Diagnosis of Central Adrenal Insufficiency in Patients With Prader-Willi Syndrome
Primary Purpose
Prader Willi Syndrome, Adrenal Insufficiency
Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Low dose (1 mcg) ACTH stimulation test
Overnight metyrapone test
Sponsored by
About this trial
This is an interventional diagnostic trial for Prader Willi Syndrome focused on measuring Metyrapone, ACTH stimulation test
Eligibility Criteria
Inclusion Criteria:
- Individuals with Prader Willi Syndrome ages 2 years and older
Exclusion Criteria:
- Individuals who are ill at the time of study
- Individuals who are pregnant at the time of study
- Individuals who it is deemed unsafe to stop taking medications known to affect the results of they study (hydrocortisone, phenytoin, estrogen, acetaminophen, oral anti-diabetic agents).
Sites / Locations
- Nationwide Children's Hospital
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Assessment of Central Adrenal Insufficiency
Arm Description
Patients will undergo low dose ACTH stimulation test followed by overnight metyrapone test to assess for central adrenal insufficiency.
Outcomes
Primary Outcome Measures
Presence of central adrenal insufficiency using low dose (1 mcg) ACTH stimulation test
Prescence of central adrenal insufficiency using overnight metyrapone test
Secondary Outcome Measures
Determine peak cortisol value on low dose (1 mcg) ACTH stimulation test that can accurately predict central adrenal insufficiency based on overnight metyrapone test with good sensitivity and specificity.
Full Information
NCT ID
NCT02368379
First Posted
February 5, 2015
Last Updated
January 25, 2018
Sponsor
Nationwide Children's Hospital
1. Study Identification
Unique Protocol Identification Number
NCT02368379
Brief Title
Diagnosis of Central Adrenal Insufficiency in Patients With Prader-Willi Syndrome
Official Title
Diagnosis of Central Adrenal Insufficiency in Patients With Prader-Willi Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
January 2018
Overall Recruitment Status
Completed
Study Start Date
March 2014 (undefined)
Primary Completion Date
August 2016 (Actual)
Study Completion Date
August 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Nationwide Children's Hospital
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to determine presence of central adrenal insufficiency in children with Prader Willi Syndrome using low dose (1 mcg) ACTH stimulation test compared to results of overnight metyrapone test.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader Willi Syndrome, Adrenal Insufficiency
Keywords
Metyrapone, ACTH stimulation test
7. Study Design
Primary Purpose
Diagnostic
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
23 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Assessment of Central Adrenal Insufficiency
Arm Type
Other
Arm Description
Patients will undergo low dose ACTH stimulation test followed by overnight metyrapone test to assess for central adrenal insufficiency.
Intervention Type
Other
Intervention Name(s)
Low dose (1 mcg) ACTH stimulation test
Intervention Description
Subjects will have baseline cortisol and ACTH drawn followed by administration of 1 mcg/m2 (max 1 mcg) of cortrosyn. Blood will be drawn at 20 and 40 minutes post cortrosyn for peak cortisol assessment of central adrenal insufficiency.
Intervention Type
Other
Intervention Name(s)
Overnight metyrapone test
Intervention Description
Subjects will receive metyrapone 30 mg/kg (max 3 grams) by mouth at midnight. Blood will be drawn at 0800 AM the following morning for ACTH, cortisol and 11 deoxycortisol for assessment of central adrenal insufficiency.
Primary Outcome Measure Information:
Title
Presence of central adrenal insufficiency using low dose (1 mcg) ACTH stimulation test
Time Frame
40 minutes
Title
Prescence of central adrenal insufficiency using overnight metyrapone test
Time Frame
8 hours
Secondary Outcome Measure Information:
Title
Determine peak cortisol value on low dose (1 mcg) ACTH stimulation test that can accurately predict central adrenal insufficiency based on overnight metyrapone test with good sensitivity and specificity.
Time Frame
0
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria:
Individuals with Prader Willi Syndrome ages 2 years and older
Exclusion Criteria:
Individuals who are ill at the time of study
Individuals who are pregnant at the time of study
Individuals who it is deemed unsafe to stop taking medications known to affect the results of they study (hydrocortisone, phenytoin, estrogen, acetaminophen, oral anti-diabetic agents).
Facility Information:
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Diagnosis of Central Adrenal Insufficiency in Patients With Prader-Willi Syndrome
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