Study of Efficacy and Safety of Osilodrostat in Cushing's Syndrome
A Phase II, Open-label, Dose Titration, Multi-center Study to Assess the Safety/Tolerability and Efficacy of Osilodrostat in Patients With All Types of Endogenous Cushing's Syndrome Except Cushing's Disease
1 other identifier
interventional
9
1 country
4
Brief Summary
The study aim was to investigate the efficacy and safety of Osilodrostat in patients with Cushing's syndrome due to causes other than Cushing's disease in Japan.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Sep 2015
Typical duration for phase_2
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 8, 2015
CompletedFirst Posted
Study publicly available on registry
June 10, 2015
CompletedStudy Start
First participant enrolled
September 24, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 7, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
October 29, 2018
CompletedResults Posted
Study results publicly available
March 11, 2020
CompletedMay 6, 2020
April 1, 2020
2.7 years
June 8, 2015
October 25, 2019
April 23, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percent Change in the Mean Urine Free Cortisol (mUFC) at the Individual Level at Week 12
Percent change from baseline in the mUFC at the individual patient level
Baseline, 12 weeks
Secondary Outcomes (36)
Percent Change From Baseline in the mUFC at Individual Patient Level at Week 24 (Day 169) and Week 48 (Day 337)
Baseline, Week 24 (day 169) and Week 48 (day 337)
Absolute Change From Baseline in the mUFC at Week 12 (Day 85), Week 24 (Day 169) and Week 48 (Day 337)
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)
Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
Percentage of Participants With mUFC Response of Complete, Partial, and Overall Response
12, 24 and 48 weeks
Absolute Change From Baseline in Morning Serum Cortisol at Individual Level
Baseline, Week 12 (day 85), Week 24 (day 169) and Week 48 (day 337)
- +31 more secondary outcomes
Study Arms (1)
Osilodrostat
EXPERIMENTALPatients in this arm took the study drug, osilodrostat.
Interventions
Osirodrostat 1mg, 5mg \& 10mg in the form of film-coated tablets was used for oral administration.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (4)
Novartis Investigative Site
Fukushima, Fukushima, 960 1295, Japan
Novartis Investigative Site
Yokohama, Kanagawa, 245-8575, Japan
Novartis Investigative Site
Sendai, Miyagi, 980 8574, Japan
Novartis Investigative Site
Chiba, 260 8677, Japan
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Novartis PharmaCeuticals
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 8, 2015
First Posted
June 10, 2015
Study Start
September 24, 2015
Primary Completion
June 7, 2018
Study Completion
October 29, 2018
Last Updated
May 6, 2020
Results First Posted
March 11, 2020
Record last verified: 2020-04
Data Sharing
- IPD Sharing
- Will share
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com