)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n =1,1,1,1) | -0.16 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1,1,1,1) | -0.08 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1,1,1,1) | 1.09 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1,1,1,1) | -0.67 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1,1,1,1) | -0.39 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 48 (n =1,1,1,1) | -0.72 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1,1,1,1) | -1.56 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 24 (n =1,1,1,1) | -0.65 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1,1,1,1) | -0.26 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1,1,1,1) | -0.54 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1,1,1,1) | 0.26 mmol/L |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1,1,1,1) | -0.55 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 24 (n =1,1,1,1) | -1.01 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1,1,1,1) | -0.16 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1,1,1,1) | -0.26 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1,1,1,1) | -0.21 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n =1,1,1,1) | 0.16 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1,1,1,1) | 0.18 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1,1,1,1) | -0.85 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1,1,1,1) | -0.33 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1,1,1,1) | -0.64 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 48 (n =1,1,1,1) | -0.57 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1,1,1,1) | 0.29 mmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1,1,1,1) | 0.49 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1,1,1,1) | 0.72 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1,1,1,1) | 0.21 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 24 (n =1,1,1,1) | 0.18 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 48 (n =1,1,1,1) | -0.08 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1,1,1,1) | -1.11 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1,1,1,1) | -0.26 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1,1,1,1) | 0.31 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1,1,1,1) | -0.19 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1,1,1,1) | -0.18 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n =1,1,1,1) | -0.11 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1,1,1,1) | -0.29 mmol/L |
| Aldosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1,1,1,1) | -0.31 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1,1,1,1) | 0.08 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1,1,1,1) | 0.11 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1,1,1,1) | -0.87 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n =1,1,1,1) | -0.47 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1,1,1,1) | -1.42 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 48 (n =1,1,1,1) | -0.76 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1,1,1,1) | 0.22 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1,1,1,1) | -0.83 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1,1,1,1) | 0.58 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1,1,1,1) | -1.36 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 24 (n =1,1,1,1) | -0.64 mmol/L |
| Estradiol | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1,1,1,1) | 0.44 mmol/L |
Secondary
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)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1Wk 12 (n =1, 1) | -44.3 mmHg |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1 Wk 24 (n =1, 1) | -48.34 mmHg |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1 Wk 48 (n =1, 1) | -40.34 mmHg |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1,1) | 11.67 mmHg |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1,1) | -50.67 mmHg |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n=1,1) | -32.33 mmHg |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1,1) | -16.0 mmHg |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1,1) | -6.67 mmHg |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1,1) | 11.0 mmHg |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n=1,1) | -17.67 mmHg |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1,1) | 4.33 mmHg |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1,1) | -1.0 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1,1) | 4.0 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1Wk 12 (n =1, 1) | -31.0 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1,1) | -6.67 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1 Wk 24 (n =1, 1) | -32.66 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n=1,1) | -13.67 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1 Wk 48 (n =1, 1) | -19.0 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1,1) | 7.33 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1,1) | -3.67 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1,1) | -0.67 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1,1) | -37.0 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1,1) | 3.33 mmHg |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n=1,1) | -26.66 mmHg |
Secondary
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)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | AIMAH-Patient (Pt)1 Wk 12 (n=1) | -2.0 cm |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | AIMAH-Pt1 Wk 24 (n=1) | -7.5 cm |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | AIMAH-Pt1 Wk 48 (n=1) | -5.5 cm |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1) | -2.0 cm |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1) | -1.0 cm |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n=1) | -8.0 cm |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1) | -4.0 cm |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1) | 1.0 cm |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1) | -0.3 cm |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12 (n=1) | 2.5 cm |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1) | 11.0 cm |
| Osilodrostat | Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1) | 9.5 cm |
Secondary
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level
Absolute change from baseline in morning serum cortisol at the individual patient level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | AIMAH-Patient (Pt) 1: Week (Wk) 12 (n = 1) | -607 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | AIMAH-Patient 1: Wk 24 (n = 1) | -571 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | AIMAH-Patient 1: Wk 48 (n = 1) | -580 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 1: Wk 12 (n =1) | -334 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 2: Wk 12(n =1) | -157 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 2: Wk 24 (n =1) | -135 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 3: Wk 12 (n =1) | -300 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 4: Wk 12 (n =1) | -30 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 5: Wk 12 (n =1) | -13 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | Ectopic corticotropin syndrome-Pt 1: Wk 12 (n =1) | -949 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | Ectopic corticotropin syndrome-Pt 1: Wk24 (n=1) | -927 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Morning Serum Cortisol at Individual Level | Ectopic corticotropin syndrome-Pt 1: Wk48 (n=1) | -861 nmol/L |
Secondary
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
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 3 Wk 12 (n = 1,1) | 7.34 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 1 Wk 12 (n = 1,1) | 6.91 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 4 Wk 12 (n = 1,1) | 3.5 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Pt 1 Wk 24 (n = 1,1) | -8.5 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 5 Wk 12 (n = 1,1) | -1.39 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 2 Wk 12 (n = 1,1) | 2.81 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 12(n = 1,1) | -15.81 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Patient (Pt) 1 Wk 12 (n = 1,1) | -4.35 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 24 (n = 1,1) | -12.41 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 2 Wk 24 (n = 1,1) | -0.64 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 48 (n = 1,1) | -2.52 nmol/L |
| Osilodrostat | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Pt 1 Wk 48 (n = 1,1) | -15.54 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 48 (n = 1,1) | -2.01 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Patient (Pt) 1 Wk 12 (n = 1,1) | 0.76 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Pt 1 Wk 24 (n = 1,1) | 0.66 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 1 Wk 12 (n = 1,1) | 0.31 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 2 Wk 12 (n = 1,1) | 0.11 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 2 Wk 24 (n = 1,1) | -0.35 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 3 Wk 12 (n = 1,1) | 0.42 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 4 Wk 12 (n = 1,1) | 0.03 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 5 Wk 12 (n = 1,1) | 0 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 12(n = 1,1) | 5.83 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 24 (n = 1,1) | -2.71 nmol/L |
| Serum 11-deoxycorticosterone | Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Pt 1 Wk 48 (n = 1,1) | -0.03 nmol/L |
Secondary
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
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Osilodrostat | Absolute Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337) | Week 12 (day 85)( n = 7) | -422.10 nmol/24hr |
| Osilodrostat | Absolute Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337) | Week 24 (day 169) (n = 3) | -7428.10 nmol/24hr |
| Osilodrostat | Absolute Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337) | Week 48 (day 337) (n = 2) | -8521.00 nmol/24hr |
Secondary
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
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 12 (n=1,1,0,1) | -71.6 percentage change |
| Osilodrostat | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 48 (n=1,1,0,1) | 14.1 percentage change |
| Osilodrostat | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 24 (n=1,1,0,1) | -50.0 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 48 (n=0,1,0,1) | 24.6 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 24 (n=0,1,0,1) | 195.1 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 12 (n=1,1,0,1) | -100 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 24 (n=1,1,0,1) | -13.1 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 48 (n=1,1,0,1) | -13.1 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =0,1,0,1) | 118.0 percentage change |
| Aldosterone | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =0,0,1,1) | -100 percentage change |
| Aldosterone | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =0,0,1,1) | -75.0 percentage change |
| Aldosterone | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =0,0,1,1) | 45.9 percentage change |
| Aldosterone | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =0,0,1,1) | 10.0 percentage change |
| Aldosterone | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =0,0,1,1) | -13.9 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 48 (n=1,1,0,1) | 15.7 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =0,1,0,1) | 1282 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 24 (n=0,1,0,1) | 427.3 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | AIMAH-Pt 1 Wk 48 (n=0,1,0,1) | 727.3 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =0,0,1,1) | -53.7 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =0,0,1,1) | 1.9 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =0,0,1,1) | -1.9 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =0,0,1,1) | 513.3 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =0,0,1,1) | 120.0 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =0,0,0,1) | -100 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 12 (n=1,1,0,1) | 72.9 percentage change |
| Estradiol | Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level | Ectopic ACTH syndrome-Pt 1: Wk 24 (n=1,1,0,1) | -15.7 percentage change |
Secondary
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)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1) | -2.1 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | AIMAH-Pt 1 Wk 24 (n =1) | -9.6 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | AIMAH-Pt 1 Wk 48 (n =1) | -6.7 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1) | 1.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1) | -3.1 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1) | -9.4 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1) | 3.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1) | 0.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1) | 0.5 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n =1) | 7.4 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1) | 12.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1) | 14.8 percentage change |
Secondary
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)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1) | -15.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | AIMAH-Patient (Pt) 1 Wk 24 (n =1) | -16.4 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | AIMAH-Patient (Pt) 1 Wk 48 (n =1) | -15.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1) | 24.2 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1) | -27.2 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1) | 3.2 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1) | -5.0 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1) | -11.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1) | -0.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12 (n =1) | -5.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1) | -7.0 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1) | 1.1 percentage change |
Secondary
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)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1) | -12.2 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n=1) | -1.8 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | AIMAH-Pt 1 Wk 24 (n=1) | -1.8 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | AIMAH-Pt 1 Wk 48 (n=1) | -3.6 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1) | 6.2 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1) | -6.5 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n=1) | -11.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1) | -20.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1) | -1.5 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12 (n=1) | 5.4 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1) | 8.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1) | 14.3 percentage change |
Secondary
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)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n =1,1,1,1) | -3.8 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1,1,1,1) | -1.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1,1,1,1) | 23.0 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1,1,1,1) | -11.1 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1,1,1,1) | -8.7 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 48 (n =1,1,1,1) | -11.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1,1,1,1) | -23.0 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 24 (n =1,1,1,1) | -10.7 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1,1,1,1) | -6.2 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1,1,1,1) | -11.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1,1,1,1) | 5.7 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1,1,1,1) | -9.5 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 24 (n =1,1,1,1) | -39.5 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1,1,1,1) | -9.4 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1,1,1,1) | -27.1 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1,1,1,1) | -11.4 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n =1,1,1,1) | 12.4 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1,1,1,1) | 15.5 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1,1,1,1) | -33.2 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1,1,1,1) | -17.7 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1,1,1,1) | -34.4 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 48 (n =1,1,1,1) | -22.3 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1,1,1,1) | 22.5 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1,1,1,1) | 38.0 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1,1,1,1) | 26.5 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1,1,1,1) | 6.3 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 24 (n =1,1,1,1) | 5.4 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 48 (n =1,1,1,1) | -2.4 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1,1,1,1) | -24.9 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1,1,1,1) | -10.6 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1,1,1,1) | 12.6 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1,1,1,1) | -5.5 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1,1,1,1) | -9.3 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n =1,1,1,1) | -4.5 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1,1,1,1) | -11.8 percentage change |
| Aldosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1,1,1,1) | -12.6 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n =1,1,1,1) | 9.1 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n =1,1,1,1) | 12.5 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Patient (Pt) 1 Wk 12 (n =1,1,1,1) | -49.4 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n =1,1,1,1) | -32.0 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n =1,1,1,1) | -28.8 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 48 (n =1,1,1,1) | -43.2 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n =1,1,1,1) | 15.0 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n =1,1,1,1) | -56.5 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n =1,1,1,1) | 46.8 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n =1,1,1,1) | -56.7 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | AIMAH-Pt 1 Wk 24 (n =1,1,1,1) | -36.4 percentage change |
| Estradiol | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n =1,1,1,1) | 33.3 percentage change |
Secondary
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
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1Wk 12 (n =1, 1) | -31.1 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1 Wk 24 (n =1, 1) | -33.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1 Wk 48 (n =1, 1) | -28.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1,1) | 9.2 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1,1) | -31.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n=1,1) | -20.0 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1,1) | -12.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1,1) | -4.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1,1) | 9.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n=1,1) | -14.1 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1,1) | 3.4 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1,1) | -0.8 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1,1) | 4.8 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1Wk 12 (n =1, 1) | -32.5 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1,1) | -8.9 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1 Wk 24 (n =1, 1) | -34.3 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12(n=1,1) | -16.5 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | AIMAH-Patient (Pt) 1 Wk 48 (n =1, 1) | -19.9 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1,1) | 9.9 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1,1) | -4.1 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1,1) | -0.8 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1,1) | -33.2 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1,1) | 5.1 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Sitting Blood Pressure (BP) at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n=1,1) | -23.9 percentage change |
Secondary
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)
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | AIMAH-Patient (Pt)1 Wk 12 (n=1) | -2.4 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | AIMAH-Pt1 Wk 24 (n=1) | -9.0 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | AIMAH-Pt1 Wk 48 (n=1) | -6.6 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 1 Wk 12 (n=1) | -1.8 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 2 Wk 12 (n=1) | -0.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 2 Wk 24 (n=1) | -7.1 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 3 Wk 12 (n=1) | -4.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 4 Wk 12 (n=1) | 1.1 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Adrenal adenoma-Pt 5 Wk 12 (n=1) | -0.4 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 12 (n=1) | 2.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1) | 12.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level | Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1) | 11.1 percentage change |
Secondary
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level
Percentage change from baseline in morning serum cortisol at the individual patient level
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 1: Wk 12 (n =1) | -78.0 percentage change |
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | AIMAH-Patient (Pt) 1: Week (Wk) 12 (n = 1) | -73.3 percentage change |
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | AIMAH-Patient 1: Wk 24 (n = 1) | -69.0 percentage change |
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | AIMAH-Patient 1: Wk 48 (n = 1) | -70.0 percentage change |
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 2: Wk 12(n =1) | -50.8 percentage change |
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 2: Wk 24 (n =1) | -43.7 percentage change |
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 3: Wk 12 (n =1) | -56.1 percentage change |
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 4: Wk 12 (n =1) | -8.7 percentage change |
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | Adrenal adenoma-Patient 5: Wk 12 (n =1) | -3.5 percentage change |
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | Ectopic corticotropin syndrome-Pt 1: Wk 12 (n =1) | -71.4 percentage change |
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | Ectopic corticotropin syndrome-Pt 1: Wk24 (n=1) | -69.7 percentage change |
| Osilodrostat | Percentage Change From Baseline in Morning Serum Cortisol at Individual Level | Ectopic corticotropin syndrome-Pt 1: Wk48 (n=1) | -64.7 percentage change |
Secondary
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
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Patient (Pt) 1 Wk 12 (n=1, 1) | -17.8 percentage change |
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Pt 1 Wk 24 (n=1, 1) | -34.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Pt 1 Wk 48 (n=1, 1) | -63.7 percentage change |
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 1 Wk 12 (n=1,1) | 143.7 percentage change |
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 2 Wk 12 (n=1,1) | 108.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 2 Wk 24 (n=1,1) | -24.8 percentage change |
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 3 Wk 12 (n=1, 1) | 127.2 percentage change |
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 4 Wk 12 (n=1, 1) | 267.2 percentage change |
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 5 Wk 12 (n=1,1) | -43.2 percentage change |
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 12 (n=1, 1) | -64.8 percentage change |
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1, 1) | -50.9 percentage change |
| Osilodrostat | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1, 1) | -10.3 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 24 (n=1, 1) | -44.9 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Patient (Pt) 1 Wk 12 (n=1, 1) | 73.1 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 3 Wk 12 (n=1, 1) | 93.3 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Pt 1 Wk 24 (n=1, 1) | 63.5 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 12 (n=1, 1) | 96.5 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | AIMAH-Pt 1 Wk 48 (n=1, 1) | -2.9 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 4 Wk 12 (n=1, 1) | 14.3 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 1 Wk 12 (n=1,1) | 100.0 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Ectopic ACTH syndrome-Pt 1 Wk 48 (n=1, 1) | -33.3 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 2 Wk 12 (n=1,1) | 10.6 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 5 Wk 12 (n=1,1) | 0.0 percentage change |
| Serum 11-deoxycorticosterone | Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels | Adrenal adenoma-Pt 2 Wk 24 (n=1,1) | -33.7 percentage change |
Secondary
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
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Osilodrostat | Percentage Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337) | Week 12 (day 85)( n = 7) | -94.47 percentage change |
| Osilodrostat | Percentage Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337) | Week 24 (day 169) (n = 3) | -91.57 percentage change |
| Osilodrostat | Percentage Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337) | Week 48 (day 337) (n = 2) | -95.04 percentage change |
Secondary
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.
Time frame: 12, 24 and 48 weeks
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response | Week 12: Complete responders | 66.7 Percentage of participants |
| Osilodrostat | Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response | Week 12: Partial responders | 11.1 Percentage of participants |
| Osilodrostat | Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response | Week 12: Overall responders | 77.8 Percentage of participants |
| Osilodrostat | Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response | Week 24: Complete responders (n =3) | 66.7 Percentage of participants |
| Osilodrostat | Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response | Week 24: Partial responders (n = 3) | 33.3 Percentage of participants |
| Osilodrostat | Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response | Week 24: Overall responders (n = 3) | 100 Percentage of participants |
| Osilodrostat | Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response | Week 48: Complete responders (n = 2) | 50.0 Percentage of participants |
| Osilodrostat | Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response | Week 48: Partial responders (n = 2) | 50.0 Percentage of participants |
| Osilodrostat | Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response | Week 48: Overall responders (n = 2) | 100 Percentage of participants |
Secondary
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
Time frame: Baseline, Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337) | AIMAH-Patient (Pt) 1: Week (Wk) 24 (n=1) | -99.5 percentage change |
| Osilodrostat | Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337) | AIMAH-Patient 1:Wk 48 (n=1) | -99.1 percentage change |
| Osilodrostat | Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337) | Adrenal adenoma-Patient 2: Wk 24(n=1) | -85.2 percentage change |
| Osilodrostat | Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337) | Ectopic corticotropin syndrome-Pt 1: Wk 24(n=1) | -91.6 percentage change |
| Osilodrostat | Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337) | Ectopic corticotropin syndrome-Pt 1: Wk 48(n=1) | -91.0 percentage change |
Secondary
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.
Time frame: Week 0
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 0 | Week 0: 0.75 hour post-dose | 0.971 ng/mL | — |
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 0 | Week 0: 2 hours post-dose | 5.11 ng/mL | — |
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 0 | Week 0: 4 hours post-dose | 3.77 ng/mL | — |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 0 | Week 0: 0.75 hour post-dose | 0.405 ng/mL | Standard Deviation 0.673 |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 0 | Week 0: 2 hours post-dose | 5.29 ng/mL | Standard Deviation 4.94 |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 0 | Week 0: 4 hours post-dose | 8.12 ng/mL | Standard Deviation 2.36 |
Secondary
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.
Time frame: Week 1, 2 hours post-dose
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 1 | 10.9 ng/mL | — |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 1 | 10.5 ng/mL | Standard Deviation 6.57 |
| Aldosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 1 | 21.3 ng/mL | — |
| Estradiol | Plasma Concentrations of Osilodrostat (LCI699) at Week 1 | 22.3 ng/mL | Standard Deviation 14.2 |
Secondary
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.
Time frame: Week 10, 2 hours post-dose
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 10 | 4.73 ng/mL | Standard Deviation 1.26 |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 10 | 11.3 ng/mL | Standard Deviation 4.57 |
Secondary
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.
Time frame: Week 12
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 12 | Week 12: 0.75 hour post-dose | 0.382 ng/mL | Standard Deviation 0.171 |
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 12 | Week 12: 2 hours post-dose | 4.62 ng/mL | Standard Deviation 2.51 |
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 12 | Week 12: 4 hours post-dose | 3.58 ng/mL | Standard Deviation 2.3 |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 12 | Week 12: 0.75 hour post-dose | 3.1 ng/mL | — |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 12 | Week 12: 2 hours post-dose | 9.98 ng/mL | — |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 12 | Week 12: 4 hours post-dose | 6.57 ng/mL | — |
Secondary
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.
Time frame: Week 16, 2 hours post-dose
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 16 | 5.71 ng/mL | Standard Deviation 1.33 |
Secondary
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.
Time frame: Week 2
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 0.75 hour post-dose | 3.89 ng/mL | Standard Deviation 2.76 |
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 4 hours post-dose | 7.01 ng/mL | Standard Deviation 2.69 |
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 2 hours post-dose | 8.93 ng/mL | Standard Deviation 2.36 |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 0.75 hour post-dose | 3.69 ng/mL | Standard Deviation 3.3 |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 4 hours post-dose | 11.1 ng/mL | — |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 2 hours post-dose | 14.3 ng/mL | Standard Deviation 7.64 |
| Aldosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 2 hours post-dose | 21.3 ng/mL | Standard Deviation 4.53 |
| Aldosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 0.75 hour post-dose | 33.1 ng/mL | Standard Deviation 19.2 |
| Aldosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 4 hours post-dose | 13.6 ng/mL | Standard Deviation 3.89 |
| Estradiol | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 0.75 hour post-dose | 34.9 ng/mL | Standard Deviation 34.6 |
| Estradiol | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 4 hours post-dose | 29.2 ng/mL | Standard Deviation 0.354 |
| Estradiol | Plasma Concentrations of Osilodrostat (LCI699) at Week 2 | Week 2: 2 hours post-dose | 33.6 ng/mL | Standard Deviation 8.84 |
Secondary
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.
Time frame: Week 20, 2 hours post-dose
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 20 | 0.794 ng/mL |
Secondary
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.
Time frame: Week 24, 2 hours post-dose
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 24 | 6.51 ng/mL | Standard Deviation 2.14 |
Secondary
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.
Time frame: Week 3, 2 hours post-dose
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 3 | 7.09 ng/mL | Standard Deviation 2.07 |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 3 | 7.62 ng/mL | — |
| Aldosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 3 | 23.2 ng/mL | — |
| Estradiol | Plasma Concentrations of Osilodrostat (LCI699) at Week 3 | 37.8 ng/mL | Standard Deviation 0.849 |
Secondary
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.
Time frame: Week 4, 2 hours post-dose
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 4 | 4.08 ng/mL | Standard Deviation 2.61 |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 4 | 9.91 ng/mL | Standard Deviation 3.53 |
| Estradiol | Plasma Concentrations of Osilodrostat (LCI699) at Week 4 | 24.0 ng/mL | Standard Deviation 21 |
Secondary
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.
Time frame: Week 6, 2 hours post-dose
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 6 | 6.74 ng/mL | Standard Deviation 2.42 |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 6 | 12.7 ng/mL | Standard Deviation 9.08 |
| Estradiol | Plasma Concentrations of Osilodrostat (LCI699) at Week 6 | 31.7 ng/mL | — |
Secondary
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.
Time frame: Week 8, 2 hours post-dose
Population: Pharmacokinetic analysis set (PAS): Consisted of all patients who received at least one dose of osilodrostat and had at least one evaluable PK concentration at any visit (post-first-dose).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Osilodrostat | Plasma Concentrations of Osilodrostat (LCI699) at Week 8 | 3.35 ng/mL | Standard Deviation 2.75 |
| Serum 11-deoxycorticosterone | Plasma Concentrations of Osilodrostat (LCI699) at Week 8 | 13.9 ng/mL | — |
Secondary
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.
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | 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 | AIMAH-Patient 1: Week (Wk) 12 (n =1) | 4 scores on a scale |
| Osilodrostat | 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 | AIMAH-Patient 1: Wk 24 (n =1) | 15 scores on a scale |
| Osilodrostat | 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 | AIMAH-Patient 1: Wk 48 (n =1) | 12 scores on a scale |
| Osilodrostat | 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 | Adrenal adenoma-Patient 1: Wk 12 (n =1) | 33 scores on a scale |
| Osilodrostat | 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 | Adrenal adenoma-Patient 2: Wk 12 (n =1) | 35 scores on a scale |
| Osilodrostat | 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 | Adrenal adenoma-Patient 2: Wk 24 (n =1) | 37 scores on a scale |
| Osilodrostat | 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 | Adrenal adenoma-Patient 3: Wk 12 (n =1) | 30 scores on a scale |
| Osilodrostat | 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 | Adrenal adenoma-Patient 4: Wk 12 (n =1) | 26 scores on a scale |
| Osilodrostat | 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 | Adrenal adenoma-Patient 5: Wk 12 (n =1) | 10 scores on a scale |
| Osilodrostat | 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 | Ectopic corticotropin syndrome-Pt 1: Wk 12 (n =1) | 9 scores on a scale |
| Osilodrostat | 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 | Ectopic corticotropin syndrome-Pt 1: Wk 24 (n =1) | 10 scores on a scale |
| Osilodrostat | 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 | Ectopic corticotropin syndrome-Pt 1: Wk 48 (n =1) | 22 scores on a scale |
Secondary
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).
Time frame: Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | AIMAH-Patient 1: Week (Wk) 12 (n=1) | 48 scores on a scale |
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | AIMAH-Patient 1: Wk 24 (n=1) | 51 scores on a scale |
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | AIMAH-Patient 1: Wk 48 (n=1) | 47 scores on a scale |
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | Adrenal adenoma-Patient 1: Wk 12(n=1) | 29 scores on a scale |
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | Adrenal adenoma-Patient 2: Wk 12(n=1) | 18 scores on a scale |
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | Adrenal adenoma-Patient 2: Wk 24 (n = 1) | 22 scores on a scale |
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | Adrenal adenoma-Patient 3: Wk 12 (n =1) | 29 scores on a scale |
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | Adrenal adenoma-Patient 4: Wk 12 (n =1) | 38 scores on a scale |
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | Adrenal adenoma-Patient 5: Wk 12 (n=1) | 45 scores on a scale |
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | Ectopic corticotropin syndrome-Pt 1: Wk 12 (n =1) | 31 scores on a scale |
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | Ectopic corticotropin syndrome-Pt 1: Wk 24 (n =1) | 33 scores on a scale |
| Osilodrostat | Total Scores in Patient-Reported Outcomes Health-related Quality of Life (QoL) as Assessed by Cushing QoL at Individual Level | Ectopic corticotropin syndrome-Pt 1: Wk 48 (n =1) | 23 scores on a scale |