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Cushing syndrome - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

Cushing syndrome

MONDO:0018912

Also known as:hypercortisolismCushing's syndromecortisol Excess

Find Clinical TrialsTrials

Overview

Cushing's syndrome (CS) encompasses a group of hormonal disorders caused by prolonged and high exposure levels to glucocorticoids that can be of either endogenous (adrenal cortex production) or exogenous (iatrogenic) origin.

TreatmentsNo approved treatments · 2 in pipeline

Clinical Trials42 registered · 24 recruiting

Patient Orgs1 organizations · National Adrenal Diseases Foundation (NADF)

Diagnosis

Biomarker and diagnostic research for Cushing syndrome has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for Cushing syndrome. An additional 2 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for Cushing syndrome, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for Cushing syndrome. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

42 clinical trials registered, 24 recruiting. Interventions under study include other interventions, drug therapy, procedural interventions, and medical devices. Pipeline includes 2 PHASE4, 6 PHASE2, 1 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT06443268](https://clinicaltrials.gov/study/NCT06443268)

Data assembled from 5 of 12 sources · Last updated Sep 20, 2026, 2:11 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Patient Support

Patient Advocacy Groups

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National Adrenal Diseases Foundation (NADF)

USWebsite ↗

Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.

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Medical References

External Resources

Orphanet

European rare disease database

Orphanet:553

GARD

Genetic and Rare Diseases Info Center

GARD:6224

Questions for Your Care Team

→Are any of the 42 active clinical trials a good fit for my situation?
→What is the current development status of N-[2,6-bis(1-methylethyl)phenyl]-N'-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt, and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→How can I connect with National Adrenal Diseases Foundation (NADF) and other patient organizations for support?

← Back to All Diseases

Research Type	Count	% of Total
Research summaries	58	47%
Disease patterns and progression	19	15%
Testing and diagnosis research	11	9%
Patient case studies	11	9%
Clinical study results	10	8%
Laboratory research	8	7%
Other research	5	4%
New treatment approaches	1	1%

Kisho

Cushing syndrome

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Patient Support

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Ectopic ACTH-dependent Cushing syndrome in 3 hospitalized patients: lessons learned from management with osilodrostat.

Severe Ectopic ACTH-Dependent Cushing Syndrome Due to a Neuroendocrine Tumor of Occult Primary Origin Presenting with Widespread Metastases and Early Vertebral Fractures: A Rare Case Report.

Cyclic Cushing syndrome as a clinical enigma and diagnostic dilemma in advanced endocrinology.

Cushing syndrome

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Patient Support

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Ectopic ACTH-dependent Cushing syndrome in 3 hospitalized patients: lessons learned from management with osilodrostat.

Severe Ectopic ACTH-Dependent Cushing Syndrome Due to a Neuroendocrine Tumor of Occult Primary Origin Presenting with Widespread Metastases and Early Vertebral Fractures: A Rare Case Report.

Cyclic Cushing syndrome as a clinical enigma and diagnostic dilemma in advanced endocrinology.