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Growth Hormone Use in Adults With Prader-Willi Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00444964
Recruitment Status : Unknown
Verified March 2007 by Children's Mercy Hospital Kansas City.
Recruitment status was:  Recruiting
First Posted : March 8, 2007
Last Update Posted : March 8, 2007
Sponsor:
Information provided by:

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.

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: Nutropin AQ Phase 3

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.

  1. 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.
  2. Improvement in quality of life measures as indicated by ratings on established behavior checklists in participants.
  3. Changes in body composition (decreased fat, increased lean body mass and bone density) as determined by DEXA in participants.
  4. 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.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: Growth Hormone Use in Adults With Prader-Willi Syndrome
Study Start Date : April 2005
Estimated Study Completion Date : February 2007


Arms and Interventions


Outcome Measures

Primary Outcome Measures :
  1. Increased IGF-1 as a function of human growth hormone dosage compared with baseline
  2. 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.
  3. Improvement in quality of life measures as indicated by ratings on established behavior checklists in participants.
  4. Changes in body composition (decreased fat, increased lean body mass and bone density) as determined by DEXA in participants.
  5. 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 Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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

ClinicalTrials.gov Identifier: NCT00444964     History of Changes
Other Study ID Numbers: 303-C02R
First Posted: March 8, 2007    Key Record Dates
Last Update Posted: March 8, 2007
Last Verified: March 2007

Additional relevant MeSH terms:
Syndrome
Prader-Willi Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs