Clinical trial

A Block-and-Replace Therapy With Osilodrostat and Concomitant Glucocorticoid Replacement

Name

HUM00246263

Description

The major goal of this study is to determine the incidence of adrenal insufficiency in patients with endogenous Cushing syndrome receiving osilodrostat treatment combined with a replacement of glucocorticoid (block-and-replace approach). The investigators are also evaluating new biomarker steroids to reflect adequate osilodrostat dosing, the durability and safety, and clinical improvement during treatment.

Trial arms

Trial start

2024-05-01

Estimated PCD

2027-05-01

Trial end

2027-08-01

Status

Recruiting

Treatment

Osilodrostat

Add methylprednisolone to osilodrostat therapy after first dose and continue during osilodrostat titration.

Arms:

Observational cohort

Other names:

Isturisa

Size

Primary endpoint

Percent of participants who experience an adrenal insufficiency event during Phase 1 (titration phase)

Through phase 1, approximately 24 weeks

Eligibility criteria

Inclusion Criteria: * Endogenous Cushing syndrome, either following surgery or not candidates for surgery * Under consideration to receive osilodrostat as part of their clinical care * Able to provide informed consent. Exclusion Criteria: * Treatment with other investigational drugs within 30 days or five half-lives (whichever is longer). * A history of hypersensitivity to osilodrostat or therapies of a similar chemical class.

Protocol

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'PROSPECTIVE'}, 'bioSpec': {'retention': 'SAMPLES_WITHOUT_DNA', 'description': 'Serum samples for steroid measurements.'}, 'enrollmentInfo': {'count': 12, 'type': 'ESTIMATED'}}

Updated at

2024-05-28

1 organization

1 product

3 indications

Organization

University of Michigan

Product

Osilodrostat

Indication

Cushing syndrome

Indication

Adrenal Insufficiency

Indication

Cushing's syndrome