Rare Cardiology News
Advertisement
Spotlight On
Cushing's syndrome
Cushing's syndrome is the collection of signs and symptoms due to prolonged exposure to glucocorticoids such as cortisol
Prevalence
1-9 / 100 000
Age of Onset
ICD-10
E24.0
E24.1
E24.2
E24.3
E24.4
E24.8
E24.9
Inheritance
Autosomal dominant
Autosomal recessive
Mitochondrial/Multigenic
X-linked dominant
X-linked recessive
Rare View
Cushing's syndrome is a collection of signs and symptoms due to prolonged exposure to glucocorticoids such as cortisol. Signs and symptoms may include high blood pressure, abdominal obesity but with thin arms and legs, reddish stretch marks, a round red face, a fat lump between the shoulders, weak muscles, weak bones, acne, and fragile skin that heals poorly.†
5 Facts you should know
FACT
Symptoms include rapid weight gain, particularly of the trunk and face with sparing of the limbs (central obesity)
FACT
Common signs include the growth of fat pads along the collarbone, on the back of the neck ("buffalo hump" or lipodystrophy), and on the face ("moon face")
FACT
It most commonly affects people who are 20 to 50 years of age
FACT
Women are affected three times more often than men
FACT
Affected women may also experience irregular menstrual periods and increased growth of body and facial hair
Interest over time
Google searches
Common signs & symptoms
Abnormal subcutaneous fat tissue distribution
Abnormal fat tissue distribution below the skin
Erectile dysfunction
Erectile abnormalities
Growth delay
Growth deficiency
Round face
Round facial appearance
Bruising susceptibility
Thin skin
Truncal obesity
Acne
Current treatments
The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition. Learn more orphan products.
Mifepristone
(Brand name: Korlym) Manufactured by Corcept Therapeutics, Inc.
FDA-approved indication: Korlym is approved for use in patients with endogenous Cushing's syndrome who have type 2 diabetes or glucose intolerance and are not candidates for surgery or have not responded to prior surgery.
Pasireotide
(Brand name: Signifor) Manufactured by Novartis Pharmaceuticals Corporation
FDA-approved indication: June 2018, approved for the treatment of patients with Cushing’s disease for whom pituitary surgery is not an option or has not been curative. Also, December 2014, approved for the treatment of patients with acromegaly who have had an inadequate response to surgery and/or for whom surgery is not an option.
Top Clinical Trials
Title | Description | Phases | Status | Interventions | More Information |
---|---|---|---|---|---|
Roll-over Study in Patients With Endogenous Cushing's Syndrome for LCI699 | The purpose of this study is the evaluation of long-term safety of osilodrostat in patients who have already received osilodrostat treatment in a previous Global Novartis-sponsored trial and who, based on investigators' judgement, will continue benefiting with its administration. | Phase 2 | Recruiting | Drug: osilodrostat | More Info |
Extension Study to Evaluate the Safety of Long-Term Use of Relacorilant in Patients With Cushing Syndrome | This study is designed to allow continued therapy with relacorilant, a potent, selective glucocorticoid receptor (GR) antagonist in patients who have successfully completed participation of a Corcept-sponsored study of relacorilant (referred to as the "parent" study) | Phase 2 | Enrolling by invitation | Drug: relacorilant | More Info |
A Study of the Efficacy and Safety of Relacorilant in Patients With Endogenous Cushing Syndrome | This is a Phase 3, double-blind, placebo-controlled, randomized-withdrawal study to assess the efficacy, safety and pharmacokinetics (PK) of relacorilant in patients with endogenous Cushing syndrome and concurrent type 2 diabetes mellitus/impaired glucose tolerance and/or uncontrolled hypertensio | Phase 3 | Recruiting | Drug: Relacorilant|Other: Placebo | More Info |
Open-label Treatment in Cushing's Syndrome | This is a long-term, open-label extension study of levoketoconazole in subjects with endogenous Cushing's Syndrome. | Phase 3 | Active, not recruiting | Drug: Levoketoconazole | More Info |
Efficacy and Safety of Relacorilant in Patients With Cortisol-Secreting Adrenal Adenomas | This is a Phase 3, randomized, double-blind, placebo-controlled study to assess the efficacy, and safety of relacorilant to treat hypercortisolism in patients with cortisol-secreting adrenal adenoma or hyperplasia associated with diabetes mellitus/ impaired glucose tolerance and/or uncontrolled systolic hypertension. | Phase 3 | Recruiting | Drug: relacorilant|Other: Placebo | More Info |
Top Treatments in Research
Agent | Class/Mechanism of Action | Development Status | Company | Clinical Studies | More Information |
---|---|---|---|---|---|
Osilodrostat | A cortisol synthesis inhibitor indicated for the treatment of adult patients with Cushing’s disease | Phase 2 | Novartis Pharmaceuticals | More Info | More Info |
Relacorilant | Relacorilant is an orally available antagonist of the glucocorticoid receptor (GR). | Phase 3 | Corcept Therapeutics | More Info | More Info |
† Genetic and Rare Diseases Information Center (GARD) - PO Box 8126, Gaithersburg, MD 20898-8126 https://rarediseases.info.nih.gov