Growth Hormone Use in Adults With Prader-Willi Syndrome

Prader-Willi Syndrome Clinical Trial

Official title:

Growth Hormone Use in Adults With Prader-Willi Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00444964
• Other study ID #: 303-C02R
• Status: Completed
• Phase: Phase 3
• Start date: April 2005
• Est. completion date: January 1, 2012

Study information

• Verified date: December 2020
• Source: Children's Mercy Hospital Kansas City
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. completion date: January 1, 2012
• Est. primary completion date: January 1, 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years to 60 Years

Eligibility

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

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• Nutropin AQ
0.0125 mg/kg/day

Locations

Country	Name	City	State
United States	The Children's Mercy Hospital	Kansas City	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Children's Mercy Hospital Kansas City

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Body Composition	Anthropometric Measures and Body Composition	12 and 24 months
Primary	Blood Chemistry	Insulin-like growth factor I- IGF-1	12 and 24 months
Primary	Physical Activity	Duration of daily physical activity	12 and 24 months