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

ConditionsEndogenous Cushing SyndromeAdrenal InsufficiencyHypercortisolism
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-75
SponsorUniversity of Michigan

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial population

Patients receiving care at the University of Michigan.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • Osilodrostat

    Drug

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

Treatment groups

12 Participants
are divided into 1 treatment group
Group A: Observational cohort1 intervention

Trial outcomes

Primary outcomes

1

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

based on evidence of hypotension (systolic BP \<90 mmHg) and/or hypoglycemia (glucose \<45 mg/dL) with antecedent symptoms (examples: anorexia, nausea, abdominal pain, orthostasis) and with a resolution of signs upon receiving rescue glucocorticoid therapy

Time frame
Through phase 1, approximately 24 weeks

Secondary outcomes

1

Correlation between AM cortisol and 11OHA4 measurements

compare biomarkers measured by mass spectrometry. This may be calculated Upon 12 participants completing phase 1 or at the end of the study, whichever comes first.

Time frame
Up to end of study, approximately 48 weeks
2

Rate of adrenal insufficient episodes per patient per year

based on evidence of hypotension (systolic BP \<90 mmHg) and/or hypoglycemia (glucose \<45 mg/dL) with antecedent symptoms (examples: anorexia, nausea, abdominal pain, orthostasis) and with a resolution of signs upon receiving rescue glucocorticoid therapy

Time frame
Up to end of phase 2 (approximately 48 weeks)
3

Frequency of cortisol withdrawal symptoms

Based on patient-reported outcomes

Time frame
Up to end of phase 2 (approximately 48 weeks)
4

Change in weight

Based on clinic measurements

Time frame
Baseline, end of phase 2 (approximately 48 weeks)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University of Michigan

Lead sponsor

RECORDATI GROUP

Collaborator

Source ClinicalTrials.gov
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