OBSOLETE: Cushing syndrome

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6Active trials8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Cushing syndrome (also called hypercortisolism) is a condition where the body is exposed to too much of a hormone called cortisol for a long period of time. Cortisol is sometimes called the 'stress hormone' because it helps the body respond to stress, but when there is too much of it, it causes serious problems throughout the body. Cushing syndrome can happen because of a tumor in the pituitary gland (a small gland in the brain), a tumor in the adrenal glands (which sit on top of the kidneys), a tumor elsewhere in the body that makes a cortisol-triggering hormone, or from taking steroid medications like prednisone for a long time. When caused by a pituitary tumor, it is sometimes called Cushing's disease specifically. The condition affects many parts of the body at once. People with Cushing syndrome often gain weight, especially around the belly and upper back, and develop a rounded 'moon face.' The skin becomes fragile, bruises easily, and may develop purple or pink stretch marks. Muscles become weak, bones lose strength, blood pressure rises, and blood sugar can increase, sometimes leading to diabetes. Mood changes, depression, and anxiety are also very common. Treatment depends on the cause. Surgery to remove a tumor is often the first choice and can be curative. Radiation therapy and medications that lower cortisol levels — such as metyrapone, ketoconazole, mifepristone (Korlym), osilodrostat (Isturisa), or pasireotide (Signifor) — are also used. With proper treatment, many people improve significantly, though recovery can take months to years. This condition is listed as 'obsolete' in some rare disease registries because it is now classified into more specific subtypes.

Also known as:

Key symptoms:

Weight gain, especially around the belly, chest, and upper backRounded, puffy 'moon face'A fatty hump between the shoulders ('buffalo hump')Purple or pink stretch marks on the skinSkin that bruises easily and heals slowlyMuscle weakness, especially in the thighs and upper armsHigh blood pressureHigh blood sugar or diabetesThinning bones (osteoporosis) and increased risk of fracturesFatigue and low energyDepression, anxiety, or mood swingsIrregular or absent menstrual periods in womenReduced sex driveIncreased body and facial hair in womenFrequent infections due to a weakened immune system

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

7 events
Nov 2025Trial Against INtractable Type 2 Diabetes (CAPTAIN-T2D)

Sparrow Pharmaceuticals — PHASE2

TrialRECRUITING
Sep 2025Salivary Cortisol and Hypercortisolism in Type 2 Diabetes

Shanghai 6th People's Hospital

TrialRECRUITING
Dec 2024Osilodrostat Therapy and 11C-methionine PET to Improve Corticotroph Adenoma Detection

Imperial College London

TrialNOT YET RECRUITING
Jun 2023Diagnostic Value of CXCR4-targeted PET/CT in ACTH-dependent and Independent Cushing's Syndrome

Peking Union Medical College Hospital — PHASE1, PHASE2

TrialRECRUITING
Oct 2022Effects of "Vitamin N" Nature Immersion Therapy on Stress Levels in Health Care Workers in the City of Bogotá

Jeadran N. Malagón-Rojas — NA

TrialACTIVE NOT RECRUITING
Jun 2022Non-interventional Study on Osilodrostat in Patients With Endogenous Cushing's Syndrome

RECORDATI GROUP

TrialACTIVE NOT RECRUITING
Jun 2022Endoscopic Ultrasound-guided Radiofrequency Ablation in Primary Aldosteronism

Haukeland University Hospital — NA

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for OBSOLETE: Cushing syndrome.

6 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

6 recruitingView all trials with filters →
Phase 21 trial
Trial Against INtractable Type 2 Diabetes (CAPTAIN-T2D)
Phase 2
Actively Recruiting
· Sites: Chandler, Arizona; Tucson, Arizona +44 more · Age: 1899 yrs
N/A2 trials
Effects of "Vitamin N" Nature Immersion Therapy on Stress Levels in Health Care Workers in the City of Bogotá
N/A
Active
· Sites: Bogotá · Age: 1862 yrs
Endoscopic Ultrasound-guided Radiofrequency Ablation in Primary Aldosteronism
N/A
Actively Recruiting
PI: Marianne Grytaas, MD phd (Haukeland University Hospital) · Sites: Bergen · Age: 1880 yrs
Other2 trials
Salivary Cortisol and Hypercortisolism in Type 2 Diabetes
Actively Recruiting
· Sites: Shanghai · Age: 1880 yrs
Non-interventional Study on Osilodrostat in Patients With Endogenous Cushing's Syndrome
Active
PI: Mario Maldonado, MD (Recordati AG - Head of Clinical Development) · Sites: Phoenix, Arizona; Atlanta, Georgia +41 more · Age: 1899 yrs

No specialists are currently listed for OBSOLETE: Cushing syndrome.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to OBSOLETE: Cushing syndrome.

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Community

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What is causing my Cushing syndrome — is it a tumor, and where is it located?,What treatment do you recommend for my specific case, and what are the chances it will work?,How long will it take to feel better after treatment, and what symptoms should I expect during recovery?,What are the risks of surgery or other treatments, and what happens if the first treatment doesn't work?,Should I be tested for a genetic condition that might have caused this?,What signs should I watch for that might mean the condition has come back?,Are there support groups or specialists in Cushing syndrome I should connect with?

Common questions about OBSOLETE: Cushing syndrome

What is OBSOLETE: Cushing syndrome?

Cushing syndrome (also called hypercortisolism) is a condition where the body is exposed to too much of a hormone called cortisol for a long period of time. Cortisol is sometimes called the 'stress hormone' because it helps the body respond to stress, but when there is too much of it, it causes serious problems throughout the body. Cushing syndrome can happen because of a tumor in the pituitary gland (a small gland in the brain), a tumor in the adrenal glands (which sit on top of the kidneys), a tumor elsewhere in the body that makes a cortisol-triggering hormone, or from taking steroid medica

Are there clinical trials for OBSOLETE: Cushing syndrome?

Yes — 6 recruiting clinical trials are currently listed for OBSOLETE: Cushing syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.