Growth Hormone Use in Adults With Prader-Willi Syndrome
Primary Purpose
Prader-Willi Syndrome
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Nutropin AQ
Sponsored by
About this trial
This is an interventional diagnostic trial for Prader-Willi Syndrome
Eligibility Criteria
Inclusion Criteria:
- 16 to 60 years old
- Male or female with diagnosed Prader-Willi syndrome confirmed by genetic testing (e.g., mPCR)
- Low IGF-1 level (e.g.,≤25%) at baseline
- Ability to provide informed consent or availability of a suitable legally authorized representative
Exclusion Criteria:
- Pregnancy
- Previous treatment with growth hormone
- Uncontrolled endocrine disease, (i.e. diabetes or thyroid)
- History of severe scoliosis
- Heart disease
- Uncontrolled high blood pressure or history of stroke
- Morbid obesity (using PWS growth charts)
- Severe sleep apnea or known breathing difficulties/obstruction (as per history or diagnostic testing results
Sites / Locations
- The Children's Mercy Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Primary Cohort
Arm Description
Nutropin AQ
Outcomes
Primary Outcome Measures
Body Composition
Anthropometric Measures and Body Composition
Blood Chemistry
Insulin-like growth factor I- IGF-1
Physical Activity
Duration of daily physical activity
Secondary Outcome Measures
Full Information
NCT ID
NCT00444964
First Posted
March 6, 2007
Last Updated
December 15, 2020
Sponsor
Children's Mercy Hospital Kansas City
1. Study Identification
Unique Protocol Identification Number
NCT00444964
Brief Title
Growth Hormone Use in Adults With Prader-Willi Syndrome
Official Title
Growth Hormone Use in Adults With Prader-Willi Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
December 2020
Overall Recruitment Status
Completed
Study Start Date
April 2005 (Actual)
Primary Completion Date
January 1, 2012 (Actual)
Study Completion Date
January 1, 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Mercy Hospital Kansas City
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The main research question this protocol aims to answer is whether treatment with growth hormone will impact body composition, quality of life, and energy balance in PWS adults, and if there is a loss of effects after cessation of treatment for at least 12 months.
Detailed Description
The main research question this protocol aims to answer is whether treatment with growth hormone will impact body composition, quality of life, and energy balance in PWS adults, and if there is a loss of effects after cessation of treatment for at least 12 months. Specific outcomes to be evaluated are as follows:
• Increased IGF-1 as a function of human growth hormone dosage compared with baseline.
Improvement of indicators or risk factors for co-morbid diseases [diabetes (by measuring insulin and glucose levels), cardiovascular disease (by measuring lipids and fatty acids), and pulmonary function] in participants.
Improvement in quality of life measures as indicated by ratings on established behavior checklists in participants.
Changes in body composition (decreased fat, increased lean body mass and bone density) as determined by DEXA in participants.
Increased energy expenditure as determined by whole-room calorimeter measures (8 hour energy expenditure, RMR, TEF, mechanical work); diet records, physical activity monitors and strength measures.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome
7. Study Design
Primary Purpose
Diagnostic
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
11 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Primary Cohort
Arm Type
Experimental
Arm Description
Nutropin AQ
Intervention Type
Drug
Intervention Name(s)
Nutropin AQ
Other Intervention Name(s)
Growth Hormone
Intervention Description
0.0125 mg/kg/day
Primary Outcome Measure Information:
Title
Body Composition
Description
Anthropometric Measures and Body Composition
Time Frame
12 and 24 months
Title
Blood Chemistry
Description
Insulin-like growth factor I- IGF-1
Time Frame
12 and 24 months
Title
Physical Activity
Description
Duration of daily physical activity
Time Frame
12 and 24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
16 to 60 years old
Male or female with diagnosed Prader-Willi syndrome confirmed by genetic testing (e.g., mPCR)
Low IGF-1 level (e.g.,≤25%) at baseline
Ability to provide informed consent or availability of a suitable legally authorized representative
Exclusion Criteria:
Pregnancy
Previous treatment with growth hormone
Uncontrolled endocrine disease, (i.e. diabetes or thyroid)
History of severe scoliosis
Heart disease
Uncontrolled high blood pressure or history of stroke
Morbid obesity (using PWS growth charts)
Severe sleep apnea or known breathing difficulties/obstruction (as per history or diagnostic testing results
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Merlin G Butler, MD
Organizational Affiliation
Children's Mercy Hospital Kansas City
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Growth Hormone Use in Adults With Prader-Willi Syndrome
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