Skip to content

Trial · Interventional

CompletedPhase 2Results postedRegistry data

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

NCTNCT02468193 (opens ClinicalTrials.gov in a new tab)Other idCLCI699C1201
Open at ClinicalTrials.govAsk about this trial

This trial does not test a peptide that PepBank tracks.

  • Measures ACTH (corticotropin), does not give it. It appears in an outcome measure.

Overview

Status
Completed
Phase
Phase 2
Enrollment
9 participants (actual)
Sponsor
Novartis Pharmaceuticals · Industry
Design
Allocation not applicable · Open label (no masking)
Countries
1 country

Japan

Start
24 Sept 2015
Primary completion
7 Jun 2018
Completion
29 Oct 2018
First posted
10 Jun 2015 (estimated)
Last update posted
6 May 2020
Conditions
Cushing's Syndrome · Ectopic Corticotropin Syndrome · Adrenal Adenoma · Adrenal Carcinoma · AIMAH · PPNAD

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20152016201720182019
  1. Ph 2

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

    NCT02468193

    24 Sept 2015 to 29 Oct 2018

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The study aim was to investigate the efficacy and safety of Osilodrostat in patients with Cushing's syndrome due to causes other than Cushing's disease in Japan.

Interventions

  • OsilodrostatDrug

Arms1

  • Experimental

    Osilodrostat

    Patients in this arm took the study drug, osilodrostat.

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12

    Time frame · Baseline, 12 weeks

    Registry description

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

Secondary outcomes(20)

  1. Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337)

    Time frame · Baseline, Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  2. Absolute Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

    Absolute change from baseline in the mUFC

  3. Percentage Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

    Percent change from baseline in the mUFC

  4. Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response

    Time frame · 12, 24 and 48 weeks

    Registry description

    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.

  5. Absolute Change From Baseline in Morning Serum Cortisol at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  6. Percentage Change From Baseline in Morning Serum Cortisol at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  7. Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  8. Percentage Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  9. Absolute Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  10. Percentage Change From Baseline in Other Adrenal Steroid Hormones at Individual Levels

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  11. Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  12. Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Fasting Glucose, at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  13. Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  14. Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, HbA1c, at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  15. Absolute Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  16. Percentage Change From Baseline in Cardiovascular-related Metabolic Parameters, Cholesterol, HDL Cholesterol, LDL Cholesterol & Triglycerides, at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  17. Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  18. Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, BMI, at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  19. Absolute Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

  20. Percentage Change From Baseline in Cardiovascular-related Metabolic Parameter, Waist Circumference, at Individual Level

    Time frame · Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

    Registry description

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

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeNumber· Baseline, 12 weeks

Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12

GroupValue (percentage change)
Osilodrostat-99.0
Secondary and other outcome results(7)
Secondary outcomeNumber· Baseline, Week 24 (day 169) and Week 48 (day 337)

Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337)

GroupValue (percentage change)
Osilodrostat-99.5
Secondary outcomeMedian· Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

Absolute Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)

GroupValue (nmol/24hr)
Osilodrostat-422.10
Secondary outcomeMedian· Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

Percentage Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)

GroupValue (percentage change)
Osilodrostat-94.47
Secondary outcomeNumber· 12, 24 and 48 weeks

Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response

GroupValue (Percentage of participants)
Osilodrostat66.7
Secondary outcomeNumber· Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

Absolute Change From Baseline in Morning Serum Cortisol at Individual Level

GroupValue (nmol/L)
Osilodrostat-607
Secondary outcomeNumber· Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

Percentage Change From Baseline in Morning Serum Cortisol at Individual Level

GroupValue (percentage change)
Osilodrostat-73.3
Secondary outcomeNumber· Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)

Absolute Change From Baseline in ACTH and Other Adrenal Steroid Hormones at Individual Level

GroupValue (pmol/L)
ACTH0
Serum 11-deoxycorticosterone2179
Aldosterone52
Estradiol705

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
OsilodrostatPatients in this arm took the study drug, osilodrostat.0 / 94 / 99 / 9

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 18 Years up to 85 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * Patients with confirmed Cushing's syndrome \[i.e. ectopic corticotropin syndrome, adrenal adenoma, adrenal carcinoma, ACTH-Independent Macronodular Adrenal Hyperplasia (AIMAH), or Primary Pigmented Nodular Adrenal Dysplasia (PPNAD)\] * For patients on medical treatment for hypercortisolism due to Cushing's syndrome, the washout periods had to be completed prior to baseline efficacy assessments Exclusion Criteria: * Patients with Cushing's disease * History of hypersensitivity to osilodrostat or to drugs of similar chemical classes * History of malignancy of any organ system, treated or untreated, within the past 5 years * Patients receiving treatment for within 4 weeks or ≤5 x half-life of the agent (whichever is longer) before first dose of osilodrostat * Patients with risk factors for QTc prolongation or Torsade de Pointes

PepBank is a research reference. Talk to a doctor before you use any peptide.