Actively Recruiting
A Block-and-Replace Therapy With Osilodrostat and Concomitant Glucocorticoid Replacement
Led by University of Michigan · Updated on 2025-08-20
12
Participants Needed
1
Research Sites
13 weeks
Total Duration
On this page
Sponsors
U
University of Michigan
Lead Sponsor
R
RECORDATI GROUP
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying patients with endogenous Cushing syndrome to understand how often adrenal insufficiency occurs when treated with osilodrostat combined with glucocorticoid replacement. The study also examines new steroid biomarkers to monitor osilodrostat dosing, and assesses treatment durability, safety, and clinical improvement. This observational study aims to provide important insights into managing this condition with a block-and-replace approach. The study has two phases. In Phase 1 (Titration), participants start with a low dose of osilodrostat and add methylprednisolone replacement. Doses are adjusted to reach specific cortisol level goals, with frequent monitoring and communication. Once these goals are met, participants enter Phase 2 (Maintenance), where they continue treatment for 48 weeks with periodic cortisol tests to track treatment effects. Participants will regularly provide blood and saliva samples for cortisol and steroid profiling, and report symptoms related to adrenal insufficiency. Researchers will track clinical measures such as blood pressure, weight, and quality of life over about 48 weeks. The main outcome is the percentage of participants experiencing adrenal insufficiency during Phase 1, along with various secondary health and hormone measures throughout the study.
CONDITIONS
Brief Title
A Block-and-Replace Therapy With Osilodrostat and Concomitant Glucocorticoid Replacement
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of endogenous Cushing syndrome, either after surgery or if not eligible for surgery
- Considering osilodrostat treatment as part of clinical care
- Able to give informed consent
- Age between 18 and 75 years
You will not qualify if you...
- Use of other investigational drugs within 30 days or five half-lives, whichever is longer
- History of allergy or hypersensitivity to osilodrostat or similar treatments
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 24 weeks
Participants receive block-and-replace therapy starting with osilodrostat dosing, combined with methylprednisolone. Frequent communication occurs at least twice weekly for the first 3 months and weekly thereafter during osilodrostat dose titration to reach cortisol targets.
Twice weekly visits or contacts for first 3 months, then weekly visits or contacts until titration is complete
Duration - Up to 48 weeks
Once maintenance doses are reached, participants are followed for ongoing cortisol monitoring and clinical assessments for up to 48 weeks from first osilodrostat dose.
Visits every 3 to 6 months during maintenance
Trial Site Locations
Total: 1 location
1
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
Research Team
R
Richard Auchus
T
Thaira Blanco Varela
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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