Trial Outcomes & Findings for Study of Efficacy and Safety of Osilodrostat in Cushing's Syndrome (NCT NCT02468193)

NCT ID: NCT02468193

Last Updated: 2020-05-06

Results Overview

Percent change from baseline in the mUFC at the individual patient level

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

Baseline, 12 weeks

Results posted on

2020-05-06

Participant Flow

Planned subjects: 10 subjects Actual enrolled: 9 subjects

Planned subjects: 10 subjects

Participant milestones

Participant milestones
Measure	Osilodrostat Patients in this arm took the study drug, osilodrostat.
Overall Study STARTED	9
Overall Study Completed Week-12 (Study Period I)	7
Overall Study Discontinued at or Prior to Week-12	2
Overall Study Completed Week-12, Did Not Enter Per. II	3
Overall Study Completed Week-12, Entered Study Per. II	4
Overall Study Completed Week-48 (Study Period II)	2
Overall Study Discontinued at or Prior to Week48	2
Overall Study Compl. Wk-48, Didn't Enter Opt. Ext. Per	0
Overall Study Compl. Wk-48, Entered Opt. Ext. Period	2
Overall Study Completed Optional Extension Period	0
Overall Study Discontinued Study in Optional Ext. Per.	2
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Osilodrostat Patients in this arm took the study drug, osilodrostat.
Overall Study Adverse Event	4
Overall Study Subject/ Guardian decision	2

Baseline Characteristics

Study of Efficacy and Safety of Osilodrostat in Cushing's Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Osilodrostat n=9 Participants Patients in this arm took the study drug, osilodrostat.
Age, Continuous	51.0 years STANDARD_DEVIATION 18.17 • n=93 Participants
Sex: Female, Male Female	7 Participants n=93 Participants
Sex: Female, Male Male	2 Participants n=93 Participants
Race/Ethnicity, Customized Japanese	9 participants n=93 Participants
Type of disease ACTH-Independent Macronodular Adrena;l Hyperplasia	1 Participants n=93 Participants
Type of disease Adrenal Adenoma	5 Participants n=93 Participants
Type of disease Ectopic Corticotropin Syndrome	3 Participants n=93 Participants

PRIMARY outcome

Timeframe: Baseline, 12 weeks

Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.

Percent change from baseline in the mUFC at the individual patient level

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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 outcome

Timeframe: 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.

Percent change from baseline in the mUFC at the individual patient level

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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 outcome

Timeframe: 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.

Absolute change from baseline in the mUFC

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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 Interval -10487.5 to -157.0	—	—	—
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 Interval -9702.0 to -367.9	—	—	—
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 Interval -9640.5 to -7401.5	—	—	—

SECONDARY outcome

Timeframe: 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.

Percent change from baseline in the mUFC

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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 Interval -99.5 to -85.2	—	—	—
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 Interval -99.0 to -52.6	—	—	—
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 Interval -99.1 to -91.0	—	—	—

SECONDARY outcome

Timeframe: 12, 24 and 48 weeks

Population: Full Analysis Set (FAS): Comprised of all enrolled patients who received at least one dose of osilodrostat.

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.

Outcome measures

Outcome measures
Measure	Osilodrostat n=9 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response Week 12: Overall responders	77.8 Percentage of participants Interval 40.0 to 97.2	—	—	—
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response Week 24: Complete responders (n =3)	66.7 Percentage of participants Interval 9.4 to 99.2	—	—	—
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response Week 24: Overall responders (n = 3)	100 Percentage of participants Interval 29.2 to 100.0	—	—	—
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response Week 48: Complete responders (n = 2)	50.0 Percentage of participants Interval 1.3 to 98.7	—	—	—
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response Week 48: Partial responders (n = 2)	50.0 Percentage of participants Interval 1.3 to 98.7	—	—	—
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response Week 12: Complete responders	66.7 Percentage of participants Interval 29.9 to 92.5	—	—	—
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response Week 12: Partial responders	11.1 Percentage of participants Interval 0.3 to 48.2	—	—	—
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response Week 24: Partial responders (n = 3)	33.3 Percentage of participants Interval 0.8 to 90.6	—	—	—
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response Week 48: Overall responders (n = 2)	100 Percentage of participants Interval 15.8 to 100.0	—	—	—

SECONDARY outcome

Timeframe: 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.

Absolute change from baseline in morning serum cortisol at the individual patient level

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level AIMAH-Patient (Pt) 1: Week (Wk) 12 (n = 1)	-607 nmol/L	—	—	—
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level AIMAH-Patient 1: Wk 24 (n = 1)	-571 nmol/L	—	—	—
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level AIMAH-Patient 1: Wk 48 (n = 1)	-580 nmol/L	—	—	—
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 1: Wk 12 (n =1)	-334 nmol/L	—	—	—
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 2: Wk 12(n =1)	-157 nmol/L	—	—	—
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 2: Wk 24 (n =1)	-135 nmol/L	—	—	—
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 3: Wk 12 (n =1)	-300 nmol/L	—	—	—
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 4: Wk 12 (n =1)	-30 nmol/L	—	—	—
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 5: Wk 12 (n =1)	-13 nmol/L	—	—	—
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level Ectopic corticotropin syndrome-Pt 1: Wk 12 (n =1)	-949 nmol/L	—	—	—
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level Ectopic corticotropin syndrome-Pt 1: Wk24 (n=1)	-927 nmol/L	—	—	—
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level Ectopic corticotropin syndrome-Pt 1: Wk48 (n=1)	-861 nmol/L	—	—	—

SECONDARY outcome

Timeframe: 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.

Percentage change from baseline in morning serum cortisol at the individual patient level

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level AIMAH-Patient (Pt) 1: Week (Wk) 12 (n = 1)	-73.3 percentage change	—	—	—
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level AIMAH-Patient 1: Wk 24 (n = 1)	-69.0 percentage change	—	—	—
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level AIMAH-Patient 1: Wk 48 (n = 1)	-70.0 percentage change	—	—	—
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 1: Wk 12 (n =1)	-78.0 percentage change	—	—	—
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 2: Wk 12(n =1)	-50.8 percentage change	—	—	—
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 2: Wk 24 (n =1)	-43.7 percentage change	—	—	—
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 3: Wk 12 (n =1)	-56.1 percentage change	—	—	—
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 4: Wk 12 (n =1)	-8.7 percentage change	—	—	—
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level Adrenal adenoma-Patient 5: Wk 12 (n =1)	-3.5 percentage change	—	—	—
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level Ectopic corticotropin syndrome-Pt 1: Wk 12 (n =1)	-71.4 percentage change	—	—	—
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level Ectopic corticotropin syndrome-Pt 1: Wk24 (n=1)	-69.7 percentage change	—	—	—
Percentage Change From Baseline in Morning Serum Cortisol at Individual Level Ectopic corticotropin syndrome-Pt 1: Wk48 (n=1)	-64.7 percentage change	—	—	—

SECONDARY outcome

Timeframe: 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.

Absolute change from baseline in several steroid hormones at individual levels: ACTH, Serum 11-deoxycorticosterone, Aldosterone, Estradiol

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=7 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone n=7 Participants adrenal steroid hormones: Aldosterone	Estradiol n=7 Participants adrenal steroid hormones: 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)	0 pmol/L	3601 pmol/L	236 pmol/L	235 pmol/L
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	454 pmol/L	170 pmol/L	400 pmol/L
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	2966 pmol/L	-27 pmol/L	-404 pmol/L
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	5145 pmol/L	-981 pmol/L	4 pmol/L
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	787 pmol/L	3 pmol/L	18 pmol/L
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	2179 pmol/L	52 pmol/L	705 pmol/L
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	0 pmol/L	450 pmol/L	-4 pmol/L
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	0 pmol/L	-147 pmol/L	77 pmol/L
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	0 pmol/L	0 pmol/L	-11 pmol/L
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	-696 pmol/L	145 pmol/L	51 pmol/L
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	-91 pmol/L	40 pmol/L	-11 pmol/L
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	-91 pmol/L	37 pmol/L	11 pmol/L

SECONDARY outcome

Timeframe: 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.

Percent change from baseline in several steroid hormones at individual levels: ACTH, Serum 11-deoxycorticosterone, Aldosterone, Estradiol

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=7 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone n=7 Participants adrenal steroid hormones: Aldosterone	Estradiol n=7 Participants adrenal steroid hormones: 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)	14.1 percentage change	-13.1 percentage change	—	15.7 percentage change
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	—	1282 percentage change
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	—	427.3 percentage change
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	—	727.3 percentage change
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	-53.7 percentage change
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	1.9 percentage change
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	-1.9 percentage change
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	513.3 percentage change
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	120.0 percentage change
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
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	-100 percentage change	—	72.9 percentage change
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	-13.1 percentage change	—	-15.7 percentage change

SECONDARY outcome

Timeframe: 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.

Absolute change from baseline in several steroid hormones at individual levels: Serum 11-deoxycortisol, Testosterone

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=7 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	0.76 nmol/L	—	—
Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels AIMAH-Pt 1 Wk 24 (n = 1,1)	-8.5 nmol/L	0.66 nmol/L	—	—
Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels AIMAH-Pt 1 Wk 48 (n = 1,1)	-15.54 nmol/L	-0.03 nmol/L	—	—
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	0.31 nmol/L	—	—
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	0.11 nmol/L	—	—
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	-0.35 nmol/L	—	—
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	0.42 nmol/L	—	—
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	0.03 nmol/L	—	—
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	0 nmol/L	—	—
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	5.83 nmol/L	—	—
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	-2.71 nmol/L	—	—
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	-2.01 nmol/L	—	—

SECONDARY outcome

Timeframe: 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.

Percent change from baseline in several steroid hormones at individual levels: Serum 11-deoxycortisol, Testosterone

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=7 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	73.1 percentage change	—	—
Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels AIMAH-Pt 1 Wk 24 (n=1, 1)	-34.9 percentage change	63.5 percentage change	—	—
Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels AIMAH-Pt 1 Wk 48 (n=1, 1)	-63.7 percentage change	-2.9 percentage change	—	—
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	100.0 percentage change	—	—
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	10.6 percentage change	—	—
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	-33.7 percentage change	—	—
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	93.3 percentage change	—	—
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	14.3 percentage change	—	—
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	0.0 percentage change	—	—
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	96.5 percentage change	—	—
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	-44.9 percentage change	—	—
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	-33.3 percentage change	—	—

SECONDARY outcome

Timeframe: 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.

Absolute change in cardiovascular-related metabolic parameter fasting glucose, associated with Cushing's syndrome (CS)

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—

SECONDARY outcome

Timeframe: 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.

Percentasge change in cardiovascular-related metabolic parameter fasting glucose, associated with Cushing's syndrome (CS)

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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 outcome

Timeframe: 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.

Absolute change in cardiovascular-related metabolic parameter HbA1c associated with Cushing's syndrome (CS)

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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 outcome

Timeframe: 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.

Percentage change in cardiovascular-related metabolic parameter HbA1c associated with Cushing's syndrome (CS)

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level AIMAH-Pt 1 Wk 24 (n=1)	-1.8 percentage change	—	—	—
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level AIMAH-Pt 1 Wk 48 (n=1)	-3.6 percentage change	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—

SECONDARY outcome

Timeframe: 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.

Absolute change in cardiovascular-related metabolic parameters: cholesterol, HDL cholesterol, LDL cholesterol \& triglycerides, associated with Cushing's syndrome (CS)

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=7 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone n=7 Participants adrenal steroid hormones: Aldosterone	Estradiol n=7 Participants adrenal steroid hormones: 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)	-0.54 mmol/L	-0.64 mmol/L	-0.26 mmol/L	0.11 mmol/L
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	-0.33 mmol/L	0.31 mmol/L	0.08 mmol/L
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	-0.16 mmol/L	-0.19 mmol/L	-1.36 mmol/L
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	0.16 mmol/L	-0.11 mmol/L	-0.47 mmol/L
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	0.49 mmol/L	-0.29 mmol/L	-0.83 mmol/L
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	0.29 mmol/L	-0.31 mmol/L	0.22 mmol/L
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	-0.85 mmol/L	0.21 mmol/L	-0.87 mmol/L
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	-1.01 mmol/L	0.18 mmol/L	-0.64 mmol/L
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	-0.57 mmol/L	-0.08 mmol/L	-0.76 mmol/L
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	-0.26 mmol/L	-1.11 mmol/L	-1.42 mmol/L
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	0.18 mmol/L	0.72 mmol/L	0.58 mmol/L
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	-0.21 mmol/L	-0.18 mmol/L	0.44 mmol/L

SECONDARY outcome

Timeframe: 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.

Percent change in cardiovascular-related metabolic parameters: cholesterol, HDL cholesterol, LDL cholesterol \& triglycerides, associated with Cushing's syndrome (CS)

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=7 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone n=7 Participants adrenal steroid hormones: Aldosterone	Estradiol n=7 Participants adrenal steroid hormones: 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)	-11.1 percentage change	-33.2 percentage change	6.3 percentage change	-49.4 percentage change
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	-39.5 percentage change	5.4 percentage change	-36.4 percentage change
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	-22.3 percentage change	-2.4 percentage change	-43.2 percentage change
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	-27.1 percentage change	-24.9 percentage change	-28.8 percentage change
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	-34.4 percentage change	-10.6 percentage change	12.5 percentage change
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	-9.4 percentage change	-5.5 percentage change	-56.7 percentage change
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	15.5 percentage change	26.5 percentage change	46.8 percentage change
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	-11.4 percentage change	-9.3 percentage change	33.3 percentage change
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	38.0 percentage change	-11.8 percentage change	-56.5 percentage change
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	22.5 percentage change	-12.6 percentage change	15.0 percentage change
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	-17.7 percentage change	12.6 percentage change	9.1 percentage change
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	12.4 percentage change	-4.5 percentage change	-32.0 percentage change

SECONDARY outcome

Timeframe: 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.

Absolute change in cardiovascular-related metabolic parameter: BMI, associated with Cushing's syndrome (CS)

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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 outcome

Timeframe: 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.

Percent change in cardiovascular-related metabolic parameter: BMI, associated with Cushing's syndrome (CS)

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level AIMAH-Pt 1 Wk 24 (n =1)	-9.6 percentage change	—	—	—
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level AIMAH-Pt 1 Wk 48 (n =1)	-6.7 percentage change	—	—	—

SECONDARY outcome

Timeframe: 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.

Absolute change in cardiovascular-related metabolic parameter: Waist circumference, associated with Cushing's syndrome (CS)

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level AIMAH-Pt1 Wk 24 (n=1)	-7.5 cm	—	—	—
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	—	—	—
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	—	—	—
Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level AIMAH-Pt1 Wk 48 (n=1)	-5.5 cm	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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 outcome

Timeframe: 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.

Percent change in cardiovascular-related metabolic parameter: Waist circumference, associated with Cushing's syndrome (CS)

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level AIMAH-Pt1 Wk 24 (n=1)	-9.0 percentage change	—	—	—
Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level AIMAH-Pt1 Wk 48 (n=1)	-6.6 percentage change	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—

SECONDARY outcome

Timeframe: 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.

Absolute change in cardiovascular-related metabolic parameter: sitting systolic BP \& sitting diastolic BP, associated with Cushing's syndrome (CS)

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=7 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	4.0 mmHg	—	—
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	-0.67 mmHg	—	—
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	-31.0 mmHg	—	—
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	-32.66 mmHg	—	—
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	-19.0 mmHg	—	—
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	-3.67 mmHg	—	—
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	-37.0 mmHg	—	—
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	-26.66 mmHg	—	—
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	-6.67 mmHg	—	—
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	-13.67 mmHg	—	—
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	7.33 mmHg	—	—
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	3.33 mmHg	—	—

SECONDARY outcome

Timeframe: 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.

Percentage change in cardiovascular-related metabolic parameter: sitting systolic BP \& sitting diastolic BP, associated with Cushing's syndrome

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=7 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	-34.3 percentage change	—	—
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	-19.9 percentage change	—	—
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	9.9 percentage change	—	—
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	-0.8 percentage change	—	—
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	-32.5 percentage change	—	—
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	-4.1 percentage change	—	—
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	-33.2 percentage change	—	—
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	-23.9 percentage change	—	—
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	-8.9 percentage change	—	—
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	5.1 percentage change	—	—
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	-16.5 percentage change	—	—
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	4.8 percentage change	—	—

SECONDARY outcome

Timeframe: 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.

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).

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure	Osilodrostat n=7 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—
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	—	—	—

SECONDARY outcome

Timeframe: 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).

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.

Outcome measures

Outcome measures
Measure	Osilodrostat n=1 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=8 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 0 Week 0: 0.75 hour post-dose	0.971 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	0.405 ng/mL Standard Deviation 0.673	—	—
Plasma Concentrations of Osilodrostat (LCI699) at Week 0 Week 0: 2 hours post-dose	5.11 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	5.29 ng/mL Standard Deviation 4.94	—	—
Plasma Concentrations of Osilodrostat (LCI699) at Week 0 Week 0: 4 hours post-dose	3.77 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	8.12 ng/mL Standard Deviation 2.36	—	—

SECONDARY outcome

Timeframe: Week 1, 2 hours post-dose

Outcome measures

Outcome measures
Measure	Osilodrostat n=1 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=5 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone n=1 Participants adrenal steroid hormones: Aldosterone	Estradiol n=2 Participants adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 1	10.9 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	10.5 ng/mL Standard Deviation 6.57	21.3 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	22.3 ng/mL Standard Deviation 14.2

SECONDARY outcome

Timeframe: Week 2

Outcome measures

Outcome measures
Measure	Osilodrostat n=2 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=2 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone n=2 Participants adrenal steroid hormones: Aldosterone	Estradiol n=2 Participants adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 2 Week 2: 4 hours post-dose	7.01 ng/mL Standard Deviation 2.69	11.1 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	13.6 ng/mL Standard Deviation 3.89	29.2 ng/mL Standard Deviation 0.354
Plasma Concentrations of Osilodrostat (LCI699) at Week 2 Week 2: 0.75 hour post-dose	3.89 ng/mL Standard Deviation 2.76	3.69 ng/mL Standard Deviation 3.3	33.1 ng/mL Standard Deviation 19.2	34.9 ng/mL Standard Deviation 34.6
Plasma Concentrations of Osilodrostat (LCI699) at Week 2 Week 2: 2 hours post-dose	8.93 ng/mL Standard Deviation 2.36	14.3 ng/mL Standard Deviation 7.64	21.3 ng/mL Standard Deviation 4.53	33.6 ng/mL Standard Deviation 8.84

SECONDARY outcome

Timeframe: Week 3, 2 hours post-dose

Outcome measures

Outcome measures
Measure	Osilodrostat n=4 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=1 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone n=1 Participants adrenal steroid hormones: Aldosterone	Estradiol n=2 Participants adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 3	7.09 ng/mL Standard Deviation 2.07	7.62 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	23.2 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	37.8 ng/mL Standard Deviation 0.849

SECONDARY outcome

Timeframe: Week 4, 2 hours post-dose

Outcome measures

Outcome measures
Measure	Osilodrostat n=4 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=2 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol n=2 Participants adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 4	4.08 ng/mL Standard Deviation 2.61	9.91 ng/mL Standard Deviation 3.53	—	24.0 ng/mL Standard Deviation 21.00

SECONDARY outcome

Timeframe: Week 6, 2 hours post-dose

Outcome measures

Outcome measures
Measure	Osilodrostat n=4 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=2 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol n=1 Participants adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 6	6.74 ng/mL Standard Deviation 2.42	12.7 ng/mL Standard Deviation 9.08	—	31.7 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation

SECONDARY outcome

Timeframe: Week 8, 2 hours post-dose

Outcome measures

Outcome measures
Measure	Osilodrostat n=5 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=1 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 8	3.35 ng/mL Standard Deviation 2.75	13.9 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	—	—

SECONDARY outcome

Timeframe: Week 10, 2 hours post-dose

Outcome measures

Outcome measures
Measure	Osilodrostat n=4 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=2 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 10	4.73 ng/mL Standard Deviation 1.26	11.3 ng/mL Standard Deviation 4.57	—	—

SECONDARY outcome

Timeframe: Week 12

Outcome measures

Outcome measures
Measure	Osilodrostat n=2 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone n=1 Participants adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 12 Week 12: 4 hours post-dose	3.58 ng/mL Standard Deviation 2.3	6.57 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	—	—
Plasma Concentrations of Osilodrostat (LCI699) at Week 12 Week 12: 0.75 hour post-dose	0.382 ng/mL Standard Deviation 0.171	3.1 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	—	—
Plasma Concentrations of Osilodrostat (LCI699) at Week 12 Week 12: 2 hours post-dose	4.62 ng/mL Standard Deviation 2.51	9.98 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	—	—

SECONDARY outcome

Timeframe: Week 16, 2 hours post-dose

Outcome measures

Outcome measures
Measure	Osilodrostat n=2 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 16	5.71 ng/mL Standard Deviation 1.33	—	—	—

SECONDARY outcome

Timeframe: Week 20, 2 hours post-dose

Outcome measures

Outcome measures
Measure	Osilodrostat n=1 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 20	0.794 ng/mL Standard Deviation NA N/A : not enough participants to calculate the standard deviation	—	—	—

SECONDARY outcome

Timeframe: Week 24, 2 hours post-dose

Outcome measures

Outcome measures
Measure	Osilodrostat n=2 Participants Patients in this arm took the study drug, osilodrostat.	Serum 11-deoxycorticosterone adrenal steroid hormones: Serum 11-deoxycorticosterone	Aldosterone adrenal steroid hormones: Aldosterone	Estradiol adrenal steroid hormones: Estradiol
Plasma Concentrations of Osilodrostat (LCI699) at Week 24	6.51 ng/mL Standard Deviation 2.14	—	—	—

Adverse Events

Osilodrostat

Serious events: 4 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Osilodrostat n=9 participants at risk Patients in this arm took the study drug, osilodrostat.
Cardiac disorders Myocardial infarction	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Endocrine disorders Adrenal insufficiency	22.2% 2/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Infections and infestations Pneumonia	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Psychiatric disorders Psychiatric symptom	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment

Other adverse events

Other adverse events
Measure	Osilodrostat n=9 participants at risk Patients in this arm took the study drug, osilodrostat.
Blood and lymphatic system disorders Iron deficiency anaemia	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Ear and labyrinth disorders Vertigo	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Endocrine disorders Adrenal insufficiency	55.6% 5/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Endocrine disorders Steroid withdrawal syndrome	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Gastrointestinal disorders Abdominal distension	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Gastrointestinal disorders Abdominal pain upper	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Gastrointestinal disorders Constipation	22.2% 2/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Gastrointestinal disorders Dental caries	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Gastrointestinal disorders Enterocolitis	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Gastrointestinal disorders Stomatitis	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
General disorders Malaise	33.3% 3/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
General disorders Oedema peripheral	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
General disorders Pyrexia	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Hepatobiliary disorders Primary biliary cholangitis	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Infections and infestations Cellulitis	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Infections and infestations Nasopharyngitis	33.3% 3/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Infections and infestations Osteomyelitis	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Injury, poisoning and procedural complications Rib fracture	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Investigations Alanine aminotransferase increased	22.2% 2/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Investigations Amylase increased	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Investigations Aspartate aminotransferase increased	22.2% 2/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Investigations Blood alkaline phosphatase increased	22.2% 2/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Investigations Gamma-glutamyltransferase increased	33.3% 3/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Investigations Weight decreased	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Investigations Weight increased	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Metabolism and nutrition disorders Decreased appetite	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Metabolism and nutrition disorders Dyslipidaemia	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Metabolism and nutrition disorders Hypokalaemia	22.2% 2/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Metabolism and nutrition disorders Hypomagnesaemia	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Metabolism and nutrition disorders Increased appetite	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Musculoskeletal and connective tissue disorders Arthralgia	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Nervous system disorders Dizziness	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Psychiatric disorders Anxiety	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Psychiatric disorders Insomnia	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Psychiatric disorders Mood altered	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Psychiatric disorders Nightmare	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Psychiatric disorders Reactive psychosis	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Skin and subcutaneous tissue disorders Alopecia	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Skin and subcutaneous tissue disorders Dermal cyst	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Skin and subcutaneous tissue disorders Dermatitis acneiform	22.2% 2/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Skin and subcutaneous tissue disorders Pruritus	22.2% 2/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Skin and subcutaneous tissue disorders Rash	22.2% 2/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
Vascular disorders Peripheral coldness	11.1% 1/9 • Adverse events (AEs) were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 68.0 weeks. AE description; Any sign or symptom that occurs during the study treatment plus the 30 days post treatment

Additional Information

Study Director

Novartis PharmaCeuticals

Phone: 862-778-8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of pooled data (i.e.,data from all sites) in clinical trial or disclosure of trial results in their entirety
Publication restrictions are in place

Restriction type: OTHER