Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)

ConditionMild Autonomous Cortisol Secretion (MACS)

Trial statusRecruiting

Trial phasePhase 4

Trial typeInterventional

Biological sexAll

Age18+

SponsorJohns Hopkins University

About this trial

To characterize the impact of Isturisa on clinical features and comorbidities associated with MACS. The investigators hypothesize that patients treated with Isturisa will exhibit significantly better metabolic indicators (such as fasting glucose, HbA1c, and lipid profile), blood pressure, weight, body composition and bone mineral density than at Baseline. The investigators also assess the effect of Isturisa on quality of life and psychological symptoms in patients with MACS. The investigators hypothesize that treatment with Isturisa will lead to significant improvements in quality-of-life scores and reductions in depression scores compared to Baseline.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Adults aged ≥18 years.

Serum cortisol >1.8 µg/dL after 1 mg overnight dexamethasone suppression test (DST).

Presence of adrenal adenoma confirmed by imaging (CT or MRI).

Ability to provide informed consent.

Disqualifiers

Known diagnosis of Cushing's syndrome or overt hypercortisolism.

Current or recent (within 3 months) treatment with glucocorticoids or medications affecting cortisol production (e.g., ketoconazole, metyrapone).

Severe hepatic impairment or renal failure.

Pregnancy or breastfeeding.

Trial design

Design model

Single group

Treatments tested in this trial

Osilodrostat (Isturisa)
Drug
The intervention aims to evaluate the impact of osilodrostat on cardiometabolic outcomes, bone mineral density, body composition, adrenal tumor size or hyperplasia, and biochemical markers of cortisol excess.

Treatment groups

10 Participants

are divided into 1 treatment group

Group A: Isturisa Treatment ArmExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

Change in fasting glucose, HbA1c

glycemic control: fasting glucose, HbA1c

Time frame

Baseline, and then every 3 months up to 2 years

Change in bone mineral density (g/cm²)

Bone mineral density (g/cm²) measured by DEXA scan.

Time frame

Baseline, 1 year, and 2 years

Change in body weight (kg) from baseline during Isturisa treatment

Change in body weight will be measured at baseline and every follow-up visit (approximately every 3 months) throughout the 2-year treatment period with Isturisa.

Time frame

Baseline and every 3 months up to 2 years

Change in lipid profile from baseline during Isturisa treatment

Serum lipid profile (LDL-C, HDL-C, total cholesterol, triglycerides) will be assessed at baseline and every follow-up visit (approximately every 3 months) during the 2-year treatment period.

Time frame

Baseline and every 3 months up to 2 years

Secondary outcomes

Change in Quality of Life as assessed by Short Form -36 (SF-36)

Assessment of changes in quality of life using the SF-36 Health Survey. The SF-36 consists of 36 items measuring 8 health domains, with total scores ranging from 0 to 100, where higher scores indicate better quality of life.

Time frame

Baseline and every 3 months up to 2 years

Change in carotid intima-media thickness (CIMT)

Carotid intima-media thickness (milimeters) will be measured using carotid ultrasound to evaluate vascular changes associated with mild autonomous cortisol secretion. CIMT will be assessed at baseline and at follow-up imaging performed approximately every 12 months during the 2-year treatment period.

Time frame

Baseline, 1 year, and 2 years

Change in adrenal adenoma/hyperplasia size (mm)

Adenoma maximal diameter (mm) measured on non-contrast CT at each imaging time point.

Time frame

Baseline, 1 year, and 2 years

Change in Depression Scores as assessed by the Beck Depression Inventory-II

Assessment of changes in depressive symptoms using the Beck Depression Inventory-II (BDI-II). The BDI-II is a 21-item validated questionnaire with total scores ranging from 0 to 63, where higher scores indicate more severe depressive symptoms.

Time frame

Baseline and every 3 months up to 2 years

Other outcomes

Change in Inflammatory Biomarkers

Exploratory evaluation of changes in inflammatory biomarkers, specifically high-sensitivity C-reactive protein (hsCRP) and interleukin-6 (IL-6), during treatment with Isturisa in patients with mild autonomous cortisol secretion (MACS). These markers are included in the optional research-specific procedures to assess potential inflammatory or physiologic effects of Isturisa beyond cortisol regulation.

Time frame

Baseline and every 3 months up to 2 years

Sponsors and contacts

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

Johns Hopkins University

Lead sponsor

Recordati Rare Diseases Inc

Collaborator

Source ClinicalTrials.gov

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