Growth Hormone Use in Adults With Prader-Willi Syndrome - Full Text View

Sponsored by:	Children's Mercy Hospital Kansas City
Information provided by:	Children's Mercy Hospital Kansas City
ClinicalTrials.gov Identifier:	NCT00444964

Purpose

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.

Condition	Intervention	Phase
Prader-Willi Syndrome	Drug: Nutropin AQ	Phase III

Genetics Home Reference related topics: Prader-Willi syndrome

MedlinePlus related topics: Prader-Willi Syndrome

Drug Information available for: Somatotropin Somatropin

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Diagnostic, Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Growth Hormone Use in Adults With Prader-Willi Syndrome

Further study details as provided by Children's Mercy Hospital Kansas City:

Primary Outcome Measures:

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

Estimated Enrollment:	10
Study Start Date:	April 2005
Estimated Study Completion Date:	February 2007

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.

Eligibility

Ages Eligible for Study:	16 Years to 60 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00444964

Locations

United States, Missouri
The Children's Mercy Hospital	Recruiting
Kansas City, Missouri, United States, 64108
Contact: Merlin G Butler, MD 816-234-3290 mgbutler@cmh.edu
Contact: Wayne Moore, MD 816-234-3844 wmoore@cmh.edu
Principal Investigator: Merlin G Butler, MD

Sponsors and Collaborators

Children's Mercy Hospital Kansas City

Investigators

Principal Investigator:

Merlin G Butler, MD

The Children's Mercy Hospital

More Information

Study ID Numbers:	303-C02R
Study First Received:	March 6, 2007
Last Updated:	March 7, 2007
ClinicalTrials.gov Identifier:	NCT00444964
Health Authority:	United States: Institutional Review Board

Study placed in the following topic categories:

Chromosomal abnormalities
Obesity
Chromosome Disorders
Prader-Willi syndrome
Mental Retardation
Genetic Diseases, Inborn
Abnormalities, Multiple

Neurologic Manifestations
Nutrition Disorders
Overnutrition
Prader-Willi Syndrome
Congenital Abnormalities
Neurobehavioral Manifestations

Additional relevant MeSH terms:

Pathologic Processes
Disease
Syndrome
Nervous System Diseases

ClinicalTrials.gov processed this record on January 16, 2009