RecruitingNCT00001595
Endocrine
Cushing Disease
Also known as ACTH-secreting pituitary adenoma, pituitary Cushing, hypercortisolism, Cushing pituitary
Cushing Disease refers specifically to hypercortisolism caused by an ACTH-secreting pituitary adenoma (corticotroph adenoma), which drives bilateral adrenocortical hyperplasia and excessive cortisol production, distinguishing it from the br
ORPHA:96253 ↗Prevalence 2–3 per million per yearOnset Any age; peak incidence in third to fifth decade; female predominanceSporadic (vast majority); rarely MEN1-associated
37
studies recruiting now
as of 7 Sept 2026
179
studies registered in total
as of 7 Sept 2026
9
countries with a recruiting site
as of 7 Sept 2026
10 Oct 2024
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT03831958
Long-Term Follow-Up of Survivors of Pediatric Cushing Disease
RecruitingPhase 2NCT06635629
Desmopressin Stimulation Test Performance in ACTH-Dependent Cushing Syndrome
RecruitingPhase 2NCT06471829
A Trial of Lu AG13909 in Adult Participants With Cushing's Disease
RecruitingNCT02830308
Evaluation of Adults With Endocrine and Metabolic-Related Conditions
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 37 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Registry: European Registry on Cushing Syndrome (ERCUSYN) · Join ↗. Registries connect patients to researchers and often hear about trials first.
About Cushing Disease
Cushing Disease refers specifically to hypercortisolism caused by an ACTH-secreting pituitary adenoma (corticotroph adenoma), which drives bilateral adrenocortical hyperplasia and excessive cortisol production, distinguishing it from the broader term Cushing syndrome which encompasses all causes of glucocorticoid excess. It accounts for approximately 70% of endogenous Cushing syndrome cases, affects women more often than men by a ratio of roughly 3:1, and is associated with significant morbidity including hypertension, diabetes, osteoporosis, immune suppression, psychiatric disorders, and a markedly elevated cardiovascular mortality if untreated. Transsphenoidal surgery remains first-line treatment, but recurrence rates of 15–40% drive ongoing development of pituitary-targeted and adrenal-targeted pharmacological therapies.
Common clinical features
Central obesity with buffalo hump and supraclavicular fat padsWide purple striae on the abdomen, thighs, and breastsProximal myopathy and muscle weaknessHypertension and glucose intolerance or frank diabetesOsteoporosis with fragility fracturesFacial plethora and moon faceEasy bruising and thin skinPsychiatric manifestations including depression and cognitive impairment
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
2 approved treatments and 11 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Pasireotide (Signifor)Approved: Osilodrostat Phosphate (Isturisa)
Phase 3Osilodrostat
Phase 3Levoketoconazole (Recorlev)
Phase 3Mifepristone (Korlym)
Phase 2Fimepinostat
Phase 2Azd-4017
Phase 2Seliciclib
Phase 2Rosiglitazone Maleate (Avandia)
Phase 2Vorinostat (Zolinza)
+ 3 more in development
Before you apply
Things trial teams commonly ask about for Cushing Disease. Not eligibility rules; those are set by each study.
- Biochemical confirmation of ACTH-dependent hypercortisolism (elevated 24-hour urinary free cortisol on two occasions, failed overnight dexamethasone suppression, elevated late-night salivary cortisol) plus pituitary imaging is required before trial enrolment.
- Prior pituitary surgery or radiotherapy history must be disclosed; trials of novel pituitary-directed agents typically enrol patients with persistent or recurrent disease after transsphenoidal surgery.
- Glucose control, blood pressure, and bone density status at baseline are used for safety stratification; bring recent HbA1c, blood pressure records, and DXA scan results to screening appointments.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).