Glucocorticoid Receptor Blockade With Mifepristone in Patients With Mild Adrenal Hypercortisolism
Primary Purpose
Mild Hypercortisolism
Status
Completed
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Mifepristone
Sponsored by
About this trial
This is an interventional treatment trial for Mild Hypercortisolism focused on measuring mild hypercortisolism, Subclinical Cushing's Syndrome, Preclinical Cushing's Syndrome
Eligibility Criteria
Inclusion Criteria:
- >18 years of age
- Incidentally noted adrenal nodule <4 cm with benign imaging characteristics
- Evidence of mild hypercortisolism
- Evidence of diabetes or abnormal glucose tolerance
Exclusion Criteria:
- contraindication to mifepristone
- Indication for unilateral adrenalectomy
- Evidence of other adrenal hormone hypersecretion
- lactating mothers
- women of childbearing age unwilling to use an effective, nonhormonal form of contraception
Sites / Locations
- Icahn School of Medicine at Mount Sinai
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Mifepristone
Arm Description
Mifepristone 300mg tablets taken once daily with dose increase of no more than 300mg once monthly and to a maximum dose of 1200mg daily as indicated by symptom response
Outcomes
Primary Outcome Measures
A1C Level
Change in hyperglycemia assessed by HbA1c, also known as glycated hemoglobin
HOMA-IR
Change in hyperglycemia assessed by Homeostatic Model Assessment of Insulin Resistance, HOMA-IR (a validated assessment of insulin resistance). HOMA-IR = fasting insulin (microU/L) x fasting glucose (nmol/L)/22.5.
Secondary Outcome Measures
Waist Circumference
Change in metabolic syndrome as assessed by waist circumference
Body Mass Index (BMI)
Change in metabolic syndrome as assessed by BMI
Fasting Lipid Profile
Change in metabolic syndrome as assessed by fasting lipid profile which includes Low-density lipoproteins ( LDL), High-density lipoproteins (HDL), and Triglycerides (Trigs) levels, and total cholesterol which is the sum of HDL plus LDL and 20% of trigs.
Weight
Change in metabolic syndrome as assessed by weight
CushingQoL
Change in Quality of Life - as assessed by the Cushing's Quality of Life questionnaire (CushingQoL). Patient completed questionnaire, 12 items, each scored on a 5 point score, resulting in a score of 12 (worst) to 60 (best) where higher scores indicate more favorable QOL.
Nottingham Health Profile (NHP)
Change in Quality of Life as assessed by the Nottingham Health Profile (NHP) which is a patient reported questionnaire to measure a patient's view of their own health status. There are 6 sections (Energy level, Pain, Emotional Reaction, Sleep, Social Isolation, and Physical Abilities. All questions have only yes/no answer options and each section score is weighted so that the possible score range for any section is 0-100. The higher the score, the greater the number and severity of problems.
Hospital Anxiety and Depression Scale (HADS)
Change in Quality of Life as assessed by the Hospital Anxiety and Depression Scale (HADS). Questionnaire with 7 items for anxiety and 7 items for depression, each item is scored on a 4 point response 0 - 3, with full range from 0 to 42, with higher score indicating more severe anxiety or depression
Quality of Life
Change in Quality of Life as assessed by the Beck Depression Inventory. a 21-question multiple choice, self-report inventory that is used for measuring the severity of anxiety. Scoring is from a 0 (not at all) to 3 (severe) with a total score range of 0-63. Higher total scores indicate more severe anxiety symptoms.
State Trait Anxiety Inventory (STAI)
Change in Quality of Life - as assessed by the State Trait Anxiety Inventory (STAI). The State-Trait Anxiety Inventory both state and trait anxiety separately. Each type of anxiety has its own scale of 20 different questions that are scored and averaged. Total scores range from 20 to 80, with higher scores correlating with greater anxiety.
Full Information
NCT ID
NCT01990560
First Posted
November 15, 2013
Last Updated
January 25, 2018
Sponsor
Icahn School of Medicine at Mount Sinai
1. Study Identification
Unique Protocol Identification Number
NCT01990560
Brief Title
Glucocorticoid Receptor Blockade With Mifepristone in Patients With Mild Adrenal Hypercortisolism
Official Title
Glucocorticoid Receptor Blockade With Mifepristone in Patients With Mild Adrenal Hypercortisolism
Study Type
Interventional
2. Study Status
Record Verification Date
January 2018
Overall Recruitment Status
Completed
Study Start Date
November 2013 (undefined)
Primary Completion Date
September 2016 (Actual)
Study Completion Date
September 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Icahn School of Medicine at Mount Sinai
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to determine whether mifepristone is an effective treatment for hyperglycemia due to mild hypercortisolism.
To test the hypothesis that GR blockade with mifepristone will decrease the severity of metabolic syndrome features as measured by waist circumference, lipid profile, body mass index, blood pressure and insulin resistance, measured by HOMA-IR score.
To test the hypothesis that GR blockade with mifepristone will improve QoL, depression and anxiety scores, measured by validated assessments, in patients with mild hypercortisolism.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mild Hypercortisolism
Keywords
mild hypercortisolism, Subclinical Cushing's Syndrome, Preclinical Cushing's Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Mifepristone
Arm Type
Experimental
Arm Description
Mifepristone 300mg tablets taken once daily with dose increase of no more than 300mg once monthly and to a maximum dose of 1200mg daily as indicated by symptom response
Intervention Type
Drug
Intervention Name(s)
Mifepristone
Other Intervention Name(s)
Korlym
Intervention Description
All patients in the study will receive daily Mifepristone for 6 months and primary and secondary outcomes will be assessed before and after the 6 month treatment period
Primary Outcome Measure Information:
Title
A1C Level
Description
Change in hyperglycemia assessed by HbA1c, also known as glycated hemoglobin
Time Frame
Baseline, 3 months, and 6 months
Title
HOMA-IR
Description
Change in hyperglycemia assessed by Homeostatic Model Assessment of Insulin Resistance, HOMA-IR (a validated assessment of insulin resistance). HOMA-IR = fasting insulin (microU/L) x fasting glucose (nmol/L)/22.5.
Time Frame
Baseline and 6 months
Secondary Outcome Measure Information:
Title
Waist Circumference
Description
Change in metabolic syndrome as assessed by waist circumference
Time Frame
Baseline and 6 months
Title
Body Mass Index (BMI)
Description
Change in metabolic syndrome as assessed by BMI
Time Frame
Baseline and 6 months
Title
Fasting Lipid Profile
Description
Change in metabolic syndrome as assessed by fasting lipid profile which includes Low-density lipoproteins ( LDL), High-density lipoproteins (HDL), and Triglycerides (Trigs) levels, and total cholesterol which is the sum of HDL plus LDL and 20% of trigs.
Time Frame
Baseline and 6 months
Title
Weight
Description
Change in metabolic syndrome as assessed by weight
Time Frame
Baseline and 6 months
Title
CushingQoL
Description
Change in Quality of Life - as assessed by the Cushing's Quality of Life questionnaire (CushingQoL). Patient completed questionnaire, 12 items, each scored on a 5 point score, resulting in a score of 12 (worst) to 60 (best) where higher scores indicate more favorable QOL.
Time Frame
Baseline and 6 months
Title
Nottingham Health Profile (NHP)
Description
Change in Quality of Life as assessed by the Nottingham Health Profile (NHP) which is a patient reported questionnaire to measure a patient's view of their own health status. There are 6 sections (Energy level, Pain, Emotional Reaction, Sleep, Social Isolation, and Physical Abilities. All questions have only yes/no answer options and each section score is weighted so that the possible score range for any section is 0-100. The higher the score, the greater the number and severity of problems.
Time Frame
Baseline and 6 months
Title
Hospital Anxiety and Depression Scale (HADS)
Description
Change in Quality of Life as assessed by the Hospital Anxiety and Depression Scale (HADS). Questionnaire with 7 items for anxiety and 7 items for depression, each item is scored on a 4 point response 0 - 3, with full range from 0 to 42, with higher score indicating more severe anxiety or depression
Time Frame
Baseline and 6 months
Title
Quality of Life
Description
Change in Quality of Life as assessed by the Beck Depression Inventory. a 21-question multiple choice, self-report inventory that is used for measuring the severity of anxiety. Scoring is from a 0 (not at all) to 3 (severe) with a total score range of 0-63. Higher total scores indicate more severe anxiety symptoms.
Time Frame
Baseline and 6 months
Title
State Trait Anxiety Inventory (STAI)
Description
Change in Quality of Life - as assessed by the State Trait Anxiety Inventory (STAI). The State-Trait Anxiety Inventory both state and trait anxiety separately. Each type of anxiety has its own scale of 20 different questions that are scored and averaged. Total scores range from 20 to 80, with higher scores correlating with greater anxiety.
Time Frame
Baseline and 6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
>18 years of age
Incidentally noted adrenal nodule <4 cm with benign imaging characteristics
Evidence of mild hypercortisolism
Evidence of diabetes or abnormal glucose tolerance
Exclusion Criteria:
contraindication to mifepristone
Indication for unilateral adrenalectomy
Evidence of other adrenal hormone hypersecretion
lactating mothers
women of childbearing age unwilling to use an effective, nonhormonal form of contraception
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alice C Levine, MD
Organizational Affiliation
Icahn School of Medicine at Mount Sinai
Official's Role
Principal Investigator
Facility Information:
Facility Name
Icahn School of Medicine at Mount Sinai
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
10732270
Citation
Young WF Jr. Management approaches to adrenal incidentalomas. A view from Rochester, Minnesota. Endocrinol Metab Clin North Am. 2000 Mar;29(1):159-85, x. doi: 10.1016/s0889-8529(05)70122-5.
Results Reference
background
PubMed Identifier
15082524
Citation
Mansmann G, Lau J, Balk E, Rothberg M, Miyachi Y, Bornstein SR. The clinically inapparent adrenal mass: update in diagnosis and management. Endocr Rev. 2004 Apr;25(2):309-40. doi: 10.1210/er.2002-0031.
Results Reference
background
PubMed Identifier
12414841
Citation
Tauchmanova L, Rossi R, Biondi B, Pulcrano M, Nuzzo V, Palmieri EA, Fazio S, Lombardi G. Patients with subclinical Cushing's syndrome due to adrenal adenoma have increased cardiovascular risk. J Clin Endocrinol Metab. 2002 Nov;87(11):4872-8. doi: 10.1210/jc.2001-011766.
Results Reference
background
PubMed Identifier
11889151
Citation
Terzolo M, Pia A, Ali A, Osella G, Reimondo G, Bovio S, Daffara F, Procopio M, Paccotti P, Borretta G, Angeli A. Adrenal incidentaloma: a new cause of the metabolic syndrome? J Clin Endocrinol Metab. 2002 Mar;87(3):998-1003. doi: 10.1210/jcem.87.3.8277.
Results Reference
background
PubMed Identifier
11701696
Citation
Garrapa GG, Pantanetti P, Arnaldi G, Mantero F, Faloia E. Body composition and metabolic features in women with adrenal incidentaloma or Cushing's syndrome. J Clin Endocrinol Metab. 2001 Nov;86(11):5301-6. doi: 10.1210/jcem.86.11.8059.
Results Reference
background
PubMed Identifier
23345100
Citation
Feelders RA, Hofland LJ. Medical treatment of Cushing's disease. J Clin Endocrinol Metab. 2013 Feb;98(2):425-38. doi: 10.1210/jc.2012-3126. Epub 2013 Jan 23.
Results Reference
background
PubMed Identifier
23393167
Citation
Lambert JK, Goldberg L, Fayngold S, Kostadinov J, Post KD, Geer EB. Predictors of mortality and long-term outcomes in treated Cushing's disease: a study of 346 patients. J Clin Endocrinol Metab. 2013 Mar;98(3):1022-30. doi: 10.1210/jc.2012-2893. Epub 2013 Feb 7.
Results Reference
background
PubMed Identifier
23559084
Citation
Neary NM, Booker OJ, Abel BS, Matta JR, Muldoon N, Sinaii N, Pettigrew RI, Nieman LK, Gharib AM. Hypercortisolism is associated with increased coronary arterial atherosclerosis: analysis of noninvasive coronary angiography using multidetector computerized tomography. J Clin Endocrinol Metab. 2013 May;98(5):2045-52. doi: 10.1210/jc.2012-3754. Epub 2013 Apr 4.
Results Reference
background
PubMed Identifier
23577182
Citation
Debono M, Chadarevian R, Eastell R, Ross RJ, Newell-Price J. Mifepristone reduces insulin resistance in patient volunteers with adrenal incidentalomas that secrete low levels of cortisol: a pilot study. PLoS One. 2013;8(4):e60984. doi: 10.1371/journal.pone.0060984. Epub 2013 Apr 5.
Results Reference
background
PubMed Identifier
18334580
Citation
Nieman LK, Biller BM, Findling JW, Newell-Price J, Savage MO, Stewart PM, Montori VM. The diagnosis of Cushing's syndrome: an Endocrine Society Clinical Practice Guideline. J Clin Endocrinol Metab. 2008 May;93(5):1526-40. doi: 10.1210/jc.2008-0125. Epub 2008 Mar 11.
Results Reference
background
PubMed Identifier
10702749
Citation
Flint A, Raben A, Blundell JE, Astrup A. Reproducibility, power and validity of visual analogue scales in assessment of appetite sensations in single test meal studies. Int J Obes Relat Metab Disord. 2000 Jan;24(1):38-48. doi: 10.1038/sj.ijo.0801083.
Results Reference
background
PubMed Identifier
14749739
Citation
Parker BA, Sturm K, MacIntosh CG, Feinle C, Horowitz M, Chapman IM. Relation between food intake and visual analogue scale ratings of appetite and other sensations in healthy older and young subjects. Eur J Clin Nutr. 2004 Feb;58(2):212-8. doi: 10.1038/sj.ejcn.1601768.
Results Reference
background
Learn more about this trial
Glucocorticoid Receptor Blockade With Mifepristone in Patients With Mild Adrenal Hypercortisolism
We'll reach out to this number within 24 hrs